Document and Entity Information
Document and Entity Information - USD ($) $ in Billions | 12 Months Ended | ||
Dec. 31, 2023 | Feb. 20, 2024 | Jun. 30, 2023 | |
Cover [Abstract] | |||
Document Type | 10-K/A | ||
Amendment Flag | true | ||
Amendment Description | Apellis Pharmaceuticals, Inc. (the “Company”) filed its Annual Report on Form 10-K for the fiscal year ended December 31, 2023 (the “Original Filing”) with the United States Securities and Exchange Commission (the “SEC”) on February 27, 2024. The Company is filing this Amendment No. 1 to the Original Filing (this “Amendment No. 1”) in order to correct (i) an inadvertent administrative error in the Critical Audit Matter section of the Report of Independent Registered Public Accounting Firm in Part II, Item 8 that resulted in an omission of text in the Original Filing and (ii) an inadvertent administrative error that resulted in the omission of the Report of Independent Registered Public Accounting Firm on internal controls over financial reporting, in Part II, Item 9A of the Original Filing. In accordance with Rule 12b-15 (“Rule 12b-15”) under the Securities Exchange Act of 1934, as amended, the Original Filing is amended and restated in its entirety. However, there have been no changes made other than the corrections stated in the immediately preceding paragraph. In addition, the Company is including in this Amendment No. 1 new certifications of its principal executive officer and principal financial officer, as required by Rule 12b-15, as Exhibits 31.3, 31.4, 32.3 and 32.4, respectively, and a new consent from Deloitte & Touche LLP, the Company’s independent registered public accounting firm, as Exhibit 23.2. Except as described above, this Amendment No. 1 speaks as of the filing date of the Original Filing and does not amend or modify any other information contained in the Original Filing to reflect events that may have occurred subsequent to such filing date. Accordingly, this Amendment No. 1 should be read in conjunction with the Original Filing and the Company’s subsequent filings with the SEC. | ||
Document Period End Date | Dec. 31, 2023 | ||
Document Fiscal Year Focus | 2023 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | APLS | ||
Entity Registrant Name | APELLIS PHARMACEUTICALS, INC. | ||
Entity Central Index Key | 0001492422 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
Entity Shell Company | false | ||
Entity Common Stock, Shares Outstanding | 120,581,749 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
Entity Public Float | $ 10.7 | ||
Entity Current Reporting Status | Yes | ||
Entity File Number | 001-38276 | ||
Entity Tax Identification Number | 27-1537290 | ||
Entity Address, Address Line One | 100 Fifth Avenue | ||
Entity Address, City or Town | Waltham | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02451 | ||
City Area Code | 617 | ||
Local Phone Number | 977-5700 | ||
Entity Interactive Data Current | Yes | ||
Security 12b Title | Common Stock, $0.0001 par value per share | ||
Security Exchange Name | NASDAQ | ||
Entity Incorporation, State or Country Code | DE | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Document Financial Statement Error Correction | false | ||
ICFR Auditor Attestation Flag | true | ||
Auditor Name | Deloitte & Touche LLP | ||
Auditor Firm ID | 34 | ||
Auditor Location | Boston, Massachusetts | ||
Documents Incorporated by Reference | The registrant intends to file a definitive proxy statement pursuant to Regulation 14A in connection with its 2024 Annual Meeting of Stockholders within 120 days of the end of the registrant’s fiscal year ended December 31, 2023. Portions of such proxy statement are incorporated by reference into Part III of this Annual Report on Form 10-K . |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 351,185 | $ 551,801 |
Accounts receivable | 206,442 | 7,727 |
Inventory | 146,362 | 85,714 |
Prepaid assets | 38,820 | 36,350 |
Restricted cash | 1,114 | 1,273 |
Other current assets | 22,408 | 36,658 |
Total current assets | 766,331 | 719,523 |
Non-current assets: | ||
Right-of-use assets | 16,745 | 18,747 |
Property and equipment, net | 4,345 | 6,148 |
Other assets | 1,309 | 15,799 |
Total assets | 788,730 | 760,217 |
Current liabilities: | ||
Accounts payable | 37,516 | 37,342 |
Accrued expenses | 127,806 | 95,139 |
Current portion of development liability | 75,830 | 29,504 |
Current portion of right-of-use liabilities | 6,441 | 5,625 |
Total current liabilities | 247,593 | 167,610 |
Long-term liabilities: | ||
Long-term development liability | 239,817 | 315,647 |
Convertible senior notes | 93,033 | 92,736 |
Right-of-use liabilities | 11,454 | 14,352 |
Other liabilities | 2,312 | |
Total liabilities | 594,209 | 590,345 |
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares issued and outstanding at December 31, 2023 and 2022 | 0 | 0 |
Common stock, $0.0001 par value; 200,000 shares authorized at December 31, 2023 and 2022; 119,556 and 110,772 shares issued and outstanding at December 31, 2023 and 2022, respectively | 12 | 11 |
Additional paid-in capital | 3,035,539 | 2,479,596 |
Accumulated other comprehensive loss | (3,542) | (875) |
Accumulated deficit | (2,837,488) | (2,308,860) |
Total stockholders’ equity | 194,521 | 169,872 |
Total liabilities and stockholders’ equity | $ 788,730 | $ 760,217 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, issued | 0 | 0 |
Preferred stock, outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, issued | 119,556,000 | 110,772,000 |
Common stock, outstanding | 119,556,000 | 110,772,000 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Revenue: | |||
Total product revenue, net | $ 396,591 | $ 75,422 | $ 66,563 |
Operating expenses: | |||
Cost of sales | 58,510 | 5,636 | 200 |
Research and development | 354,387 | 387,236 | 345,869 |
Cost of research collaboration | 75,000 | ||
License expense | 5,000 | ||
General and administrative | 500,815 | 277,163 | 176,771 |
Total operating expenses: | 913,712 | 670,035 | 602,840 |
Net operating loss | (517,121) | (594,613) | (536,277) |
Loss on conversion of debt | (32,890) | (100,589) | |
Loss from remeasurement of development derivative liability | (97,675) | ||
Interest income | 20,933 | 8,914 | 418 |
Interest expense | (29,581) | (32,626) | (13,241) |
Other(expense)/ income, net | (727) | (288) | 1,362 |
Net loss before taxes | (526,496) | (651,503) | (746,002) |
Income tax expense | 2,132 | 669 | 352 |
Net loss | (528,628) | (652,172) | (746,354) |
Other comprehensive income/(loss): | |||
Unrealized (loss)/gain on marketable securities | (1) | 9 | |
Unrealized (loss)/gain on pension plans | (2,618) | 1,646 | |
Foreign currency (loss) | (49) | (430) | (1,982) |
Total other comprehensive income/(loss) | (2,667) | 1,215 | (1,973) |
Comprehensive loss, net of tax | $ (531,295) | $ (650,957) | $ (748,327) |
Net loss per common share, basic | $ (4.45) | $ (6.15) | $ (8.84) |
Net loss per common share, diluted | $ (4.45) | $ (6.15) | $ (8.84) |
Weighted-average number of common shares used in net loss per common share, basic | 118,678 | 106,114 | 84,421 |
Weighted-average number of common shares used in net loss per common share, diluted | 118,678 | 106,114 | 84,421 |
Product revenue, net [Member] | |||
Revenue: | |||
Total product revenue, net | $ 366,281 | $ 65,092 | $ 15,147 |
Licensing and other revenue [Member] | |||
Revenue: | |||
Total product revenue, net | $ 30,310 | $ 10,330 | $ 51,416 |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholder's Equity - USD ($) shares in Thousands, $ in Thousands | Total | Cumulative Effect, Period of Adoption, Adjustment [Member] | Common Stock [Member] | Additional Paid-In Capital [Member] | Additional Paid-In Capital [Member] Cumulative Effect, Period of Adoption, Adjustment [Member] | Accumulated Other Comprehensive Loss [Member] | Accumulated Deficit [Member] | Accumulated Deficit [Member] Cumulative Effect, Period of Adoption, Adjustment [Member] |
Beginning balance at Dec. 31, 2020 | $ 204,557 | $ (149,734) | $ 8 | $ 1,131,013 | $ (165,747) | $ (117) | $ (926,347) | $ 16,013 |
Beginning balance, Shares at Dec. 31, 2020 | 76,130 | |||||||
Accounting Standards Update description | ASU 2020-06 | |||||||
Issuance of common stock in follow-on offering, net of offering costs | $ 380,362 | $ 1 | 380,361 | |||||
Issuance of common stock in follow-on offering, shares | 10,063 | |||||||
Issuance of common stock upon exercise of stock options | 14,691 | 14,691 | ||||||
Issuance of common stock upon exercise of stock options, shares | 1,063 | |||||||
Issuance of shares in exchange of Convertible Notes, including issuance cost | 421,093 | $ 1 | 421,092 | |||||
Issuance of shares in exchange of Convertible Notes, including issuance cost, shares | 10,065 | |||||||
Forfeiture of accrued interest in exchange of Convertible Notes | 4,171 | 4,171 | ||||||
Vesting of restricted stock units, net of shares withheld for taxes | (1,788) | (1,788) | ||||||
Vesting of restricted stock units, net of shares withheld for taxes. shares | 91 | |||||||
Share-based compensation expense | 70,667 | 70,667 | ||||||
Issuance of common stock to employee stock purchase plan | 2,970 | 2,970 | ||||||
Issuance of common stock to employee stock purchase plan, shares | 112 | |||||||
Unrealized gain (loss) on available-for-sale investments | 9 | 9 | ||||||
Net loss | (746,354) | (746,354) | ||||||
Foreign currency loss | (1,982) | (1,982) | ||||||
Ending balance at Dec. 31, 2021 | 198,662 | $ 10 | 1,857,430 | (2,090) | (1,656,688) | |||
Ending balance, Shares at Dec. 31, 2021 | 97,524 | |||||||
Issuance of common stock in follow-on offering, net of offering costs | 380,120 | $ 1 | 380,119 | |||||
Issuance of common stock in follow-on offering, shares | 8,564 | |||||||
Issuance of common stock upon exercise of stock options | 21,483 | 21,483 | ||||||
Issuance of common stock upon exercise of stock options, shares | 1,223 | |||||||
Issuance of shares in exchange of Convertible Notes, including issuance cost | 129,636 | 129,636 | ||||||
Issuance of shares in exchange of Convertible Notes, including issuance cost, shares | 3,073 | |||||||
Forfeiture of accrued interest in exchange of Convertible Notes | 1,287 | 1,287 | ||||||
Vesting of restricted stock units, net of shares withheld for taxes | (5,682) | (5,682) | ||||||
Vesting of restricted stock units, net of shares withheld for taxes. shares | 252 | |||||||
Share-based compensation expense | 91,085 | 91,085 | ||||||
Issuance of common stock to employee stock purchase plan | 4,238 | 4,238 | ||||||
Issuance of common stock to employee stock purchase plan, shares | 136 | |||||||
Unrealized gain (loss) on available-for-sale investments | (1) | (1) | ||||||
Unrealized gain (loss) on pension benefit plan | 1,646 | 1,646 | ||||||
Net loss | (652,172) | (652,172) | ||||||
Foreign currency loss | (430) | (430) | ||||||
Ending balance at Dec. 31, 2022 | 169,872 | $ 11 | 2,479,596 | (875) | (2,308,860) | |||
Ending balance, Shares at Dec. 31, 2022 | 110,772 | |||||||
Issuance of common stock and pre-funded warrants in common stock offering | 384,387 | $ 1 | 384,386 | |||||
Issuance of common stock and pre-funded warrants in common stock offering, shares | 4,008 | |||||||
Issuance of common stock upon exercise of stock options | $ 71,274 | 71,274 | ||||||
Issuance of common stock upon exercise of stock options, shares | 3,950 | 3,858 | ||||||
Vesting of restricted stock units, net of shares withheld for taxes | $ (11,040) | (11,040) | ||||||
Vesting of restricted stock units, net of shares withheld for taxes. shares | 806 | |||||||
Share-based compensation expense | 105,945 | 105,945 | ||||||
Issuance of common stock to employee stock purchase plan | 5,378 | 5,378 | ||||||
Issuance of common stock to employee stock purchase plan, shares | 112 | |||||||
Unrealized gain (loss) on pension benefit plan | (2,618) | (2,618) | ||||||
Net loss | (528,628) | (528,628) | ||||||
Foreign currency loss | (49) | (49) | ||||||
Ending balance at Dec. 31, 2023 | $ 194,521 | $ 12 | $ 3,035,539 | $ (3,542) | $ (2,837,488) | |||
Ending balance, Shares at Dec. 31, 2023 | 119,556 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Operating Activities | |||
Net loss | $ (528,628) | $ (652,172) | $ (746,354) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Share-based compensation expense | 105,945 | 91,085 | 70,667 |
Loss on conversion of debt | 32,890 | 100,589 | |
Loss from remeasurement of development derivative liability | 97,675 | ||
Loss on disposal of fixed assets | 120 | ||
Forfeiture of accrued interest in exchange of convertible notes | 1,287 | 4,171 | |
Depreciation expense | 1,784 | 1,552 | 1,379 |
Amortization of right of use assets | (80) | (65) | 113 |
Amortization of discounts for convertible notes, net of financing costs | 297 | 459 | 964 |
Accretion of discount to development liability | 25,996 | 26,917 | 1,192 |
Changes in operating assets and liabilities: | |||
Accounts receivable | (198,715) | 2,375 | (10,103) |
Inventory | (60,647) | (69,397) | (16,317) |
Prepaid assets | (1,870) | (11,479) | (13,487) |
Other current assets | 14,243 | 32,936 | (40,928) |
Other assets | 11,700 | 17,490 | (12,782) |
Accounts payable | 170 | 18,689 | 10,487 |
Accrued expenses | 34,950 | (6,312) | (10,392) |
Net cash used in operating activities | (594,735) | (513,745) | (563,126) |
Investing Activities | |||
Purchase of property and equipment | (773) | (1,524) | (1,103) |
Proceeds from sale of fixed assets | 99 | ||
Purchase of available-for-sale securities | (331,863) | (171,281) | |
Proceeds from maturity of available-for-sale securities | 393,280 | 420,000 | |
Net cash (used in) provided by investing activities | (674) | 59,893 | 247,616 |
Financing Activities | |||
Proceeds from issuance of common stock, net of issuance costs | 380,120 | 380,363 | |
Proceeds from issuance of common stock, pre-funded warrant offering, net of issuance costs | 384,387 | ||
Payments for development derivative liability | (4,000) | ||
Payments for development liability | (55,500) | (34,500) | |
Proceeds from exercise of stock options | 71,274 | 21,483 | 14,691 |
Proceeds from issuance of common stock under employee share purchase plan | 5,378 | 4,238 | 2,970 |
Payments of employee tax withholding related to equity-based compensation | (11,040) | (5,682) | (1,788) |
Net cash provided by financing activities | 394,499 | 365,659 | 392,236 |
Effect of exchange rate changes on cash and cash equivalents | 135 | (488) | (2,016) |
Net (decrease) increase in cash and cash equivalents | (200,775) | (88,681) | 74,710 |
Cash, cash equivalents and restricted cash at beginning of period | 553,074 | 641,755 | 567,045 |
Cash, cash equivalents and restricted cash at end of period | 352,299 | 553,074 | 641,755 |
Reconciliation of cash, cash equivalents and restricted cash to the consolidated balance sheets: | |||
Cash and cash equivalents | 351,185 | 551,801 | 640,192 |
Restricted cash | 1,114 | 1,273 | 1,563 |
Total cash, cash equivalents, and restricted cash | 352,299 | 553,074 | 641,755 |
Supplemental disclosure of cash flow information: | |||
Cash paid for interest | 3,286 | 5,003 | 10,265 |
Cash paid for income taxes | 4,915 | 49 | |
Proceeds from income tax refunds net of income taxes paid | $ 1,759 | ||
Convertible Notes exchanged for common stock | $ 98,086 | $ 328,017 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Pay vs Performance Disclosure | |||
Net Income (Loss) | $ (528,628) | $ (652,172) | $ (746,354) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Dec. 31, 2023 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | The following table describes, for the quarterly period covered by this report, each trading arrangement for the sale or purchase of our securities adopted or terminated by our directors and officers that is either (1) a contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c), or a Rule 10b5-1 trading arrangement, or (2) a “non-Rule 10b5-1 trading arrangement” (as defined in Item 408(c) of Regulation S-K): Name (Title) Action Taken Type of Trading Nature of Trading Duration of Trading Aggregate Number of Cedric Francois President and Chief Executive Adoption 11/30/2023 Rule 10b5-1 trading arrangement Sale Until 2/28/2025 , or such earlier date upon which all transactions are completed or expire without execution Up to 420,000 shares Nur Nicholson Chief Technical Operations Termination 12/01/2023 Rule 10b5-1 trading arrangement Sale (1 ) (1 ) Timothy E Sullivan Chief Financial Officer Adoption 12/01/2023 Rule 10b5-1 trading arrangement Sale Until 2/28/2025 , or such earlier date upon which all transactions are completed or expire without execution Up to 150,000 shares Pascal Deschatelets Chief Scientific Officer Adoption 12/03/2023 Rule 10b5-1 trading arrangement Sale Until 9/09/2024 , or such earlier date upon which all transactions are completed or expire without execution Up to 296,028 shares (1) This trading plan related to 67,000 shares of our common stock and had a scheduled expiration date of 12/27/2024 . |
Cedric Francois [Member] | |
Trading Arrangements, by Individual | |
Name | Cedric Francois |
Title | President and Chief ExecutiveOfficer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | 11/30/2023 |
Arrangement Duration | 457 days |
Aggregate Available | 420,000 |
Trd Arr Expiration Date | 2/28/2025 |
Nur Nicholson [Member] | |
Trading Arrangements, by Individual | |
Name | Nur Nicholson |
Title | Chief Technical Operations |
Rule 10b5-1 Arrangement Terminated | true |
Termination Date | 12/01/2023 |
Arrangement Duration | 393 days |
Aggregate Available | 67,000 |
Trd Arr Expiration Date | 12/27/2024 |
Timothy E Sullivan [Member] | |
Trading Arrangements, by Individual | |
Name | Timothy E Sullivan |
Title | Chief Financial Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | 12/01/2023 |
Arrangement Duration | 456 days |
Aggregate Available | 150,000 |
Trd Arr Expiration Date | 2/28/2025 |
Pascal Deschatelets [Member] | |
Trading Arrangements, by Individual | |
Name | Pascal Deschatelets |
Title | Chief Scientific Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | 12/03/2023 |
Arrangement Duration | 282 days |
Aggregate Available | 296,028 |
Trd Arr Expiration Date | 9/09/2024 |
Nature of Organization and Oper
Nature of Organization and Operations | 12 Months Ended |
Dec. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of Organization and Operations | 1. Nature of Organization and Operations Apellis Pharmaceuticals, Inc. (the “Company”) is a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic compounds to treat diseases with high unmet needs through the inhibition of the complement system, which is an integral component of the immune system, at the level of C3, the central protein in the complement cascade. The Company was incorporated in September 2009 under the laws of the State of Delaware. The Company’s principal executive offices are located in Waltham, Massachusetts. The Company’s operations since inception have been limited to organizing and staffing the Company, acquiring rights to product candidates, business planning, raising capital, developing its product candidates, commercializing EMPAVELI (pegcetacoplan) for the treatment of paroxysmal nocturnal hemoglobinuria (“PNH”) and commercializing SYFOVRE (pegcetacoplan injection) for the treatment of geographic atrophy secondary to age-related macular degeneration (“GA”). The Company is subject to risks common in the biotechnology industry including, but not limited to, raising additional capital, development by its competitors of new technological innovations, its ability to successfully complete preclinical and clinical development of product candidates and receive timely regulatory approval of products, market acceptance of the Company’s products, protection of proprietary technology, healthcare cost containment initiatives, and compliance with governmental regulations, including those of the U.S. Food and Drug Administration (“FDA”). Follow-on Public Offerings On February 22, 2023, the Company issued and sold 4,007,936 shares of common stock and, in lieu of common stock to investor who so chose, pre-funded warrants to purchase 2,380,956 shares of common stock in a follow-on offering, including 833,333 shares sold pursuant to the underwriters’ exercise in full of their option to purchase additional shares of common stock. The price to the public of the shares of common stock was $ 63.00 per share and the price to the public of the pre-funded warrants was $ 62.9999 per pre-funded warrant. The pre-funded warrants have an exercise price equal to $ 0.0001 per share and do not expire. The pre-funded warrants were accounted for as equity instruments. The Company received total net proceeds of $ 384.4 million, after deducting underwriting discounts and commissions of $ 18.8 million and offering costs of $ 0.3 million. On March 28, 2022, the Company issued and sold 8,563,830 shares of its common stock at a price per share to the public of $ 47.00 in a follow-on public offering including an additional 1,117,021 shares of its common stock that were sold at the follow-on public offering price of $ 47.00 per share pursuant to the underwriters’ agreement in full exercise of their option to purchase additional shares of common stock. The Company received net proceeds of approximately $ 380.1 million after deducting underwriting discounts and commissions of approximately $ 22.1 million and offering costs of $ 0.3 million for these transactions. On November 18, 2021, the Company issued and sold 10,062,500 shares of its common stock at a price per share to the public of $ 40.00 in a follow-on public offering including an additional 1,312,500 shares of its common stock that were sold at the follow-on public offering price of $ 40.00 per share pursuant to the underwriters’ agreement in full exercise of their option to purchase additional shares of common stock. The Company received net proceeds of approximately $ 380.4 million after deducting underwriting discounts and commissions of approximately $ 22.1 million and offering costs of $ 0.6 million for these transactions. Liquidity and Going Concern The accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern. From inception to December 31, 2023, the Company has incurred cash outflows from operations, losses from operations, and had an accumulated deficit of $ 2.8 billion, primarily as a result of expenses incurred through a combination of research and development activities related to the Company’s various product candidates and expenses supporting those activities. The Company has primarily financed its operations through public offerings of its common stock, convertible debt, private placements of preferred stock prior to its initial public offering, the development funding agreement with SFJ, and the collaboration agreement with Sobi. The Company has financed a portion of its operations through product sales but has not yet achieved profitability. The Company believes that its cash and cash equivalents of $ 351.2 million at December 31, 2023 and the cash anticipated to be generated from the unwind of the capped call transactions, together with cash anticipated to be generated from sales of EMPAVELI and from SYFOVRE will be sufficient to fund its operations and capital expenditure requirements for at least twelve months from the date of issuance of these consolidated financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States (“U.S. GAAP”) and following the requirements of the Securities and Exchange Commission (the “SEC”). Revenue Recognition The Company’s revenues consist of product sales of EMPAVELI and SYFOVRE and revenue derived from its collaboration arrangement with Sobi. See Note 12, License and Collaboration Agreements for further discussion related to the Sobi Collaboration and License Agreement. The Company accounts for contracts with its customers in accordance with ASC Topic 606, Revenue from Contracts with Customers, ( “ASC 606”). Pursuant to ASC 606, for arrangements or transactions between participants determined to be within the scope of the contracts with customers guidance, the Company performs the following five steps to determine the appropriate amount of revenue to be recognized as the Company fulfills its obligations: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. Revenue is recognized when, or as, the Company satisfies a performance obligation by transferring a promised good or service to a customer. An asset is transferred when, or as, the customer obtains control of that asset. For performance obligations that are satisfied over time, the Company recognizes revenue using an input or output measure of progress that best depicts the satisfaction of the relevant performance obligation. Product Revenue The Company’s revenue from net product sales was generated in the United States following the FDA’s approval for marketing of EMPAVELI for the treatment of PNH in May 2021 and SYFOVRE in February 2023. The Company sells EMPAVELI and SYFOVRE principally through arrangements with specialty pharmacies (“SPs”) and specialty distributors (“SDs”), who are the Company’s customers. The customers subsequently resell the product to patients and health care providers. The Company applies the ASC 606 five step process discussed above to the contracts with SPs and SDs. The Company provides limited right of return to the customers in cases of shipment errors or expiring or defective products. Product revenues are recognized when the customers take control of the product, which typically occurs upon delivery to the customers. The Company recognizes revenue from product sales at the net sales price which includes estimates of variable consideration for which reserves are established and reflects each of these as a reduction to revenue. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which the Company is entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from estimates, the Company may need to adjust its estimates, which would affect net revenue in the period of adjustment. The following are the Company’s significant categories of variable consideration: Distribution Fees : Distribution fees include distribution service fees paid to SPs and SDs based on a contractually fixed percentage of the wholesale acquisition cost (WAC). The Company does not receive a distinct good or service in exchange for the payment. Distribution fees are recorded as an offset to revenue based on contractual terms at the time revenue from the sale is recognized. Chargebacks : Chargebacks are discounts and fees related to contracts with various third-party payers including pharmacy benefit managers, private healthcare insurers and government healthcare programs that purchase from SDs at a discounted price. SDs charge back to the Company the difference between the price initially paid by SDs and the discounted price paid to SDs by these entities. The Company issues credit notes for the chargeback which are applied to future sales. Product Returns : Consistent with industry practice, the Company offers SPs and SDs limited product return rights for shipment errors or expiring or defective products; provided that the return is within a specified period around the product expiration date as set forth in the applicable individual distribution agreement. The Company does not allow product returns for product that has been dispensed to a patient. As the Company receives inventory reports from SPs and SDs and has visibility into the inventory distribution channel, it is able to make a reasonable estimate of future potential product returns based on this on-hand channel inventory data and sell-through data obtained from SPs and SDs. In arriving at its estimate for product returns, the Company also considers historical product returns (to the extent available), the underlying product demand, and industry data specific to the specialty pharmaceutical distribution industry. Licensing and Collaboration Revenue The Company analyzes license and collaboration arrangements pursuant to ASC Topic 808, Collaborative Arrangement Guidance and Considerations, (“ASC 808”) to assess whether such arrangements, or transactions between arrangement participants, involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities or are more akin to a vendor-customer relationship. In making this evaluation, the Company considers whether the activities of the collaboration are considered to be distinct and deemed to be within the scope of the collaborative arrangement guidance or if they are more reflective of a vendor-customer relationship and, therefore, within the scope of ASC 606. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For elements of collaboration arrangements that are not accounted for pursuant to guidance in ASC 606, an appropriate recognition method is determined and applied consistently, generally by analogy to the revenue from contracts with customers guidance. Amounts related to transactions with a counterparty in a collaborative arrangement that is not a customer are presented as collaboration revenue and in a separate line item from revenue recognized from contracts with customers, if any, in the consolidated statements of operations. Pursuant to ASC 606, for arrangements or transactions between arrangement participants determined to be within the scope of the contracts with customers guidance, the Company performs the five-step process discussed above to determine the appropriate amount of revenue to be recognized as the Company fulfills its obligations. We evaluate the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determine whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract. Goods or services that meet these criteria are considered distinct performance obligations. The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential transaction price and the likelihood that the transaction price will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and, if so, these options are considered performance obligations. The Company has not currently identified any such material rights. Revenue is recognized when, or as, the Company satisfies a performance obligation by transferring a promised good or service to a customer. An asset is transferred when, or as, the customer obtains control of that asset. For performance obligations that are satisfied over time, the Company recognizes revenue using an input or output measure of progress that best depicts the satisfaction of the relevant performance obligation. After contract inception, the transaction price is reassessed at every period end and updated for changes such as resolution of uncertain events. Any change in the overall transaction price is allocated to the performance obligations on the same methodology as at contract inception. See Note 12, License and Collaboration Agreements, for further discussion related to the Sobi collaboration agreement. Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results may differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes, and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: development liability, accrued expenses, prepaid expenses, convertible debt, reserves for variable consideration, reserves for excess or obsolete inventories, and income taxes. Fair Value of Financial Instruments The Company is required to disclose information on the fair value of financial instruments and inputs that enable an assessment of the fair value. The three levels of the fair value hierarchy prioritize valuation inputs based upon the observable nature of those inputs as follows: Level 1 – Quoted prices in active markets for identical assets or liabilities; Level 2 – Inputs other than quoted prices included within level 1 that are observable for the asset or liability, either directly or indirectly; Level 3 – Unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability. The Company’s financial instruments, in addition to those presented in Note 8, Long-term Debt, and Note 11, Fair Value Measurements, include cash and cash equivalents, accounts payable and accrued liabilities. Management believes that the carrying amounts of cash and cash equivalents, accounts payable and accrued expenses approximate the fair value due to the short-term nature of those instruments. Cash and Cash Equivalents Cash and cash equivalents are defined as cash in banks and investment instruments having maturities of three months or less from their acquisition date. The carrying amounts reported in the consolidated balance sheets for cash and cash equivalents are valued at cost, which approximates the fair value. See Note 11, Fair Value Measurements, for additional information. Accounts Receivable The Company’s accounts receivable primarily arise from product sales. They are generally stated at the invoiced amount and do not bear interest. The accounts receivable from product sales represents receivables due from the Company’s SPs or SDs. The Company has had no historical write offs of its accounts receivable as of December 31, 2023, and its payment terms are generally 30-65 days for EMPAVELI and 60-150 days for SYFOVRE. The Company monitors the financial performance and creditworthiness of its customers and provides reserves against trade receivables for expected credit losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2023 and 2022, the credit pr ofiles for the Company’s customers were deemed to be in good standing and an allowance for credit losses was not considered necessary. Inventory Inventory is recorded at the lower of cost or net realizable value, with cost determined on a first-in, first-out basis. Inventory costs include third-party contract manufacturing, third-party packaging services, and freight. The Company performs an assessment of the recoverability of capitalized inventory during each reporting period, and it writes down any excess and obsolete inventories to their estimated realizable value in the period in which the impairment is first identified. Such impairment charges, should they occur, are recorded within cost of sales. The determination of whether inventory costs will be realizable requires estimates by management. If actual market conditions are less favorable than projected by management, additional write-downs of inventory may be required which would be recorded as a cost of sales in the consolidated statements of operations and comprehensive loss. Prior to regulatory approval of its product candidates, the Company expensed costs associated with the manufacturing of its product candidates to research and development expense unless the Company was reasonably certain such costs have future commercial use and net realizable value. When the Company believes regulatory approval and subsequent commercialization of its product candidates is probable, and the Company also expects future economic benefit from the sales of the product candidates to be realized, the Company will then capitalize the costs of production as inventory. Inventory that can be used in either the production of clinical or commercial product is expensed as research and development expense when selected for use in a clinical manufacturing campaign. Prior to receiving FDA approval for EMPAVELI on May 14, 2021, the Company included in research and development expense the costs associated with the manufacture of EMPAVELI inventory to be sold upon commercialization. As a result, the manufacturing costs related to the EMPAVELI inventory build-up incurred before FDA approval were expensed in a prior period and are, therefore, excluded from the cost of goods sold and inventory. As of December 31, 2023, the remaining pre-FDA approved inventory was $ 19.4 million, which primarily consisted of raw materials. Shipping and handling costs for product shipments are recorded as incurred in cost of sales along with costs associated with manufacturing the product and any inventory write-downs. Development Liability From December 15, 2021 and thereafter until the final annual payment due December 2027, the development liability will be accreted from its initial carrying amount to the total payment amount using the effective interest rate method under ASC 835 over the remaining life of the SFJ agreement as defined in Note 6. The difference between the carrying amount and the total payment amount is presented as discount to the development liability. The accretion is recorded as interest expense in the consolidated income statement. Foreign Currency The functional currency of each of the Company’s subsidiaries is its local currency, except for the wholly owned subsidiaries in Switzerland and Netherlands where the functional currency is the U.S. dollar. Revenues and expenses of the subsidiaries have been translated into U.S. dollars at average exchange rates prevailing during the respective periods. Assets and liabilities have been translated at the rates of exchange on the balance sheet date. The resulting translation gain and loss adjustments are recorded directly as a separate component of stockholders’ equity. Research and Development Costs incurred in connection with research and development activities are expensed as incurred. Research and development expenses include (i) employee-related expenses, including salaries, benefits, travel and share-based compensation expense; (ii) external research and development expenses incurred under arrangements with third parties, such as contract research and contract manufacturing organizations, investigational sites and consultants, including share-based compensation expense for consultants; (iii) the cost of acquiring, developing and manufacturing clinical study materials; and (iv) costs associated with preclinical and clinical activities and regulatory operations. The Company enters into consulting, research and other agreements with commercial entities, researchers, universities and others for the provision of goods and services. Such arrangements are generally cancellable upon reasonable notice and payment of costs incurred. Costs are considered incurred based on an evaluation of the progress to completion of specific tasks under each contract using information and data provided by the Company’s clinical sites and vendors. These costs consist of direct and indirect costs associated with specific projects, as well as fees paid to various entities that perform certain research on behalf of the Company. Depending upon the timing of payments to the service providers, the Company recognizes prepaid expenses or accrued expenses related to these costs. These accrued or prepaid expenses are based on management’s estimates of the work performed under service agreements, milestones achieved and experience with similar contracts. The Company monitors each of these factors and adjusts estimates accordingly. Income Taxes The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Valuation allowances are provided if, based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position, as well as consideration of the available facts and circumstanc es. As of December 31, 2023 and 2022, the Company did not have any significant uncertain tax positions. Share-Based Compensation - Options The Company measures stock-based compensation cost at the accounting measurement date based on the fair value of the option and recognizes the expense related to awards to employees on a straight-line basis over the requisite service period of the option, which is typically the vesting period. Forfeitures are recognized as they occur. The Company estimates the fair value of each option using the Black-Scholes option pricing model that considers the fair value of its common stock, the exercise price, the expected life of the option, the expected volatility of its common stock, expected dividends on its common stock, and the risk-free interest rate over the expected life of the option. The Company uses the simplified method described in the SEC's Staff Accounting Bulletin No. 107, Share-Based Payment, to determine the expected life of the option grants. The estimate of expected volatility is based on a review of the historical volatility of similar publicly held companies in the biotechnology field over a period commensurate with the option's expected term. The Company has never declared or paid any cash dividends on its common stock and does not expect to do so in the foreseeable future. Accordingly, it uses an expected dividend yield of zero. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant valuation for a period commensurate with the option's expected term. These assumptions are subjective and changes in them could significantly impact the value of the option and hence the related compensation expense. Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk include cash, cash equivalents, and marketable securities. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and the issuers of its cash equivalents and marketable securities. The Company maintains its cash, cash equivalents and marketable securities with highly-rated, federally-insured financial institutions. At times, such amounts may exceed federally-insured limits. The Company has not experienced any losses on its deposits since inception, and management believes that minimal credit risk exists with respect to these financial institutions. Net Loss per Share Basic net loss per common share is calculated by dividing net loss by the weighted-average shares outstanding during the period. For purposes of the diluted net loss per share calculation, convertible notes and common stock options are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, basic and diluted net loss per share were the same for all periods presented. Comprehensive Loss The Company’s components of comprehensive loss other than its net loss, are foreign currency gains/losses recorded from the remeasurement of the long-term intra-entity loan transaction to the Company’s wholly owned subsidiaries, foreign currency gain/loss from the translation of the Company’s wholly owned subsidiaries into U.S. dollars, unrealized gains and losses on marketable securities, and actuarial gains (losses) and prior service costs in connection with the Company's defined benefit plan. Recent Accounting Pronouncements In December 2023, the Financial Accounting Standards Board ("FASB") issued an amendment to the accounting guidance on income taxes which requires entities to provide additional information in the rate reconciliation and additional disaggregated disclosures about income taxes paid. This guidance requires public entities to disclose in their rate reconciliation table additional categories of information about federal, state, and foreign income taxes and to provide more details about the reconciling items in some categories if the items meet a quantitative threshold. The guidance is effective for annual periods beginning after December 15, 2024. The Company is currently evaluating this guidance to determine the impact it may have on its consolidated financial statements disclosures. In November 2023, the FASB issued an amendment to the accounting guidance on segment reporting. The amendments require disclosure of significa nt segment expenses and other segment items and requires entities to provide in interim periods all disclosures about a reportable segment's profit or loss and assets that are currently required annually. The amendment also requires disclosure of the title and position of the chief operating decision maker ("CODM") and an explanation of how the CODM uses the reported measure(s) of segment profit or loss in assessing segment performance and deciding how to allocate resources. The guidance is effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024. Retrospective application is required, and early adoption is permitted. The Company is currently evaluating the impact the guidance will have on its consolidated financial statements. |
Product Revenues, Accounts Rece
Product Revenues, Accounts Receivable, and Reserves for Product Sales | 12 Months Ended |
Dec. 31, 2023 | |
Product Revenues Accounts Receivable And Reserves For Product Sales [Abstract] | |
Product Revenues, Accounts Receivable, and Reserves for Product Sales | 3. Product Revenues, Accounts Receivable, and Reserves for Product Sales The Company received FDA approval for the sale of EMPAVELI in the United States in May 2021 and approval for the sale of SYFOVRE in the United States in February 2023. The Company’s product revenues, net of sales discounts and allowances and reserves as of December 31, 2023, 2022 and 2021 totaled $ 366.3 million , $ 65.1 million and $ 15.1 million. The Company’s product revenues consist of sales of EMPAVELI and SYFOVRE to specialty pharmacies and specialty distributors. The table reflects product revenue by major source for the following periods (in thousands): Year Ended December 31, 2023 2022 2021 Products: EMPAVELI $ 91,033 $ 65,092 $ 15,147 SYFOVRE 275,248 — — Total product revenue, net $ 366,281 $ 65,092 $ 15,147 The Company’s accounts receivable balance of $ 206.4 million as of December 31, 2023 and $ 7.7 million as of December 31, 2022 primarily consisted of EMPAVELI and SYFOVRE product sales receivable and licensing and other revenue receivables from its collaboration with Sobi. The Company does not have a reserve related to expected credit losses against its accounts receivable balance and expects to collect its accounts receivable in the ordinary course of business. The Company’s product sales reserves totaled $ 16.6 million and $ 2.4 million as of December 31, 2023 and 2022. respectively. These amounts are included in accrued expenses on the Company’s consolidated balance sheet as of December 31, 2023. The following table summarizes activity in each of the product revenue allowance and reserve categories as of December 31, 2023 (in thousands): Chargebacks, Discounts, and Fees Government and other rebates Returns Total Ending balance at December 31, 2021 $ 67 $ 716 $ 167 $ 951 Provision related to sales in the current year 614 4,569 1,185 6,368 Adjustments related to prior period sales ( 156 ) ( 246 ) ( 385 ) ( 787 ) Credits and payments made ( 362 ) ( 3,103 ) ( 716 ) ( 4,181 ) Ending balance at December 31, 2022 $ 164 $ 1,936 $ 251 $ 2,351 Provision related to sales in the current year 17,690 26,661 4,698 49,050 Adjustments related to prior period sales ( 112 ) ( 1,223 ) ( 2,481 ) ( 3,817 ) Credits and payments made ( 12,068 ) ( 18,476 ) ( 415 ) ( 30,960 ) Ending balance at December 31, 2023 $ 5,674 $ 8,898 $ 2,053 $ 16,625 Significant customers - Gross product revenues and product sales receivable from the Company's customers who individually accounted for 10% of more of total gross product revenues and/or 10% or more of total product sales receivable consisted of the following: Percent of Total Gross Product Revenues Year Ended December 31, 2023 2022 2021 Customer A 24 % 99 % 99 % Customer C 16 % — — Customer D 54 % 1 % — Percent of Product Sales Receivable As of December 31, 2023 2022 Customer A 4 % 96 % Customer C 22 % 3 % Customer D 66 % — |
Inventory
Inventory | 12 Months Ended |
Dec. 31, 2023 | |
Inventory Disclosure [Abstract] | |
Inventory | 4. Inventory The Comp any’s inventory of EMPAVELI and SYFOVRE consisted of the following as of December 31, 2023, and December 31, 2022 (in thousands): December 31, 2023 2022 Raw materials $ 32,724 $ 29,847 Semi-finished goods 82,924 54,101 Finished goods 30,714 1,766 Total inventories $ 146,362 $ 85,714 Inventory amounts written down as a result of excess, obsolete, unmarketability or other reasons are charged to cost of sales. For the years ended December 31, 2023 and 2022, the Company recognized write-downs of $ 6.4 million and $ 1.5 million, respectively. |
Prepaid and Other Current Asset
Prepaid and Other Current Assets | 12 Months Ended |
Dec. 31, 2023 | |
Prepaid Expense and Other Assets, Current [Abstract] | |
Prepaid and Other Current Assets | 5. Prepaid and Other Current Assets Prepaid as sets and other current assets consisted of the following as of December 31, 2023, and December 31, 2022 (in thousands): December 31, 2023 2022 Down payments for inventory $ 16,296 $ 13,987 Prepaid research and development 13,931 15,181 Other prepaid expenses 8,593 7,182 Total prepaid expenses $ 38,820 $ 36,350 December 31, 2023 2022 Royalties receivable 3,054 $ 1,442 ERC credit — 8,711 Receivable from collaboration agreement (1) 15,000 20,000 Deposits and other current assets 4,354 6,505 Total other current assets $ 22,408 $ 36,658 (1) In January 2024 the Company waived the remaining reimbursement payment of $ 15.0 million in connection with the decision to discontinue the CAD program. Under the provisions of the Coronavirus Aid, Relief, and Economic Security Act (the “CARES Act”) signed into law on March 27, 2020 and the subsequent extension of the CARES Act, the Company was eligible for a refundable employee retention credit (“ERC”) subject to certain criteria. The ERC provides eligible employers with less than 500 employees a refundable tax credit against the employer’s share of social security taxes. The ERC is equal to 70 % of qualified wages paid to employees during 2021 calendar year for a maximum credit of $ 7,000 per employee for each calendar quarter through September 30, 2021. In November 2022, the Company filed for an $ 8.7 million refund under the CARES Act relating to the ERC. The full refund amount was received in 2023. |
Development Liability
Development Liability | 12 Months Ended |
Dec. 31, 2023 | |
Research and Development [Abstract] | |
Development Liability | 6. Development Liability On February 28, 2019, the Company entered into a development funding agreement with SFJ (the “SFJ agreement”), under which SFJ agreed to provide funding to the Company to support the development of pegcetacoplan for the treatment of patients with PNH. Pursuant to the agreement, SFJ paid the Company $ 60.0 million following the signing of the agreement and agreed to pay the Company up to an additional $ 60.0 million in the aggregate in three equal installments upon the achievement of specified development milestones with respect to the Company’s Phase 3 program for pegcetacoplan in PNH and subject to the Company having cash resources at the time sufficient to fund at least 10 months of the Company’s operations. In June 2019 we amended the SFJ agreement to include an additional $ 20 million funding payment. SFJ paid us $ 80.0 million under the amended SFJ agreement between June 2019 and January 2020. Under the SFJ agreement, following regulatory approval by the FDA in May 2021 for the use of systemic pegcetacoplan as a treatment for PNH, the Company became obligated to pay SFJ an initial payment of $ 4.0 million and then an additional $ 226.0 million in the aggregate in six additional annual payments with the majority of the payments being made from the third anniversary to the sixth anniversary of regulatory approval. The Company paid SFJ the initial payment of $ 4.0 million in June 2021, its first annual payment of $ 11.5 million in May 2022 and its second annual payment of $ 24.5 million in May 2023. The subsequent annual payments remaining are due and payable in May of each year from 2024 through 2027. Following regulatory approval of systemic pegcetacoplan by the European Medicines Agency (“EMA”) in December 2021, the Company became obligated to pay SFJ an initial payment of $ 5.0 million and then an additional $ 225.0 million in the aggregate in six additional annual payments with the majority of the payments being made from the third anniversary to the sixth anniversary of regulatory approval. The Company paid SFJ the initial payment of $ 5.0 million in January 2022 its first annual payment of $ 18.0 million in December 2022 and its second annual payment of $ 31.0 million in December 2023. The subsequent annual payments are due and payable in December of each year from 2024 through 2027. The Company has paid SFJ a total of $ 94.0 million as of December 31, 2023. Additionally, the Company granted a security interest to SFJ in all of its assets, excluding intellectual property and license agreements to which it is a party. In connection with the grant of the security interest, the Company agreed to certain affirmative and negative covenants, including restrictions on its ability to pay dividends, incur additional debt or enter into licensing transactions with respect to its intellectual property, other than specified types of licenses. From December 15, 2021 to the final annual payment due in December 2027, the development liability will be accreted from its initial carrying amount to the total payment amount using the effective interest rate method under the ASC Topic 835, Interest, over the remaining life of the SFJ agreement. The difference between the carrying amount and the total payment amount is presented as a discount to the development liability. The accretion is recorded as interest expense in the unaudited condensed consolidated statement of operations. The following t able summarizes the development liability (in thousands): December 31, 2023 2022 Effective Development liability $ 366,000 $ 421,500 7.91 % Less: Unamortized discount to development liability ( 50,353 ) ( 76,349 ) Less: Current portion of development liability, net of discount ( 75,830 ) ( 29,504 ) Total long term development liability 239,817 315,647 As of December 31, 2023 and December 31, 2022 interest expense of $ 26.0 million and $ 26.9 million was recorded for the accretion of the development liability. For the period from December 15, 2021 through December 31, 2021, $ 1.2 million of interest expense was recorded for the accretion of the development liability. Future minimum SFJ payments as of December 31, 2023 is as follows (in thousands): 2024 $ 98,750 2025 103,000 2026 109,000 2027 55,250 Total future minimum payments 366,000 |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2023 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses consisted of the following as of December 31, 2023, and December 31, 2022 (in thousands): December 31, 2023 2022 Accrued research and development $ 28,318 $ 34,849 Accrued royalties 10,197 907 Accrued payroll liabilities 51,781 43,212 Product revenue reserves 16,625 2,351 Other 20,885 13,820 Total accrued expenses $ 127,806 $ 95,139 |
Long-term Debt
Long-term Debt | 12 Months Ended |
Dec. 31, 2023 | |
Debt Disclosure [Abstract] | |
Long-term Debt | 8. Long-term Debt Convertible Senior Notes On September 16, 2019, the Company completed a private offering of the 2019 Convertible Notes with an aggregate principal amount of $ 220.0 million issued pursuant to an indenture (the “Indenture”) with U.S. Bank National Association, as trustee (the “Trustee”). The net proceeds from the sale of the 2019 Convertible Notes were approximately $ 212.9 million after deducting the initial purchasers’ discounts and commissions of $ 6.6 million and offering expenses of $ 0.5 million paid by the Company. The Company used $ 28.4 million of the net proceeds from the sale of the Convertible Notes to pay the cost of the capped call transactions described below. On May 12, 2020, the Company issued the 2020 Convertible Notes with an aggregate principal amount of $ 300.0 million. The net proceeds from the sale of the 2020 Convertible Notes were approximately $ 322.9 million after deducting the purchasers’ discounts and commission of $ 5.7 million and offering expenses of $ 0.3 million. The Company used $ 43.1 million of the net proceeds from the sale to pay the cost of the additional capped call transactions in May 2020 described below. The 2019 Convertible Notes and the 2020 Convertible Notes are referred to together as the Convertible Notes. The Convertible Notes are senior unsecured obligations of the Company and bear interest at a rate of 3.5 % per year payable semiannually in arrears on March 15 and September 15 of each year, beginning on March 15, 2020. The Convertible Notes will mature on September 15, 2026 , unless converted earlier, redeemed or repurchased in accordance with their terms. The Convertible Notes are convertible into shares of the Company’s common stock at an initial conversion rate of 25.3405 shares per $1,000 principal amount of Convertible Notes (equivalent to an initial conversion price of approximately $ 39.4625 per share of common stock). The conversion rate is subject to customary anti-dilution adjustments. In addition, following certain events that occur prior to the maturity date or if the Company deliver a notice of redemption, the Company will increase the conversion rate for a holder who elects to convert its Convertible Notes in connection with such corporate event or a notice of redemption, as the case may be, in certain circumstances as provided in the indenture. Prior to March 15, 2026, the Convertible Notes are convertible only upon the occurrence of certain events: • during any calendar quarter, if the last reported sale price of the Company’s common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter is greater than or equal to 130 % of the conversion price on each applicable trading day; • during the five-business day period after any five consecutive trading day period in which the trading price per $ 1,000 principal amount of the Convertible Notes for each such trading day was less than 98 % of the product of the last reported sale price of the Company’s common stock and the conversion rate on each such trading day; • if the Company calls any or all of the Convertible Notes for redemption, at any time prior to the close of business on the second scheduled trading day immediately preceding the redemption date; or • upon the occurrence of corporate events specified in the Indenture. On or after March 15, 2026 until the close of business on the second scheduled trading day immediately preceding the maturity date of the Convertible Notes, holders may convert the Convertible Notes at any time. Upon conversion of the Convertible Notes, the Company will pay or deliver, as the case may be, cash, shares of the Company’s common stock or a combination of cash and shares of common stock, at the Company’s election. Prior to September 20, 2023, the Company could not redeem the Convertible Notes. From and after September 20, 2023 , the Company may redeem for cash all or a portion of the Convertible Notes, at its option, if the last reported sale price of the Company’s common stock has been at least 130 % of the conversion price then in effect for at least 20 trading days (whether or not consecutive), including the trading day immediately preceding the date on which the Company provides a notice of redemption, during any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which the Company provides notice of redemption. The redemption price will be equal to 100 % of the principal amount of the Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. If the Company calls any Convertible Notes for redemption, it will constitute a “make-whole fundamental change” with respect to such Convertible Notes, in which case the conversion r ate applicable to the conversion of such Notes, if converted in connection with the redemption, will be increased in certain circumstances. The Company has not called for redemption or redeemed any of the Convertible Notes as of December 31, 2023. If the Company undergoes a “fundamental change,” as defined in the Indenture, prior to maturity, subject to certain conditions, holders may require the Company to repurchase for cash all or any portion of their Convertible Notes at a fundamental change repurchase price equal to 100 % of the principal amount of the notes to be repurchased, plus any accrued and unpaid interest to, but excluding, the fundamental change repurchase date. The Company used an effective interest rate of 10.5 % to determine the liability component of the 2019 and 2020 Convertible Notes. This resulted in the recognition of $ 145.1 million and $ 204.5 million as the liability component of the 2019 and 2020 Convertible Notes, respectively, and the recognition of the residual amount of $ 74.9 million and $ 95.5 million as the debt discount with a corresponding increase to additional paid in capital for the equity component of the 2019 and 2020 Convertible Notes, respectively. The 2020 aggregate debt issuance costs of $ 6.0 million were allocated to the liability and equity components in the amounts of $ 3.7 million and $ 2.3 million, respectively. The 2019 Convertible Notes aggregate debt issuance costs of $ 7.1 million were allocated to the liability and equity components in the amounts of $ 4.7 million and $ 2.4 million, respectively. In January 2021, July 2021 and July 2022, the Company entered into separate, privately negotiated exchange agreements to modify the conversion terms with certain holders of its 2019 Convertible Notes and 2020 Convertible Notes. Under the terms of these exchange agreements, in January 2021, July 2021 and July 2022, the holders exchanged approximately $ 126.1 million of 2019 Convertible Notes, $ 201.1 million of 2019 Convertible Notes and 2020 Convertible Notes, and $ 98.1 million of 2020 Convertible Notes, respectively, in aggregate principal amount held by them for an aggregate of 3,906,869 shares, 5,992,217 shares and 3,027,018 shares, respectively, of common stock issued by the Company. In accordance with ASC Topic 470-20, “Debt – Debt with Conversion and Other Options,” (“ASC 470-20”) the Company accounted for the exchange as an induced conversion based on the short period of time the conversion offer was open and the substantive conversion feature offer. The Company accounted for the conversion of the debt as an inducement by expensing the fair value of the shares that were issued in excess of the original terms of the Convertible Notes. As a result of the January 2021 exchange transactions, the Company reduced net debt outstanding and increased net equity on the consolidated balance sheet by $ 122.8 million, consisting of the par value of the 2019 Convertible Notes exchanged of $ 126.1 million less the $ 3.3 million of remaining debt issuance costs associated with the exchanged notes. The Company also increased shares outstanding by 3,906,869 shares consisting of 3,196,172 shares issued at the initial conversion rate in the Indenture of 25.3405 plus an additional 710,697 shares. Additionally, the Company issued 69,491 shares as settlement of debt issuance costs paid to the Company’s financial advisor in connection with the exchange transaction. For the three months ended March 31, 2021, the Company recorded a loss on conversion of debt of $ 39.5 million comprised of $ 36.4 million related to the value of the shares issued in excess of the original conversion terms at the fair market value and $ 3.1 million for the value of the 69,491 shares issued in payment of issuance costs at their then fair value. Upon exchange of the 2019 Convertible Notes, the holders forfeited accrued interest through the date of the exchange of $ 1.7 million, which the Company charged to interest expense and to equity. As a result of the July 2021 exchange transactions, the Company reduced net debt outstanding and increased net equity on the consolidated balance sheet by $ 197.0 million, consisting of the par value of the Convertible Notes exchanged of $ 201.1 million less the $ 4.1 million of remaining debt issuance costs associated with the exchanged notes. The Company also increased shares outstanding by 5,992,217 shares consisting of 5,097,166 shares issued at the initial conversion rate in the Indenture of 25.3405 plus an additional 895,051 shares. Additionally, the Company issued 78,419 shares as settlement of issuance costs paid to the Company’s financial advisor in connection with the exchange transaction. For the three months ended September 30, 2021, the Company recorded a loss on conversion of debt of $ 61.1 million comprised of $ 55.9 million related to the value of the shares issued in excess of the original conversion terms at the fair market value and $ 5.2 million for the value of the 78,419 shares issued in payment of issuance costs at their then fair value. Upon exchange of the Convertible Notes, the holders forfeited accrued interest through the date of the exchange of $ 2.5 million, which the Company charged to interest expense and to equity. As a result of the July 2022 exchange transactions, the Company reduced net debt outstanding and increased net equity on the consolidated balance sheet by $ 96.8 million, consisting of the par value of the Convertible Notes exchanged of $ 98.1 million less the $ 1.3 million of remaining debt issuance costs associated with the exchanged notes. The Company also increased shares outstanding by 3,027,018 shares consisting of 2,485,548 shares issued at the initial conversion rate in the Indenture of 25.3405 plus an additional 541,470 shares. Additionally, the Company issued 46,132 shares as settlement of issuance costs paid to the Company’s financial advisor in connection with the exchange transaction. The Company recorded a loss on conversion of debt of $ 32.9 million comprised of $ 30.4 million related to the value of the shares issued in excess of the original conversion terms at the fair market value and $ 2.5 million for the value of the 46,132 shares. The conditional conversion feature of the Convertible Notes was triggered as of June 30, 2021, and as a result the Convertible Notes were convertible at the option of the holders until September 30, 2021. During this period, certain holders of the Convertible Notes converted approximately $ 0.7 million of aggregate principal amount of Convertible Notes into an aggregate of 18,775 shares, which were issued in October 2021. The conditional conversion feature of the Convertible Notes was again triggered as of September 30, 2022, and as a result the Convertible Notes became convertible at the option of the holders until December 31, 2022. No Convertible Notes were converted during this period. The conditional conversion feature of the Convertible Notes was triggered as of December 31, 2023, and as a result the Convertible Notes are convertible at the option of the holders until March 31, 2024. As of December 31, 2023 and 2022, the Company held in treasury Convertible Notes in principal amo unt of $ 425.4 million w hich have not been cancelled. The outstanding balance of the Convertible Notes as of December 31, 2023 and 2022 consisted of the following (in thousands): December 31, 2023 2022 Liability Principal 93,897 93,897 Less: debt discount and issuance costs, net ( 864 ) ( 1,161 ) Net carrying amount 93,033 92,736 The following table sets forth total interest expense recognized related to the Convertible Notes during the twelve months ended December 31, 2023, 2022 and 2021 (in thousands): Year Ended December 31, 2023 2022 2021 Amortization of debt issuance costs 297 459 964 Contractual interest expense 3,286 5,248 11,086 Total interest expense $ 3,583 $ 5,707 $ 12,050 Future minimum payments on Convertible Notes payable as of December 31, 2023 is as follows (in thousands): 2024 $ 3,286 2025 3,286 2026 96,225 Total future minimum payments 102,797 Less: interest ( 8,900 ) Less: debt discount and issuance costs, net ( 864 ) Less: current portion — Convertible senior notes 93,033 Capped Call Transactions On September 11, 2019, and May 6, 2020 concurrently with the pricing of the 2019 Convertible Notes and the 2020 Convertible Notes, the Company entered into capped call transactions with two counterparties. The capped call transactions are expected generally to reduce the potential dilution to the Company’s common stock upon any conversion of Convertible Notes and/or offset any cash payments the Company is required to make in excess of the principal amount of converted Convertible Notes, as the case may be, in the event that the market price per share of the Company’s common stock, as measured under the terms of the capped call transactions, is greater than the strike price of the capped call transactions, which is initially $ 39.4625 (the conversion price of the Convertible Notes) and is subject to anti-dilution adjustments substantially similar to those applicable to the conversion rate of such Convertible Notes. If, however, the market price per share of the Company’s common stock, as measured under the terms of the capped call transactions, exceeds the cap price of the capped call transactions, which is initially $ 63.14 per share, there would nevertheless be dilution and/or there would not be an offset of such potential cash payments, in each case, to the extent that such market price exceeds the cap price of the capped call transactions. Pursuant to ASC 815-40 Derivatives and Hedging , the Company determined that the capped call transactions should be classified as equity instruments and the capped call premium paid in the amount of $ 28.4 million and $ 43.1 million were recorded as reductions to additional paid-in capital at December 31, 2021 for the 2019 and 2020 Convertible Notes, respectively |
Leases
Leases | 12 Months Ended |
Dec. 31, 2023 | |
Leases [Abstract] | |
Leases | 9. Leases The underlying assets of the Company’s leases primarily relate to office space leases, but also include some equipment leases. The Company determines if an arrangement qualifies as a lease at its inception. As a practical expedient permitted under Topic 842, the Company has elected to account for the lease and non-lease components as a single lease component for all leases of which it is the lessee. Lease payments, which may include lease and non-lease components, are included in the measurement of the Company’s lease liabilities to the extent that such payments are either fixed amounts or variable amounts that depend on a rate or index as stipulated in the lease contract. When the Company cannot readily determine the rate implicit in the lease, the Company determines its incremental borrowing rate by using the rate of interest that it would have to pay to borrow on a collateralized basis over a similar term, an amount equal to the lease payments in a similar economic environment. The Company enters into lease agreements with terms generally ranging from 2 - 7 years . Some of the Company’s lease agreements include Company options to extend the lease on a month-to-month basis or for set periods for up to five years . Many leases also include options to terminate the leases within one year or per other contractual terms. Renewal and termination options were generally not included in the lease term for the Company’s existing operating leases. As of December 31, 2023 and 2022 , all leases were classified as operating lease assets and liabilities. Additional information related to the operating lease assets and liabilities is as follows (in thousands): December 31, December 31, 2023 2022 Operating lease assets $ 16,745 $ 18,747 Operating lease liabilities $ 17,895 $ 19,977 Weighted average remaining term in years 2.83 3.57 Weighted average discount rate used to measure 7.20 % 7.26 % For the years ended December 31, 2023, 2022, and 2021 , the total lease cost for operating lease expense was approximately $ 7.0 million, $ 6.2 million, and $ 5.6 million, respectively. Supplemental cash flow information related to operating leases for the years ended December 31, 2023, 2022 and 2021 is as follows (in thousands): 2023 2022 2021 Operating cash flows from operating leases $ 7,939 $ 7,375 $ 5,989 Operating lease assets obtained in exchange for lease obligations 2,700 — $ 5,675 The maturity of the Company’s operating lease liabilities as of December 31, 2023 are as follows (in thousands): 2024 $ 7,393 2025 6,238 2026 5,299 2027 859 Total future minimum lease payments 19,789 Less imputed interest ( 1,894 ) Total operating lease liabilities $ 17,895 |
Other Comprehensive Income and
Other Comprehensive Income and Accumulated Other Comprehensive Income | 12 Months Ended |
Dec. 31, 2023 | |
Equity [Abstract] | |
Other Comprehensive Income and Accumulated Other Comprehensive Income | 10. Other Comprehensive Income and Accumulated Other Comprehensive Income The following tables summarize the changes in accumulated other comprehensive income/(loss), by component for the years ended December 31, 2023 and 2022 (in thousands): Unrealized Gains (Losses) from Marketable Securities Foreign Currency Translation Adjustment Unrealized Gains (Losses) from Pension Plan Total Accumulated Other Comprehensive Income (Loss) Balances, December 31, 2022 $ — $ ( 2,521 ) $ 1,646 $ ( 875 ) Net other comprehensive income (loss) — ( 49 ) ( 2,618 ) $ ( 2,667 ) Balances, December 31, 2023 $ — $ ( 2,570 ) $ ( 972 ) $ ( 3,542 ) Unrealized Gains (Losses) from Marketable Securities Foreign Currency Translation Adjustment Unrealized Gains (Losses) from Pension Plan Total Accumulated Other Comprehensive Income (Loss) Balances, December 31, 2021 $ 1 $ ( 2,091 ) $ — $ ( 2,090 ) Net other comprehensive income (loss) ( 1 ) ( 430 ) 1,646 $ 1,215 Balances, December 31, 2022 $ — $ ( 2,521 ) $ 1,646 $ ( 875 ) |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 11. Fair Value Measurements The Company is required to disclose information on the fair value of financial instruments and inputs that enable an assessment of the fair value. The three levels of the fair value hierarchy prioritize valuation inputs based upon the observable nature of those inputs as follows: Level 1 – Quoted prices in active markets for identical assets or liabilities; Level 2 – Inputs other than quoted prices included within level 1 that are observable for the asset or liability, either directly or indirectly; Level 3 – Unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability. The following table presents the fair value of financial instruments recorded originally at amortized cost or fair value and not re-measured on a recurring basis (in thousands): December 31, 2023 Balance Sheet Classification: Type of Instrument Level 1 Level 2 Level 3 Total Financial Assets: Cash and cash equivalents: Money market funds $ 276,391 $ — $ — $ 276,391 Total Financial Assets $ 276,391 $ — $ — $ 276,391 December 31, 2022 Balance Sheet Classification: Type of Instrument Level 1 Level 2 Level 3 Total Financial Assets: Cash and cash equivalents: Money market funds $ 527,728 $ — $ — $ 527,728 Total Financial Assets $ 527,728 $ — $ — $ 527,728 The Company's Convertible Notes and development liability are financial instruments that are reported in the consolidated financial statements at historical cost. The Convertible Notes are Level 1 within the fair value level hierarchy as of December 31, 2023 and December 31, 2022 . The fair value of the Convertible Notes was $ 140.8 million as of December 31, 2023 and $ 143.9 million as of December 31, 2022 . The Convertible Notes accrue a semi-annual coupon at an annual rate of 3.5 %, which was included in accrued expenses in the consolidated balance sheets as of December 31, 2023 and December 31, 2022. The fair value of the development liability was $ 306.9 million and $ 315.8 million as of December 31, 2023 and December 31, 2022 , respectively. The development liability is Level 2 within the fair value hierarchy based on the discounting of fixed cash flows using an observed bond yield for borrowers with similar credit rating. |
License and Collaboration Agree
License and Collaboration Agreements | 12 Months Ended |
Dec. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
License and Collaboration Agreements | 12. License and Collaboration Agreements Sobi License and Collaboration Agreement In October 2020, the Company and its subsidiaries Apellis International GmbH (f/k/a Apellis Switzerland GmbH) and APL DEL Holdings, LLC entered into a Collaboration and License Agreement with Sobi, concerning the development and commercialization of pegcetacoplan and specified other structurally and functionally similar compstatin analogues or derivatives for use systemically or for local non-ophthalmological administration (collectively referred to as the “Licensed Products”). Under the Sobi collaboration agreement, the Company granted Sobi an exclusive (subject to certain retained rights of the Company), sublicensable license of certain patent rights and know-how to develop and commercialize Licensed Products in all countries outside of the United States. The Company retains the right to commercialize Licensed Products in the United States, and, subject to specified limitations, to develop Licensed Products worldwide for commercialization in the United States. Under the Sobi collaboration agreement, the Company and Sobi have agreed to collaborate to develop Licensed Products for the treatment of PNH, CAD, HSCT-TMA, C3G, IC-MPGN and ALS, or collectively the “Initial Indications”, and any other indications subsequently agreed upon by the parties, for commercialization by or on behalf of the Company in the United States and by or on behalf of Sobi outside of the United States. If the parties do not agree to jointly pursue any development activities for the Licensed Products (whether for an Initial Indication or otherwise), the party proposing to pursue such activities may conduct such activities at its sole expense (with the non-proposing party having the right to obtain rights to the data generated by such development activities by paying a specified percentage of that expense), subject to agreed-upon exceptions that limit each party’s unilateral development rights. The initial development plan sets forth the initial development activities to be conducted by each of the Company and Sobi, with the Company bearing all costs incurred in conducting the activities set forth in such initial development plan, as well as certain specified additional costs that are not included in the initial development plan that may be incurred by the parties in developing Licensed Products for PNH in the European Union and the United Kingdom. The Company and Sobi have formed several governance committees to oversee the development and manufacture, and to review and discuss the commercialization, of Licensed Products. The Company and SOBI have discontinued to development programs in ALS and CAD. The Company shall supply Licensed Products to Sobi for development and for commercialization outside of the United States in accordance with a supply agreement to be negotiated by the parties. The collaboration agreement grants Sobi the right to perform or have performed drug product manufacturing of Licensed Products for development and for commercialization outside the United States and to manufacture or have manufactured drug substance under certain circumstances. Sobi paid the Company an upfront payment of $ 250.0 million in November 2020 and has agreed to pay up to an aggregate of $ 915.0 million upon the achievem ent of specified one-time regulatory and commercial milestone events, of which the Company received $ 50.0 million in April 2022 for the achievement of a regulatory development milestone in Europe and $ 5.0 million in October 2023 for the achievement of a regulatory development milestone in Japan. Sobi also agreed to reimburse the Company for up to $ 80.0 million in development costs, of which the Company received $ 25.0 million in January 2021, $ 20.0 million in January 2022 and $ 20.0 million in January 2023. In January 2024, the Company waived the remaining reimbursement payment of $ 15.0 million in connection with the to discontinue the CAD program. The Company is also entitled to receive tiered, double-digit royalties (ranging from high teens to high twenties) on sales of Licensed Products outside of the United States, subject to customary deductions and third-party payment obligations, until the latest to occur of: (i) expiration of the last-to-expire of specified licensed patent rights; (ii) expiration of regulatory exclusivity; and (iii) ten ( 10 ) years after the first commercial sale of the applicable Licensed Product, in each case on a Licensed Product-by-Licensed Product and country-by-country basis. The Company received $ 10.0 million in royalties from Sobi during 2023. Under the Sobi collaboration agreement, the Company remains responsible for its license fee obligations (including royalty obligations) to the Trustees of the University of Pennsylvania (“Penn”), as a licensor of the Company and for its payment obligations to SFJ. Sobi Accounting Analysis The Company has determined that the agreement is within the scope of ASC 808 as a contractual arrangement that involves a joint operating activity whereby both parties are (i) active participants in the activity and (ii) exposed to certain significant risks and rewards dependent on the commercial success of the activity. ASC Topic 808 does not address measurement or recognition matters but allows for analogizing to ASC 606. Pursuant to ASC 606, the Company performed the following five steps: (i) identified the contract(s) with a customer; (ii) identified the performance obligations in the contract; (iii) determined the transaction price; (iv) allocated the transaction price to the performance obligations in the contract; and (v) recognized revenue when (or as) the entity satisfies a performance obligation. The Company identified the following material distinct promises under the Sobi Agreement: (1) licenses to develop and commercialize pegcetacoplan or, Licenses to IP, and (2) performance of research and development services. The Company determined the promises to be distinct because Sobi can benefit from each of the license and the development services on their own or with readily available services. The Company could have provided the license without any development services and Sobi would have been able to benefit from it by obtaining development services from another provider as the Licensed Products are at a more mature stage in their life cycle. Under the agreement, Sobi agreed to pay the Company i) a fixed amount of $ 250.0 million in an upfront payment in November 2020; ii) a fixed amount of an additional $ 80.0 million in development reimbursements, payable yearly in four tranches in amounts determined based upon actual expenses incurred by the Company; iii) up to an aggregate of $ 915.0 million upon the achievement of specified one-time regulatory and commercial milestone events; and iv) tiered, double-digit royalties, ranging from high teens to high twenties, on sales of Licensed Products outside of the United States, subject to customary deductions and third-party payment obligations. At contract inception, the $ 250.0 million non-refundable payment and the $ 80.0 million reimbursements were fixed proceeds. The Company evaluated whether Sobi is a customer for either of the distinct promises in the agreement. Under the Licenses to IP, the Company determined that Sobi is a customer as the know-how provided and the right granted by the Company to Sobi are outputs of the Company’s business activities for which the Company will receive consideration. With respect to research and development activity, management determined that there is no vendor relationship as performing research and development activities for others is not a part of the Company’s ongoing central operations. Based upon the evaluation of the relative fair values, the Company allocated the purchase price of $ 250.0 million and the related milestones and royalties to the license of IP and $ 80.0 million to performance of research and development activities. The milestone and royalty payments are subject to activities outside the control of the Company. Per ASC 606, the Company considers this to be a customer/ vendor relationship, therefore, the Company will include the regulatory milestone payments in the total transaction price when it is probable that a significant reversal of revenue would not occur in a future period. The Company will recognize commercial milestone and royalty revenue at the later of (i) when the related sales occur or (ii) when the performance obligation to which the commercial milestone or royalty has been allocated has been satisfied. In case of commercial milestone or royalty payments, the Company will recognize revenue in the same period that the sales are completed for which the Company is contractually entitled to the milestone or percentage-based royalty payment. Management will periodically assess the elements of the contract and re-evaluate revenue recognition as necessary. Pursuant to ASC 606, during the year ended December 31, 2020, the Company has recognized the $ 250.0 million in revenue as this is the amount allocated to the license. The $ 80.0 million reimbursement for research and development activities does not constitute a customer/vendor relationship and thus is not in the scope of ASC 606. As ASC 808 does not include recognition guidance, the Company has established an accounting policy to recognize the payments under the reimbursement as a receivable on the balance sheet in an amount that is to be reimbursed based upon expense incurred by the Company, with a contra- research and development expense recognized in the statement of operations, over time as the expenses are incurred. Under the Sobi collaboration agreement, for the year ended December 31, 2023 , the Company recognized $ 10.0 million of royalty revenue. For the year ended December 31, 2022 , the Company recognized $ 3.0 million of royalty revenue. For the year ended December 31, 2023 , the Company did no t recognize any contra-research and development expense in the consolidated statement of operations related to the $ 80.0 million reimbursement commitment from Sobi. For the year ended December 31, 2022 , the Company recognized $ 5.0 million for contra-research and development expense in the consolidated statement of operations related to the $ 80.0 million reimbursement commitment from Sobi. As of December 31, 2023, the Company recorded $ 15.0 million in current assets, which represents the receivable for contra-research and development expenses incurred but not yet reimbursed from Sobi. The total receivable balance as of December 31, 2023 was for contra-research and development expenses incurred but not yet reimbursed from Sobi. In January 2024, the Company waived the remaining reimbursement payment of $ 15.0 million in connection with the decision to discontinue the CAD program. As of December 31, 2022 , the Company recorded a receivable of $ 35.0 million, with $ 20.0 million in other current assets and $ 15.0 million in other assets on the consolidated balance sheet. The total receivable balance as of December 31, 2022 is for contra-research and development expenses incurred but not yet reimbursed from Sobi. The Company received the $ 20.0 million recorded in other current assets in January 2023. University of Pennsylvania License Agreement The Company is a party to a license agreement with the Trustees of the University of Pennsylvania (“Penn”) for an exclusive, worldwide license to specified patent rights. The Company is required to pay annual maintenance fees of $ 0.1 million until the first sale of a licensed product. The Company is also required to make milestone payments aggregating up to $ 3.2 million based upon the achievement of specified development and regulatory milestones and up to $ 5.0 million based upon the achievement of specified annual sales milestones with respect to each licensed product, and to pay low single-digit royalties based on net sales of each licensed product and with minimum quarterly royalty thresholds. In addition, the Company is obligated to pay a specified portion of income it receives from sublicensees. In April 2023, the Company paid $ 2.3 million for the achievement of a regulatory milestone as a result of the FDA approval of SYFOVRE in February 2023. In 2023 the Company incurred $ 5.0 million as a result of the achievement of sales milestones for SYFOVRE of which $ 2.0 million the Company paid in October 2023 and remaining of $ 3.0 million is accrued for as of December 31, 2023. As of December 31, 2023 , the Company has incurred an aggregate royalty expense of $ 8.9 million on sales of SYFOVRE. In addition, the Company is also party to a license agreement with Penn for an exclusive, worldwide license to specified patent rights for the development and commercialization of products in fields of use, as defined therein. The Company is required to pay annual maintenance fees of $ 0.1 million until the first sale of a licensed product. The Company is required to make milestone payments aggregating up to $ 1.7 million, based upon the achievement of development and regulatory approval milestones, and up to $ 2.5 million, based upon the achievement of annual sales milestones with respect to each of the first two licensed products. The license agreement also requires the Company to pay low single digit royalties based on net sales of each licensed product, subject to minimum quarterly royalty thresholds. In addition, the Company is obligated to pay a specified portion of income it receives from sublicensees. In January 2021, the Company paid $ 25.0 million for sublicense fee owed to Penn related to the Sobi Agreement and another licensing transaction. In August 2021, the Company paid $ 1.0 million to Penn upon the achievement of a development milestone, net of a credit for the annual license maintenance payment. In June 2022, the Company paid an additional $ 5.0 million to Penn upon the achievement of a development milestone. In January 2023, the Company paid $ 1.0 million to Penn upon the achievement of a sales milestone for EMPAVELI in 2022. As December 31, 2023, the Company recorded in accrued expenses $ 0.5 million for a sublicense fee owed to Penn related to Sobi obtaining regulatory approval in Japan. Additionally, as of December 31, 2023, the Company recorded in accrued expenses $ 1.5 million as a result of the achievement of a sales milestone for EMPAVELI and Aspaveli. As of December 31, 2023 , the Company has incurred an aggregate royalty expense of $ 7.9 million on sales of EMPAVELI and Aspaveli. Beam Research Collaboration In June 2021, the Company entered into an exclusive five-year research collaboration with Beam Therapeutics, Inc. (“Beam”) focused on the use of Beam’s proprietary base editing technology to discover new treatments for complement-driven diseases. The Company and Beam agreed to collaborate on up to six research programs focused on C3 and other complement targets in the eye, liver and brain. Under the terms of the collaboration agreement, Apellis is responsible for selecting specific genes within the complement system in various organs including the eye, liver and brain (the “Target List”) and providing analytical support while Beam will apply its base editing technology and conduct preclinical research on up to six base editing programs for the Target List. During the first five years of the collaboration agreement, Beam is prohibited from developing on its own or with a third party any base editing therapies associated with the items on the Target List but does not prevent Beam from licensing its intellectual property to a third-party for another purpose outside of the Target List. The Company will have exclusive rights to license each of the six programs and will assume responsibility for subsequent development and commercialization. Beam may elect to enter a 50-50 co-development and U.S. co-commercialization agreement with the Company with respect to any one program licensed under the collaboration and upon such election any license agreement in place at that time, would be terminated. As part of the Beam collaboration agreement, the Company agreed to pay a $ 50.0 million up-front, non-refundable payment to Beam, which the Company paid in July 2021. In June 2022, the Company paid $ 25.0 million, which was recorded as a cost of research collaboration expense for the year ended December 31, 2021, as it was considered probable of achievement. The Company and Beam are each responsible for their own costs during the research collaboration. If and after the opt-in license rights are exercised for each of the up to six programs, Beam will be eligible to receive development, regulatory and sales milestones from the Company, as well as royalty payments on sales. The Beam collaboration agreement has an initial term of five years and may be extended up to two years on a per year program-by-program basis. The Company analyzed the research collaboration agreement pursuant to ASC 808 to assess whether the agreement involved joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. Since each party is actively participating in this activity and exposed to significant risks and rewards related to the activity through each party’s costs will be accounted for under ASC 808. Since ASC 808 does not provide recognition guidance, the Company referred to the guidance under ASC 730 to arrangements involving payments by the Company. ASC 730 requires the Company to recognize research and developments costs as expense as incurred since the payment was made for the use of Beam’s intellectual property and research and development services and there is no alternative use. |
Employee Retirement Plans
Employee Retirement Plans | 12 Months Ended |
Dec. 31, 2023 | |
Retirement Benefits [Abstract] | |
Employee Retirement Plans | 13. Employee Retirement Plans In July 2010, the Company adopted an employee profit-sharing plan (the “401(k) Plan”), qualified under Section 401(k) of the Internal Revenue Code (the “IRC”). All of the Company’s full-time employees who have attained the age of 21 are eligible to participate in the 401(k) Plan immediately upon employment. Pursuant to the 401(k) Plan, employees may elect to reduce their current compensation by up to the statutorily prescribed annual limit and have the amount of the reduction contributed to the 401(k) Plan. In 2023, 2022 and 2021 , the Company recorded $ 5.7 million, $ 4.3 million, and $ 3.1 million respectively, for employer contributions made to the 401(k) Plan. The Company maintains a pension plan covering employees of its Swiss subsidiary, Apellis International GmbH (f/k/a Apellis Switzerland GmbH) (the “Swiss Plan”). The Swiss Plan is a government-mandated retirement fund that provides employees with a minimum benefit. Employer and employee contributions are made to the Swiss Plan based on various percentages of salary and wages that vary according to employee age and other factors. As is customary with Swiss pension plans, the assets of the Swiss Plan are invested in a collective fund, which are held and invested by a Swiss insurance company. The investment strategy of the Swiss Plan is managed by an independent asset manager with the objective of achieving a consistent long-term return which will provide sufficient funding for future pension obligations while limiting risk. As of a December 31, 2023 , the Swiss Plan had a funded status of $ 2.2 million, which resulted from fair value of plan assets of $ 16.2 million and projected benefit obligation of $ 18.4 million. The accumulated benefit obligation at December 31, 2023 was $ 2.6 million. The Company’s net periodic benefit cost for the year ended December 31, 2023 was $ 0.9 million. The contributions to the Swiss Plan for the year ended December 31, 2023 were not material. As of a December 31, 2022, the Swiss Plan had a funded status of $ 18.0 thousand, which resulted from fair value of plan assets of $ 12.9 million and projected benefit obligation of $ 12.9 million. The accumulated benefit obligation at December 31, 2022 was $ 1.6 million. The Company’s net periodic benefit cost for the year ended December 31, 2022 was $ 0.6 million. The contributions to the Swiss Plan for the year ended December 31, 2022 were not material. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 14. Income Taxes The components of loss from continuing operations before provision for income taxes are as follows (in thousands): Year Ended December 31, 2023 2022 2021 United States $ ( 48,495 ) $ ( 82,815 ) $ ( 314,673 ) Foreign ( 478,001 ) ( 568,688 ) ( 431,329 ) Total $ ( 526,496 ) $ ( 651,503 ) $ ( 746,002 ) Provision for income taxes for the years ended December 31, 2023, 2022, and 2021 are as follows (in thousands): Year Ended December 31, 2023 Year Ended December 31, 2022 Year Ended December 31, 2021 Current income tax expense: U.S. Federal $ — $ — $ — U.S. State and Local 1,869 520 205 Foreign 263 149 147 Total current income tax expense 2,132 669 352 Deferred income tax expense: U.S. Federal — — — U.S. State and Local — — — Foreign — — — Total deferred income tax expense — — — Total tax expense $ 2,132 $ 669 $ 352 A reconciliation between the U.S. federal statutory tax rate and the Company's effective tax rate is summarized as follows (in thousands): Year Ended December 2023 2022 2021 Amount Percentage of income before income taxes Amount Percentage of income before income taxes Amount Percentage of income before income taxes Statutory U.S. federal income tax $ ( 110,564 ) 21.0 % $ ( 136,816 ) 21.0 % $ ( 156,660 ) 21.0 % Foreign tax rate differential 42,100 ( 8.0 ) 50,219 ( 7.7 ) 38,677 ( 5.2 ) State income taxes, net of federal benefit ( 13,438 ) 2.6 9,051 ( 1.4 ) ( 14,145 ) 1.9 Change in valuation allowances 119,592 ( 23.2 ) 94,668 ( 14.5 ) 133,668 ( 17.9 ) Tax credits ( 11,566 ) 2.2 ( 19,966 ) 3.0 ( 20,005 ) 2.6 Share Based Compensation ( 26,881 ) 5.1 — — — — Change in state apportionment — — ( 35 ) — — — Loss on debt conversion — — 6,626 ( 1.0 ) 19,548 ( 2.6 ) Permanent and other 2,889 ( 0.1 ) ( 3,078 ) 0.5 ( 731 ) 0.1 Effective income tax provision $ 2,132 ( 0.4 ) $ 669 ( 0.1 ) $ 352 ( 0.1 ) The Company’s effective income tax rate for the year ended December 31, 2023 compared to the year ended December 31, 2022 increased primarily as a result of operations in state jurisdictions. The Company accounts for income taxes in accordance with ASC Topic 740. Deferred income tax assets and liabilities are determined based upon temporary differences between the financial reporting and tax basis of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The following table presents the principal components of the Company’s deferred tax assets and liabilities (in thousands): December 31, 2023 2022 Deferred tax assets: Intangible assets $ 192,444 $ 187,839 Share-based compensation 31,712 29,673 Net operating loss carryforwards 329,135 228,985 Research and development credits 67,667 57,419 Orphan drug credits 34,023 30,160 Development derivative liability 75,190 79,374 Convertible debt 5,582 6,850 Fixed Assets 18 — Lease liability 2,888 4,304 Accruals 17,401 16,272 Total deferred tax assets 756,060 640,876 Deferred tax liabilities: Fixed assets — ( 91 ) Right-of-use asset ( 2,672 ) ( 4,032 ) 481(a) adjustment — ( 233 ) Total deferred tax liabilities ( 2,672 ) ( 4,356 ) Net deferred tax assets before allowance: 753,388 636,520 Less valuation allowance ( 753,388 ) ( 636,520 ) Net deferred tax assets $ — $ — The Tax Cuts and Jobs Act (TCJA) requires taxpayers to capitalize and amortize research and development (R&D) expenditures under section 174 for tax years beginning after December 31, 2021. This rule became effective for the Company on January 1, 2022 and resulted in the capitalization of R&D costs of approximately $ 42.3 million and $ 47.6 million for tax year ending December 31, 2023 and 2022, respectively. The Company will amortize these costs for tax purposes over 5 years if the R&D was performed in the U.S. and over 15 years if the R&D was performed outside the U.S. ASC Topic 740 requires a valuation allowance to reduce the deferred tax assets reported if, based on the weight of available evidence, it is more likely than not that some portion or all of the deferred tax assets will not be realized. After consideration of all the evidence, both positive and negative, the Company has recorded full valuation allowances against its domestic and foreign deferred tax assets on December 31, 2023, because management has determined that is it more likely than not that these assets will not be realized. The valuation allowance increased by $ 116.9 million from December 31, 2022 to December 31, 2023, primarily due to increases in operating losses and research and development tax credits. On December 31, 2023, the Company had approximately $ 494.1 million, $ 524.0 million and $ 1,663.3 million of federal, state and foreign net operating loss carryforward, respectively. On December 31, 2022, the Company had approximately $ 312.8 million, $ 395.4 million and $ 1,204.7 million of federal, state and foreign net operating loss carryforward, respectively. The Company also had federal and state research and development tax credit carryforwards $ 85.7 million and $ 20.2 million, respectively of as of December 31, 2023. Federal net operating loss carryforward in the amount of $ 449.8 million may be carried forward indefinitely. The remaining federal and state net operating loss, research and development tax credit carryforwards begin to expire in 2025 . The Company’s foreign net operating loss carryforwards will begin to expire in 2027 . Under the provisions of the Internal Revenue Code (“IRC”), the net operating loss (“NOL”), and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. NOL and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the IRC, respectively, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of the Company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. The Company has completed several financings since its inception that it believes may have resulted in a change in control as defined by Sections 382 and 383 of the IRC. The Company does not have any unrecognized tax benefits during any periods presented and does not expect this to significantly change in the next twelve months. There were no interest and penalties recorded in the statement of operations during any period and no amounts accrued for interest and penalties on December 31, 2023 or 2022. The Company and its subsidiaries file income tax returns in the United States, as well as various state and foreign jurisdictions. Generally, the tax years 2020 through 2022 remain open and subject to examination by the major taxing jurisdictions to which the Company is subject. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, or state or foreign tax authorities, to the extent utilized in a future period. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 15. Commitments and Contingencies The Company has certain non-cancelable purchase obligations related to the manufacturing of drug substance and drug product, with Bachem Americas, Inc., under which the Company has agreed to purchase a significant portion of its requirements for the pegcetacoplan drug substance over the next five years and a commercial supply agreement with NOF Corporation, under which the Company has agreed to purchase activated polyethylene glycol derivative, or PEG, which is a component of pegcetacoplan. Under these agreements, as of December 31, 2023 , the Company is obligated to pay up to an aggregate of $ 94.6 million to these vendors. In addition, the Company has other non-cancelable purchase agreements as of December 31, 2023 , under which it is obligated to pay up to $ 16.1 million to vendors. The Company is a party to a master lease agreement under which the Company leases vehicles with initial terms of 36 months from the date of delivery. If the Company were unable to take delivery of a previously ordered vehicle, the Company may incur nominal fees . Indemnifications —In the ordinary course of business, the Company enters into agreements that may include indemnification provisions. Pursuant to such agreements, the Company may indemnify, hold harmless and defend indemnified parties for losses suffered or incurred by the indemnified party. Some of the provisions will limit losses to those arising from third-party actions. In some cases, the indemnification will continue after the termination of the agreement. The maximum potential amount of future payments the Company could be required to make under these provisions is not determinable. The Company has no t incurred any cost to defend lawsuits or settle claims related to these indemnification provisions. Legal —During the normal course of business, the Company may be a party to legal claims that may not be covered by insurance. The Company cannot predict the outcome of the litigation described in Item 3 with certainty. The Company intends to defend vigorously against this litigation. |
Equity Incentive Plans
Equity Incentive Plans | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Equity Incentive Plans | 16. Equity Incentive Plans Share-based Compensation The Company’s Board of Directors adopted, and its stockholders approved, an equity incentive plan in 2010 (as amended, the “2010 Plan”). The Board of Directors and stockholders amended the 2010 Plan in August 2017 to increase the number of shares of common stock reserved for issuance thereunder to 6,188,466 . The 2010 Plan allowed for the grant of incentive stock options and non-qualified stock options to purchase common stock for employees, directors and consultants under terms and conditions established by the Board of Directors. Incentive stock options and nonqualified stock options were granted at exercise prices that were no less than 100 % of the estimated fair value per share of the common stock on the date of grant. If an individual owns capital stock representing more than 10 % of the voting shares, the price of each share was at least 110 % of the fair value on the date of grant. The Board of Directors determined the fair value of common stock with the assistance of a third-party specialist. Options expire 10 years from the issuance date. Following the adoption of the 2017 Stock Incentive Plan, the Company no longer grants stock options or other awards under the 2010 Plan. In October 2017, the Company’s Board of Directors adopted, and its stockholders approved, the 2017 Stock Incentive Plan (the “2017 Plan”), which became effective on November 8, 2017. The 2017 Plan provides for the grant of incentive stock options, non-statutory stock options, stock appreciation rights, awards of restricted stock, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the 2017 plan is the sum of (i) 1,359,587 shares of common stock, plus (ii) an additional number of shares of common stock equal to the sum of (a) the number of shares of common stock reserved for issuance under the 2010 equity incentive plan that remained available for future issuance immediately prior to the effectiveness of the 2017 Plan, which was 299,568 shares, and (b) the number of shares of common stock subject to outstanding awards under the 2010 equity incentive plan upon effectiveness of the 2017 plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by us at their original issuance price pursuant to a contractual repurchase right plus (iii) an annual increase, to be added the first day of each fiscal year, beginning with the fiscal year ending December 31, 2018 and continuing until, and including, the fiscal year ending December 31, 2027, equal to the lowest of 4,219,409 shares of common stock, 4.0 % of the number of shares of common stock outstanding on the first day of the fiscal year and an amount determined by the board of directors. In January 2024, the shares available for future issuance under the 2017 plan were increased by 4,219,409 shares pursuant to the annual increase described above. As of December 31, 2023, there were 6,497,120 shares available for future grants under the 2017 Plan. Additionally, since 2019, the Company has granted equity awards as equity inducement awards material to entry into employment with the Company to certain newly hired employees outside of the Company’s existing plans in accordance with Nasdaq listing rule 5635(c)(4). In February 2020, the Board of Directors adopted the 2020 Inducement Stock Incentive Plan (the “2020 Plan”), which permitted the Company to grant equity awards to newly hired employees in accordance with Nasdaq listing rule 5635(c)(4). The aggregate number of shares reserved for issuance under the 2020 Plan was initially 750,000 shares. The Board of Directors amended the 2020 Plan to add 200,000 shares on January 1, 2024, which increased the total number of shares reserved for issuance to 1,950,000 shares as of January 1, 2024. As of December 31, 2023, there were 354,466 shares available for future grants under the 2020 Plan. In October 2017, the Company’s board of directors adopted, and the Company’s stockholders approved the 2017 Employee Stock Purchase Plan (“ESPP”), which became effective upon the IPO and provides participating employees with the opportunity to purchase up to an aggregate of 468,823 shares of common stock. The number of shares of common stock reserved for issuance under the 2017 ESPP will automatically increase on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2018 and continuing until, and including, the fiscal year ending December 31, 2027, equal to the lowest of (i) 937,646 shares of common stock, (ii) 1.0 % of the number of shares of common stock outstanding on the first day of the fiscal year and (iii) an amount determined by the board of directors. The board of directors initiated the first offering under ESPP in October 2019. The Company has reserved the following shares of common stock for future issuance (in thousands): December 31, 2023 2022 2021 Shares reserved under 2017 Equity Incentive Plan 16,989 14,271 11,014 Shares reserved under 2017 Employee Stock Purchase Plan 553 665 801 Shares reserved under 2020 Inducement Stock Incentive Plan 1,638 1,857 1,761 Total 19,180 16,793 13,576 Total share-based compensation expense related to the various plans during the years ended was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Research and development $ 45,644 $ 42,052 $ 30,586 General and administrative 60,301 49,033 40,081 Total share-based compensation expense $ 105,945 $ 91,085 $ 70,667 Stock Options— Options granted generally vest over 48 months. Options granted to employees on or after December 5, 2013 generally vest in installments of (i) 25% at the one-year anniversary and (ii) in either 36 equal monthly or 12 equal quarterly installments beginning in the thirteenth month after the initial vesting commencement date (as defined) subject to the employee’s continuous service with the Company. Options granted before December 5, 2013 vest over four years in equal annual installments of 25 % at each anniversary of the grant date. Under the Executive Separation Benefits and Retention Plan and by resolutions adopted by the Compensation Committee in October 2019, the stock options granted to the Company’s executives and employees will become fully vested upon the occurrence of a change in control, as defined in the Executive Separation Benefits and Retention Plan, if such executive or employee is terminated without cause or resigns for good reason within 12 months after such change in control. The following table summarizes the Company’s stock option activity: Weighted - Weighted - Average Average Aggregate Exercise Contractual Intrinsic Shares Price Life Value (in thousands) Per Share (in years) (in thousands) Outstanding, December 31, 2022 12,295 $ 26.31 6.24 317,031 Granted 838 52.48 Exercised ( 3,950 ) 20.11 Forfeited ( 519 ) 43.65 Outstanding, December 31, 2023 8,664 $ 30.65 Options exercisable, December 31, 2023 6,712 $ 26.20 5.39 $ 226,419 Expected to vest, December 31, 2023 1,952 $ 45.97 8.13 $ 27,515 The aggregate intrinsic values of options outstanding, exercisable, vested and expected to vest were calculated as the difference between the exercise price of the options and the fair value of the common stock as of December 31, 2023. During the years ended December 31, 2023, 2022 and 2021 , the Company granted stock options to purchase an aggregate of 0.8 million, 1.3 million and 2.7 million shares of its common stock, respectively with weighted average grant date fair values of $ 34.26 , $ 23.62 and $ 30.72 , respectively. The aggregate intrinsic value of options exercised during the years ended December 31, 2023, 2022 and 2021 were $ 181.0 million, $ 44.8 million, and $ 39.9 million respectively calculated as the difference between the exercise price of the underlying options and the estimated fair value of the common stock for the options on the respective date of exercise. The fair market value of options vested during the years ended December 31, 2023, 2022 and 2021 were $ 46.8 million, $ 59.0 million and $ 53.2 million, respectively. On December 31, 2023 , unrecognized compensation expense related to unvested options, was $ 46.1 million, which the Company expects to recognize over an estimated weighted-average period of 2.18 years. The assumptions used in the Black-Scholes model to estimate the grant date fair value are as follows: Year Ended December 31, 2023 2022 2021 Risk-free interest rate 3.50 - 4.01 % 1.15 - 3.37 % 0.41 - 1.34 % Dividend yield 0 % 0 % 0 % Volatility 68.4 - 71.0 % 68.3 - 70.4 % 71.7 - 74.4 % Expected terms (years) 3.81 - 6.08 3.81 - 6.08 5.31 - 6.08 Restricted Stock Units— The fair value of RSU’s is estimated based upon the closing market price of the Company’s common stock on the date of grant. RSUs generally vest annually over a four-year period. The following table summarizes the Company’s RSU's activity: Number of Stock Units Weighted Average Grant Date Fair Value Per Share Unvested Balance at December 31, 2022 3,572 $ 42.23 Granted 2,916 52.19 Vested ( 1,015 ) 41.52 Forfeited ( 1,172 ) 48.43 Unvested Balance at December 31, 2023 4,301 47.48 The aggregate intrinsic value of restricted grants vested during the year ended December 31, 2023 , was $ 55.3 million. The fair market value of restricted stock units vested during the year ended December 31, 2023 was $ 42.2 million. On December 31, 2023 , there was approximately $ 155.5 million of related unrecognized compensation cost which the Company expects to recognize over a remaining weighted average period of 2.6 years. Employee Stock Purchase Plan— On December 31, 2023 , 552,881 shares of common stock remained available for issuance pursuant to the ESPP. Eligible employees who elect to participate in an offering under the ESPP may have up to 15 percent of their earnings withheld, subject to certain limitations, to purchase shares of common stock pursuant to the ESPP. The price of common stock purchased under the ESPP is equal to 85 percent of the lower of the fair market value of the common stock at the commencement date of each offering period or the relevant purchase date. During the year ended December 31, 2023 , a total of 112,064 shares of common stock were issued under the ESPP at average per share price of $ 48.00 . During the year ended December 31, 2023 , the Company recorded cash received from the issuance of stock to the ESPP of $ 5.4 million and recorded $ 2.1 million of stock-based compensation expense related to the ESPP. No additional shares were reserved to the ESPP in 2023. |
Net Loss per Common Share
Net Loss per Common Share | 12 Months Ended |
Dec. 31, 2023 | |
Earnings Per Share [Abstract] | |
Net Loss per Common Share | 17. Net Loss per Common Share The following table presents the calculation of basic and diluted net loss per common share (amounts in thousands except per share amounts): Year Ended December 31, 2023 2022 2021 Numerator: Net loss $ ( 528,628 ) $ ( 652,172 ) $ ( 746,354 ) Denominator: Weighted-average number of common shares used in net loss per common share - basic and diluted 118,678 106,114 84,421 Net loss per common share -- basic and diluted $ ( 4.45 ) $ ( 6.15 ) $ ( 8.84 ) Shares outstanding presented below were excluded from the calculation of diluted net loss per share, prior to the use of the if-converted-method and treasury stock method, as their effect is anti-dilutive (in thousands): Year Ended December 31, 2023 2022 2021 Convertible notes 2,379 2,379 4,865 Common stock under option 8,664 12,295 12,562 Restricted stock units 4,301 3,572 1,224 Total 15,344 18,246 18,651 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States (“U.S. GAAP”) and following the requirements of the Securities and Exchange Commission (the “SEC”). |
Revenue Recognition | Revenue Recognition The Company’s revenues consist of product sales of EMPAVELI and SYFOVRE and revenue derived from its collaboration arrangement with Sobi. See Note 12, License and Collaboration Agreements for further discussion related to the Sobi Collaboration and License Agreement. The Company accounts for contracts with its customers in accordance with ASC Topic 606, Revenue from Contracts with Customers, ( “ASC 606”). Pursuant to ASC 606, for arrangements or transactions between participants determined to be within the scope of the contracts with customers guidance, the Company performs the following five steps to determine the appropriate amount of revenue to be recognized as the Company fulfills its obligations: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. Revenue is recognized when, or as, the Company satisfies a performance obligation by transferring a promised good or service to a customer. An asset is transferred when, or as, the customer obtains control of that asset. For performance obligations that are satisfied over time, the Company recognizes revenue using an input or output measure of progress that best depicts the satisfaction of the relevant performance obligation. |
Product Revenue | Product Revenue The Company’s revenue from net product sales was generated in the United States following the FDA’s approval for marketing of EMPAVELI for the treatment of PNH in May 2021 and SYFOVRE in February 2023. The Company sells EMPAVELI and SYFOVRE principally through arrangements with specialty pharmacies (“SPs”) and specialty distributors (“SDs”), who are the Company’s customers. The customers subsequently resell the product to patients and health care providers. The Company applies the ASC 606 five step process discussed above to the contracts with SPs and SDs. The Company provides limited right of return to the customers in cases of shipment errors or expiring or defective products. Product revenues are recognized when the customers take control of the product, which typically occurs upon delivery to the customers. The Company recognizes revenue from product sales at the net sales price which includes estimates of variable consideration for which reserves are established and reflects each of these as a reduction to revenue. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which the Company is entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from estimates, the Company may need to adjust its estimates, which would affect net revenue in the period of adjustment. The following are the Company’s significant categories of variable consideration: Distribution Fees : Distribution fees include distribution service fees paid to SPs and SDs based on a contractually fixed percentage of the wholesale acquisition cost (WAC). The Company does not receive a distinct good or service in exchange for the payment. Distribution fees are recorded as an offset to revenue based on contractual terms at the time revenue from the sale is recognized. Chargebacks : Chargebacks are discounts and fees related to contracts with various third-party payers including pharmacy benefit managers, private healthcare insurers and government healthcare programs that purchase from SDs at a discounted price. SDs charge back to the Company the difference between the price initially paid by SDs and the discounted price paid to SDs by these entities. The Company issues credit notes for the chargeback which are applied to future sales. Product Returns : Consistent with industry practice, the Company offers SPs and SDs limited product return rights for shipment errors or expiring or defective products; provided that the return is within a specified period around the product expiration date as set forth in the applicable individual distribution agreement. The Company does not allow product returns for product that has been dispensed to a patient. As the Company receives inventory reports from SPs and SDs and has visibility into the inventory distribution channel, it is able to make a reasonable estimate of future potential product returns based on this on-hand channel inventory data and sell-through data obtained from SPs and SDs. In arriving at its estimate for product returns, the Company also considers historical product returns (to the extent available), the underlying product demand, and industry data specific to the specialty pharmaceutical distribution industry. |
Licensing and Collaboration Revenue | Licensing and Collaboration Revenue The Company analyzes license and collaboration arrangements pursuant to ASC Topic 808, Collaborative Arrangement Guidance and Considerations, (“ASC 808”) to assess whether such arrangements, or transactions between arrangement participants, involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities or are more akin to a vendor-customer relationship. In making this evaluation, the Company considers whether the activities of the collaboration are considered to be distinct and deemed to be within the scope of the collaborative arrangement guidance or if they are more reflective of a vendor-customer relationship and, therefore, within the scope of ASC 606. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For elements of collaboration arrangements that are not accounted for pursuant to guidance in ASC 606, an appropriate recognition method is determined and applied consistently, generally by analogy to the revenue from contracts with customers guidance. Amounts related to transactions with a counterparty in a collaborative arrangement that is not a customer are presented as collaboration revenue and in a separate line item from revenue recognized from contracts with customers, if any, in the consolidated statements of operations. Pursuant to ASC 606, for arrangements or transactions between arrangement participants determined to be within the scope of the contracts with customers guidance, the Company performs the five-step process discussed above to determine the appropriate amount of revenue to be recognized as the Company fulfills its obligations. We evaluate the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determine whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract. Goods or services that meet these criteria are considered distinct performance obligations. The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential transaction price and the likelihood that the transaction price will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and, if so, these options are considered performance obligations. The Company has not currently identified any such material rights. Revenue is recognized when, or as, the Company satisfies a performance obligation by transferring a promised good or service to a customer. An asset is transferred when, or as, the customer obtains control of that asset. For performance obligations that are satisfied over time, the Company recognizes revenue using an input or output measure of progress that best depicts the satisfaction of the relevant performance obligation. After contract inception, the transaction price is reassessed at every period end and updated for changes such as resolution of uncertain events. Any change in the overall transaction price is allocated to the performance obligations on the same methodology as at contract inception. See Note 12, License and Collaboration Agreements, for further discussion related to the Sobi collaboration agreement. |
Segment Information | Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results may differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes, and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: development liability, accrued expenses, prepaid expenses, convertible debt, reserves for variable consideration, reserves for excess or obsolete inventories, and income taxes. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments The Company is required to disclose information on the fair value of financial instruments and inputs that enable an assessment of the fair value. The three levels of the fair value hierarchy prioritize valuation inputs based upon the observable nature of those inputs as follows: Level 1 – Quoted prices in active markets for identical assets or liabilities; Level 2 – Inputs other than quoted prices included within level 1 that are observable for the asset or liability, either directly or indirectly; Level 3 – Unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability. The Company’s financial instruments, in addition to those presented in Note 8, Long-term Debt, and Note 11, Fair Value Measurements, include cash and cash equivalents, accounts payable and accrued liabilities. Management believes that the carrying amounts of cash and cash equivalents, accounts payable and accrued expenses approximate the fair value due to the short-term nature of those instruments. |
Cash and Cash Equivalents | Cash and Cash Equivalents Cash and cash equivalents are defined as cash in banks and investment instruments having maturities of three months or less from their acquisition date. The carrying amounts reported in the consolidated balance sheets for cash and cash equivalents are valued at cost, which approximates the fair value. See Note 11, Fair Value Measurements, for additional information. |
Accounts Receivable | Accounts Receivable The Company’s accounts receivable primarily arise from product sales. They are generally stated at the invoiced amount and do not bear interest. The accounts receivable from product sales represents receivables due from the Company’s SPs or SDs. The Company has had no historical write offs of its accounts receivable as of December 31, 2023, and its payment terms are generally 30-65 days for EMPAVELI and 60-150 days for SYFOVRE. The Company monitors the financial performance and creditworthiness of its customers and provides reserves against trade receivables for expected credit losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2023 and 2022, the credit pr ofiles for the Company’s customers were deemed to be in good standing and an allowance for credit losses was not considered necessary. |
Inventory | Inventory Inventory is recorded at the lower of cost or net realizable value, with cost determined on a first-in, first-out basis. Inventory costs include third-party contract manufacturing, third-party packaging services, and freight. The Company performs an assessment of the recoverability of capitalized inventory during each reporting period, and it writes down any excess and obsolete inventories to their estimated realizable value in the period in which the impairment is first identified. Such impairment charges, should they occur, are recorded within cost of sales. The determination of whether inventory costs will be realizable requires estimates by management. If actual market conditions are less favorable than projected by management, additional write-downs of inventory may be required which would be recorded as a cost of sales in the consolidated statements of operations and comprehensive loss. Prior to regulatory approval of its product candidates, the Company expensed costs associated with the manufacturing of its product candidates to research and development expense unless the Company was reasonably certain such costs have future commercial use and net realizable value. When the Company believes regulatory approval and subsequent commercialization of its product candidates is probable, and the Company also expects future economic benefit from the sales of the product candidates to be realized, the Company will then capitalize the costs of production as inventory. Inventory that can be used in either the production of clinical or commercial product is expensed as research and development expense when selected for use in a clinical manufacturing campaign. Prior to receiving FDA approval for EMPAVELI on May 14, 2021, the Company included in research and development expense the costs associated with the manufacture of EMPAVELI inventory to be sold upon commercialization. As a result, the manufacturing costs related to the EMPAVELI inventory build-up incurred before FDA approval were expensed in a prior period and are, therefore, excluded from the cost of goods sold and inventory. As of December 31, 2023, the remaining pre-FDA approved inventory was $ 19.4 million, which primarily consisted of raw materials. Shipping and handling costs for product shipments are recorded as incurred in cost of sales along with costs associated with manufacturing the product and any inventory write-downs. |
Development Liability | Development Liability From December 15, 2021 and thereafter until the final annual payment due December 2027, the development liability will be accreted from its initial carrying amount to the total payment amount using the effective interest rate method under ASC 835 over the remaining life of the SFJ agreement as defined in Note 6. The difference between the carrying amount and the total payment amount is presented as discount to the development liability. The accretion is recorded as interest expense in the consolidated income statement. |
Foreign Currency | Foreign Currency The functional currency of each of the Company’s subsidiaries is its local currency, except for the wholly owned subsidiaries in Switzerland and Netherlands where the functional currency is the U.S. dollar. Revenues and expenses of the subsidiaries have been translated into U.S. dollars at average exchange rates prevailing during the respective periods. Assets and liabilities have been translated at the rates of exchange on the balance sheet date. The resulting translation gain and loss adjustments are recorded directly as a separate component of stockholders’ equity. |
Research and Development | Research and Development Costs incurred in connection with research and development activities are expensed as incurred. Research and development expenses include (i) employee-related expenses, including salaries, benefits, travel and share-based compensation expense; (ii) external research and development expenses incurred under arrangements with third parties, such as contract research and contract manufacturing organizations, investigational sites and consultants, including share-based compensation expense for consultants; (iii) the cost of acquiring, developing and manufacturing clinical study materials; and (iv) costs associated with preclinical and clinical activities and regulatory operations. The Company enters into consulting, research and other agreements with commercial entities, researchers, universities and others for the provision of goods and services. Such arrangements are generally cancellable upon reasonable notice and payment of costs incurred. Costs are considered incurred based on an evaluation of the progress to completion of specific tasks under each contract using information and data provided by the Company’s clinical sites and vendors. These costs consist of direct and indirect costs associated with specific projects, as well as fees paid to various entities that perform certain research on behalf of the Company. Depending upon the timing of payments to the service providers, the Company recognizes prepaid expenses or accrued expenses related to these costs. These accrued or prepaid expenses are based on management’s estimates of the work performed under service agreements, milestones achieved and experience with similar contracts. The Company monitors each of these factors and adjusts estimates accordingly. |
Income Taxes | Income Taxes The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Valuation allowances are provided if, based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position, as well as consideration of the available facts and circumstanc es. As of December 31, 2023 and 2022, the Company did not have any significant uncertain tax positions. |
Share Based Compensation - Option | Share-Based Compensation - Options The Company measures stock-based compensation cost at the accounting measurement date based on the fair value of the option and recognizes the expense related to awards to employees on a straight-line basis over the requisite service period of the option, which is typically the vesting period. Forfeitures are recognized as they occur. The Company estimates the fair value of each option using the Black-Scholes option pricing model that considers the fair value of its common stock, the exercise price, the expected life of the option, the expected volatility of its common stock, expected dividends on its common stock, and the risk-free interest rate over the expected life of the option. The Company uses the simplified method described in the SEC's Staff Accounting Bulletin No. 107, Share-Based Payment, to determine the expected life of the option grants. The estimate of expected volatility is based on a review of the historical volatility of similar publicly held companies in the biotechnology field over a period commensurate with the option's expected term. The Company has never declared or paid any cash dividends on its common stock and does not expect to do so in the foreseeable future. Accordingly, it uses an expected dividend yield of zero. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant valuation for a period commensurate with the option's expected term. These assumptions are subjective and changes in them could significantly impact the value of the option and hence the related compensation expense. |
Concentrations of Credit Risk | Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk include cash, cash equivalents, and marketable securities. The Company is exposed to credit risk in the event of default by the financial institutions holding its cash and the issuers of its cash equivalents and marketable securities. The Company maintains its cash, cash equivalents and marketable securities with highly-rated, federally-insured financial institutions. At times, such amounts may exceed federally-insured limits. The Company has not experienced any losses on its deposits since inception, and management believes that minimal credit risk exists with respect to these financial institutions. |
Net Loss per Share | Net Loss per Share Basic net loss per common share is calculated by dividing net loss by the weighted-average shares outstanding during the period. For purposes of the diluted net loss per share calculation, convertible notes and common stock options are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, basic and diluted net loss per share were the same for all periods presented. |
Comprehensive Loss | Comprehensive Loss The Company’s components of comprehensive loss other than its net loss, are foreign currency gains/losses recorded from the remeasurement of the long-term intra-entity loan transaction to the Company’s wholly owned subsidiaries, foreign currency gain/loss from the translation of the Company’s wholly owned subsidiaries into U.S. dollars, unrealized gains and losses on marketable securities, and actuarial gains (losses) and prior service costs in connection with the Company's defined benefit plan. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In December 2023, the Financial Accounting Standards Board ("FASB") issued an amendment to the accounting guidance on income taxes which requires entities to provide additional information in the rate reconciliation and additional disaggregated disclosures about income taxes paid. This guidance requires public entities to disclose in their rate reconciliation table additional categories of information about federal, state, and foreign income taxes and to provide more details about the reconciling items in some categories if the items meet a quantitative threshold. The guidance is effective for annual periods beginning after December 15, 2024. The Company is currently evaluating this guidance to determine the impact it may have on its consolidated financial statements disclosures. In November 2023, the FASB issued an amendment to the accounting guidance on segment reporting. The amendments require disclosure of significa nt segment expenses and other segment items and requires entities to provide in interim periods all disclosures about a reportable segment's profit or loss and assets that are currently required annually. The amendment also requires disclosure of the title and position of the chief operating decision maker ("CODM") and an explanation of how the CODM uses the reported measure(s) of segment profit or loss in assessing segment performance and deciding how to allocate resources. The guidance is effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024. Retrospective application is required, and early adoption is permitted. The Company is currently evaluating the impact the guidance will have on its consolidated financial statements. |
Product Revenues, Accounts Re_2
Product Revenues, Accounts Receivable, and Reserves for Product Sales (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Product Revenues Accounts Receivable And Reserves For Product Sales [Abstract] | |
Schedule of Disaggregation of Product Revenue by Major Source | The table reflects product revenue by major source for the following periods (in thousands): Year Ended December 31, 2023 2022 2021 Products: EMPAVELI $ 91,033 $ 65,092 $ 15,147 SYFOVRE 275,248 — — Total product revenue, net $ 366,281 $ 65,092 $ 15,147 |
Summary of Product Revenue Allowance and Reserve Categories | The following table summarizes activity in each of the product revenue allowance and reserve categories as of December 31, 2023 (in thousands): Chargebacks, Discounts, and Fees Government and other rebates Returns Total Ending balance at December 31, 2021 $ 67 $ 716 $ 167 $ 951 Provision related to sales in the current year 614 4,569 1,185 6,368 Adjustments related to prior period sales ( 156 ) ( 246 ) ( 385 ) ( 787 ) Credits and payments made ( 362 ) ( 3,103 ) ( 716 ) ( 4,181 ) Ending balance at December 31, 2022 $ 164 $ 1,936 $ 251 $ 2,351 Provision related to sales in the current year 17,690 26,661 4,698 49,050 Adjustments related to prior period sales ( 112 ) ( 1,223 ) ( 2,481 ) ( 3,817 ) Credits and payments made ( 12,068 ) ( 18,476 ) ( 415 ) ( 30,960 ) Ending balance at December 31, 2023 $ 5,674 $ 8,898 $ 2,053 $ 16,625 |
Schedule of Customer Accounted for 10% of More of Gross Product Revenues and Accounts Receivable | Significant customers - Gross product revenues and product sales receivable from the Company's customers who individually accounted for 10% of more of total gross product revenues and/or 10% or more of total product sales receivable consisted of the following: Percent of Total Gross Product Revenues Year Ended December 31, 2023 2022 2021 Customer A 24 % 99 % 99 % Customer C 16 % — — Customer D 54 % 1 % — Percent of Product Sales Receivable As of December 31, 2023 2022 Customer A 4 % 96 % Customer C 22 % 3 % Customer D 66 % — |
Inventory (Tables)
Inventory (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Inventory Disclosure [Abstract] | |
Schedule Of Inventory Current | The Comp any’s inventory of EMPAVELI and SYFOVRE consisted of the following as of December 31, 2023, and December 31, 2022 (in thousands): December 31, 2023 2022 Raw materials $ 32,724 $ 29,847 Semi-finished goods 82,924 54,101 Finished goods 30,714 1,766 Total inventories $ 146,362 $ 85,714 Inventory amounts written down as a result of excess, obsolete, unmarketability or other reasons are charged to cost of sales. For the years ended December 31, 2023 and 2022, the Company recognized write-downs of $ 6.4 million and $ 1.5 million, respectively. |
Prepaid and Other Current Ass_2
Prepaid and Other Current Assets (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Prepaid Expense and Other Assets, Current [Abstract] | |
Summary of Prepaid Assets and Other Current Assets | Prepaid as sets and other current assets consisted of the following as of December 31, 2023, and December 31, 2022 (in thousands): December 31, 2023 2022 Down payments for inventory $ 16,296 $ 13,987 Prepaid research and development 13,931 15,181 Other prepaid expenses 8,593 7,182 Total prepaid expenses $ 38,820 $ 36,350 December 31, 2023 2022 Royalties receivable 3,054 $ 1,442 ERC credit — 8,711 Receivable from collaboration agreement (1) 15,000 20,000 Deposits and other current assets 4,354 6,505 Total other current assets $ 22,408 $ 36,658 (1) In January 2024 the Company waived the remaining reimbursement payment of $ 15.0 million in connection with the decision to discontinue the CAD program. |
Development Liability (Tables)
Development Liability (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Research and Development [Abstract] | |
Summary of Development Liability | The following t able summarizes the development liability (in thousands): December 31, 2023 2022 Effective Development liability $ 366,000 $ 421,500 7.91 % Less: Unamortized discount to development liability ( 50,353 ) ( 76,349 ) Less: Current portion of development liability, net of discount ( 75,830 ) ( 29,504 ) Total long term development liability 239,817 315,647 |
Schedule of Future Minimum SFJ Payments | Future minimum SFJ payments as of December 31, 2023 is as follows (in thousands): 2024 $ 98,750 2025 103,000 2026 109,000 2027 55,250 Total future minimum payments 366,000 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following as of December 31, 2023, and December 31, 2022 (in thousands): December 31, 2023 2022 Accrued research and development $ 28,318 $ 34,849 Accrued royalties 10,197 907 Accrued payroll liabilities 51,781 43,212 Product revenue reserves 16,625 2,351 Other 20,885 13,820 Total accrued expenses $ 127,806 $ 95,139 |
Long-term Debt (Tables)
Long-term Debt (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Debt Disclosure [Abstract] | |
Schedule of Outstanding Balance of Convertible Notes | The outstanding balance of the Convertible Notes as of December 31, 2023 and 2022 consisted of the following (in thousands): December 31, 2023 2022 Liability Principal 93,897 93,897 Less: debt discount and issuance costs, net ( 864 ) ( 1,161 ) Net carrying amount 93,033 92,736 |
Schedule of Interest Expense Recognized Related to Convertible Notes | The following table sets forth total interest expense recognized related to the Convertible Notes during the twelve months ended December 31, 2023, 2022 and 2021 (in thousands): Year Ended December 31, 2023 2022 2021 Amortization of debt issuance costs 297 459 964 Contractual interest expense 3,286 5,248 11,086 Total interest expense $ 3,583 $ 5,707 $ 12,050 |
Schedule of Future Minimum Payments on Convertible Notes Payable | Future minimum payments on Convertible Notes payable as of December 31, 2023 is as follows (in thousands): 2024 $ 3,286 2025 3,286 2026 96,225 Total future minimum payments 102,797 Less: interest ( 8,900 ) Less: debt discount and issuance costs, net ( 864 ) Less: current portion — Convertible senior notes 93,033 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Leases [Abstract] | |
Schedule of Additional Information Related to Operating Lease Assets and Liabilities | Additional information related to the operating lease assets and liabilities is as follows (in thousands): December 31, December 31, 2023 2022 Operating lease assets $ 16,745 $ 18,747 Operating lease liabilities $ 17,895 $ 19,977 Weighted average remaining term in years 2.83 3.57 Weighted average discount rate used to measure 7.20 % 7.26 % |
Supplemental Cash Flow Information Related to Operating Leases | Supplemental cash flow information related to operating leases for the years ended December 31, 2023, 2022 and 2021 is as follows (in thousands): 2023 2022 2021 Operating cash flows from operating leases $ 7,939 $ 7,375 $ 5,989 Operating lease assets obtained in exchange for lease obligations 2,700 — $ 5,675 |
Maturity of Operating Lease Liabilities | The maturity of the Company’s operating lease liabilities as of December 31, 2023 are as follows (in thousands): 2024 $ 7,393 2025 6,238 2026 5,299 2027 859 Total future minimum lease payments 19,789 Less imputed interest ( 1,894 ) Total operating lease liabilities $ 17,895 |
Other Comprehensive Income an_2
Other Comprehensive Income and Accumulated Other Comprehensive Income (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Equity [Abstract] | |
Summary of Changes in Accumulated Other Comprehensive Income/(Loss), by Component | The following tables summarize the changes in accumulated other comprehensive income/(loss), by component for the years ended December 31, 2023 and 2022 (in thousands): Unrealized Gains (Losses) from Marketable Securities Foreign Currency Translation Adjustment Unrealized Gains (Losses) from Pension Plan Total Accumulated Other Comprehensive Income (Loss) Balances, December 31, 2022 $ — $ ( 2,521 ) $ 1,646 $ ( 875 ) Net other comprehensive income (loss) — ( 49 ) ( 2,618 ) $ ( 2,667 ) Balances, December 31, 2023 $ — $ ( 2,570 ) $ ( 972 ) $ ( 3,542 ) Unrealized Gains (Losses) from Marketable Securities Foreign Currency Translation Adjustment Unrealized Gains (Losses) from Pension Plan Total Accumulated Other Comprehensive Income (Loss) Balances, December 31, 2021 $ 1 $ ( 2,091 ) $ — $ ( 2,090 ) Net other comprehensive income (loss) ( 1 ) ( 430 ) 1,646 $ 1,215 Balances, December 31, 2022 $ — $ ( 2,521 ) $ 1,646 $ ( 875 ) |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Fair Value, Nonrecurring [Member] | |
Schedule of Financial Instruments and the Related Fair Value Hierarchy of the Valuation Techniques Utilized | The following table presents the fair value of financial instruments recorded originally at amortized cost or fair value and not re-measured on a recurring basis (in thousands): December 31, 2023 Balance Sheet Classification: Type of Instrument Level 1 Level 2 Level 3 Total Financial Assets: Cash and cash equivalents: Money market funds $ 276,391 $ — $ — $ 276,391 Total Financial Assets $ 276,391 $ — $ — $ 276,391 December 31, 2022 Balance Sheet Classification: Type of Instrument Level 1 Level 2 Level 3 Total Financial Assets: Cash and cash equivalents: Money market funds $ 527,728 $ — $ — $ 527,728 Total Financial Assets $ 527,728 $ — $ — $ 527,728 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Components of Loss From Continuing Operation Before Provision for Income Taxes | The components of loss from continuing operations before provision for income taxes are as follows (in thousands): Year Ended December 31, 2023 2022 2021 United States $ ( 48,495 ) $ ( 82,815 ) $ ( 314,673 ) Foreign ( 478,001 ) ( 568,688 ) ( 431,329 ) Total $ ( 526,496 ) $ ( 651,503 ) $ ( 746,002 ) |
Schedule of Provision for Income Tax Expense | Provision for income taxes for the years ended December 31, 2023, 2022, and 2021 are as follows (in thousands): Year Ended December 31, 2023 Year Ended December 31, 2022 Year Ended December 31, 2021 Current income tax expense: U.S. Federal $ — $ — $ — U.S. State and Local 1,869 520 205 Foreign 263 149 147 Total current income tax expense 2,132 669 352 Deferred income tax expense: U.S. Federal — — — U.S. State and Local — — — Foreign — — — Total deferred income tax expense — — — Total tax expense $ 2,132 $ 669 $ 352 |
Schedule of Reconciliation Between U.S. Federal Statutory Rate and Effective Tax rate | A reconciliation between the U.S. federal statutory tax rate and the Company's effective tax rate is summarized as follows (in thousands): Year Ended December 2023 2022 2021 Amount Percentage of income before income taxes Amount Percentage of income before income taxes Amount Percentage of income before income taxes Statutory U.S. federal income tax $ ( 110,564 ) 21.0 % $ ( 136,816 ) 21.0 % $ ( 156,660 ) 21.0 % Foreign tax rate differential 42,100 ( 8.0 ) 50,219 ( 7.7 ) 38,677 ( 5.2 ) State income taxes, net of federal benefit ( 13,438 ) 2.6 9,051 ( 1.4 ) ( 14,145 ) 1.9 Change in valuation allowances 119,592 ( 23.2 ) 94,668 ( 14.5 ) 133,668 ( 17.9 ) Tax credits ( 11,566 ) 2.2 ( 19,966 ) 3.0 ( 20,005 ) 2.6 Share Based Compensation ( 26,881 ) 5.1 — — — — Change in state apportionment — — ( 35 ) — — — Loss on debt conversion — — 6,626 ( 1.0 ) 19,548 ( 2.6 ) Permanent and other 2,889 ( 0.1 ) ( 3,078 ) 0.5 ( 731 ) 0.1 Effective income tax provision $ 2,132 ( 0.4 ) $ 669 ( 0.1 ) $ 352 ( 0.1 ) |
Schedule of Deferred Tax Assets and Liabilities | The following table presents the principal components of the Company’s deferred tax assets and liabilities (in thousands): December 31, 2023 2022 Deferred tax assets: Intangible assets $ 192,444 $ 187,839 Share-based compensation 31,712 29,673 Net operating loss carryforwards 329,135 228,985 Research and development credits 67,667 57,419 Orphan drug credits 34,023 30,160 Development derivative liability 75,190 79,374 Convertible debt 5,582 6,850 Fixed Assets 18 — Lease liability 2,888 4,304 Accruals 17,401 16,272 Total deferred tax assets 756,060 640,876 Deferred tax liabilities: Fixed assets — ( 91 ) Right-of-use asset ( 2,672 ) ( 4,032 ) 481(a) adjustment — ( 233 ) Total deferred tax liabilities ( 2,672 ) ( 4,356 ) Net deferred tax assets before allowance: 753,388 636,520 Less valuation allowance ( 753,388 ) ( 636,520 ) Net deferred tax assets $ — $ — |
Equity Incentive Plans (Tables)
Equity Incentive Plans (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Shares of Common Stock Reserved for Future Issuance | The Company has reserved the following shares of common stock for future issuance (in thousands): December 31, 2023 2022 2021 Shares reserved under 2017 Equity Incentive Plan 16,989 14,271 11,014 Shares reserved under 2017 Employee Stock Purchase Plan 553 665 801 Shares reserved under 2020 Inducement Stock Incentive Plan 1,638 1,857 1,761 Total 19,180 16,793 13,576 |
Summary of Share-based Compensation Expense | Total share-based compensation expense related to the various plans during the years ended was as follows (in thousands): Year Ended December 31, 2023 2022 2021 Research and development $ 45,644 $ 42,052 $ 30,586 General and administrative 60,301 49,033 40,081 Total share-based compensation expense $ 105,945 $ 91,085 $ 70,667 |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity: Weighted - Weighted - Average Average Aggregate Exercise Contractual Intrinsic Shares Price Life Value (in thousands) Per Share (in years) (in thousands) Outstanding, December 31, 2022 12,295 $ 26.31 6.24 317,031 Granted 838 52.48 Exercised ( 3,950 ) 20.11 Forfeited ( 519 ) 43.65 Outstanding, December 31, 2023 8,664 $ 30.65 Options exercisable, December 31, 2023 6,712 $ 26.20 5.39 $ 226,419 Expected to vest, December 31, 2023 1,952 $ 45.97 8.13 $ 27,515 |
Summary of Assumption Used to Estimate the Grant Date Fair Value | The assumptions used in the Black-Scholes model to estimate the grant date fair value are as follows: Year Ended December 31, 2023 2022 2021 Risk-free interest rate 3.50 - 4.01 % 1.15 - 3.37 % 0.41 - 1.34 % Dividend yield 0 % 0 % 0 % Volatility 68.4 - 71.0 % 68.3 - 70.4 % 71.7 - 74.4 % Expected terms (years) 3.81 - 6.08 3.81 - 6.08 5.31 - 6.08 |
Schedule of Unvested Restricted Stock Units Activity | Restricted Stock Units— The fair value of RSU’s is estimated based upon the closing market price of the Company’s common stock on the date of grant. RSUs generally vest annually over a four-year period. The following table summarizes the Company’s RSU's activity: Number of Stock Units Weighted Average Grant Date Fair Value Per Share Unvested Balance at December 31, 2022 3,572 $ 42.23 Granted 2,916 52.19 Vested ( 1,015 ) 41.52 Forfeited ( 1,172 ) 48.43 Unvested Balance at December 31, 2023 4,301 47.48 |
Net Loss per Common Share (Tabl
Net Loss per Common Share (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Earnings Per Share [Abstract] | |
Summary of Basic and Diluted Net Loss per Common Share | The following table presents the calculation of basic and diluted net loss per common share (amounts in thousands except per share amounts): Year Ended December 31, 2023 2022 2021 Numerator: Net loss $ ( 528,628 ) $ ( 652,172 ) $ ( 746,354 ) Denominator: Weighted-average number of common shares used in net loss per common share - basic and diluted 118,678 106,114 84,421 Net loss per common share -- basic and diluted $ ( 4.45 ) $ ( 6.15 ) $ ( 8.84 ) |
Summary of Shares Outstanding that were Excluded from Calculation of Diluted Net Loss Per Share | Shares outstanding presented below were excluded from the calculation of diluted net loss per share, prior to the use of the if-converted-method and treasury stock method, as their effect is anti-dilutive (in thousands): Year Ended December 31, 2023 2022 2021 Convertible notes 2,379 2,379 4,865 Common stock under option 8,664 12,295 12,562 Restricted stock units 4,301 3,572 1,224 Total 15,344 18,246 18,651 |
Nature of Organization and Op_2
Nature of Organization and Operations - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |||||
Feb. 22, 2023 | Mar. 28, 2022 | Nov. 18, 2021 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Nature Of Organization And Operations [Line Items] | ||||||
Proceeds from follow-on public offering after deducting underwriting discounts and commissions | $ 380,120 | $ 380,363 | ||||
Accumulated deficit | $ (2,837,488) | (2,308,860) | ||||
Cash and cash equivalents | $ 351,185 | $ 551,801 | $ 640,192 | |||
Follow-on Public Offerings [Member] | ||||||
Nature Of Organization And Operations [Line Items] | ||||||
Issuance of common stock and pre-funded warrants in common stock offering, shares | 4,007,936 | |||||
Pre funded warrant price | $ 62.9999 | |||||
Common stock pre funded warrants per warrant | 0.0001 | |||||
Issuance of common stock in follow-on offering, shares | 8,563,830 | 10,062,500 | ||||
Price of common stock | $ 63 | $ 47 | $ 40 | |||
Proceeds from follow-on public offering after deducting underwriting discounts and commissions | $ 384,400 | $ 380,100 | $ 380,400 | |||
Underwriting discounts and commissions | 18,800 | 22,100 | 22,100 | |||
Offering Costs | $ 300 | $ 300 | $ 600 | |||
Over Allotment Option [Member] | ||||||
Nature Of Organization And Operations [Line Items] | ||||||
Issuance of common stock in follow-on offering, shares | 1,117,021 | 1,312,500 | ||||
Price of common stock | $ 47 | $ 40 | ||||
Common Stock [Member] | ||||||
Nature Of Organization And Operations [Line Items] | ||||||
Issuance of common stock and pre-funded warrants in common stock offering, shares | 4,008,000 | |||||
Issuance of common stock in follow-on offering, shares | 8,564,000 | 10,063,000 | ||||
Common Stock [Member] | Follow-on Public Offerings [Member] | ||||||
Nature Of Organization And Operations [Line Items] | ||||||
Issuance of common stock and pre-funded warrants in common stock offering, shares | 2,380,956 | |||||
Common Stock [Member] | Over Allotment Option [Member] | ||||||
Nature Of Organization And Operations [Line Items] | ||||||
Issuance of common stock and pre-funded warrants in common stock offering, shares | 833,333 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) $ in Thousands | 1 Months Ended | 8 Months Ended | 12 Months Ended | ||
Jan. 31, 2022 USD ($) | Jun. 30, 2021 USD ($) | Jan. 31, 2020 USD ($) | Dec. 31, 2023 USD ($) Segment | Dec. 31, 2022 USD ($) | |
Schedule Of Summary Of Significant Accounting Policies [Line Items] | |||||
Number of operating segment | Segment | 1 | ||||
Inventory | $ 146,362 | $ 85,714 | |||
SFJ Agreement [Member] | |||||
Schedule Of Summary Of Significant Accounting Policies [Line Items] | |||||
Payment made under agreement | $ 5,000 | $ 4,000 | $ 80,000 | 94,000 | |
Regulatory Approval Granted US Food and Drug Administration [Member] | |||||
Schedule Of Summary Of Significant Accounting Policies [Line Items] | |||||
Inventory | $ 19,400 |
Product Revenues, Accounts Re_3
Product Revenues, Accounts Receivable, and Reserves for Product Sales - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Total product revenue, net | $ 396,591 | $ 75,422 | $ 66,563 |
Accounts receivable | 206,442 | 7,727 | |
Accrued Expenses [Member] | |||
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Product sales reserves | 16,600 | 2,400 | |
Product revenue, net [Member] | |||
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Total product revenue, net | $ 366,281 | $ 65,092 | $ 15,147 |
Product Revenues, Accounts Re_4
Product Revenues, Accounts Receivable, and Reserves for Product Sales - Schedule of Disaggregation of Product Revenue by Major Source (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Total product revenue, net | $ 396,591 | $ 75,422 | $ 66,563 |
EMPAVELI | |||
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Total product revenue, net | 91,033 | 65,092 | 15,147 |
SYFOVRE [Member] | |||
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Total product revenue, net | 275,248 | ||
Product revenue, net [Member] | |||
Product Revenues Accounts Receivable And Reserves For Product Sales [Line Items] | |||
Total product revenue, net | $ 366,281 | $ 65,092 | $ 15,147 |
Product Revenues, Accounts Re_5
Product Revenues, Accounts Receivable, and Reserves for Product Sales - Summary of Product Revenue Allowance and Reserve Categories (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Chargebacks, Discounts, and Fees [Member] | ||
SEC Schedule, 12-09, Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning Balance | $ 164 | $ 67 |
Provision related to sales in the current year | 17,690 | 614 |
Adjustments related to prior period sales | (112) | (156) |
Credits and payments made | (12,068) | (362) |
Ending balance | 5,674 | 164 |
Government and Other Rebates [Member] | ||
SEC Schedule, 12-09, Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning Balance | 1,936 | 716 |
Provision related to sales in the current year | 26,661 | 4,569 |
Adjustments related to prior period sales | (1,223) | (246) |
Credits and payments made | (18,476) | (3,103) |
Ending balance | 8,898 | 1,936 |
Returns [Member] | ||
SEC Schedule, 12-09, Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning Balance | 251 | 167 |
Provision related to sales in the current year | 4,698 | 1,185 |
Adjustments related to prior period sales | (2,481) | (385) |
Credits and payments made | (415) | (716) |
Ending balance | 2,053 | 251 |
Product Revenue Allowance and Reserves [Member] | ||
SEC Schedule, 12-09, Movement in Valuation Allowances and Reserves [Roll Forward] | ||
Beginning Balance | 2,351 | 951 |
Provision related to sales in the current year | 49,050 | 6,368 |
Adjustments related to prior period sales | (3,817) | (787) |
Credits and payments made | (30,960) | (4,181) |
Ending balance | $ 16,625 | $ 2,351 |
Product Revenues, Accounts Re_6
Product Revenues, Accounts Receivable, and Reserves for Product Sales - Schedule of Customer Accounted for 10% of More of Gross Product Revenues and Accounts Receivable (Detail) - Customer Concentration Risk [Member] | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Gross Product Revenues [Member] | Customer A [Member] | |||
Revenue, Major Customer [Line Items] | |||
Concentration risk | 24% | 99% | 99% |
Gross Product Revenues [Member] | Customer C [Member] | |||
Revenue, Major Customer [Line Items] | |||
Concentration risk | 16% | ||
Gross Product Revenues [Member] | Customer D [Member] | |||
Revenue, Major Customer [Line Items] | |||
Concentration risk | 54% | 1% | |
Accounts Receivable [Member] | Customer A [Member] | |||
Revenue, Major Customer [Line Items] | |||
Concentration risk | 4% | 96% | |
Accounts Receivable [Member] | Customer C [Member] | |||
Revenue, Major Customer [Line Items] | |||
Concentration risk | 66% | ||
Accounts Receivable [Member] | Customer D [Member] | |||
Revenue, Major Customer [Line Items] | |||
Concentration risk | 22% | 3% |
Inventory - Schedule Of Invento
Inventory - Schedule Of Inventory Current (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 32,724 | $ 29,847 |
Semi-finished goods | 82,924 | 54,101 |
Finished goods | 30,714 | 1,766 |
Total inventories | $ 146,362 | $ 85,714 |
Inventory - Additional Informat
Inventory - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Inventory Disclosure [Abstract] | ||
Inventory write-downs | $ 6.4 | $ 1.5 |
Prepaid and Other Current Ass_3
Prepaid and Other Current Assets - Summary of Prepaid Assets and Other Current Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 | |
Prepaid Expense and Other Assets, Current [Abstract] | |||
Down payments for inventory | $ 16,296 | $ 13,987 | |
Prepaid research and development | 13,931 | 15,181 | |
Other prepaid expenses | 8,593 | 7,182 | |
Total prepaid expenses | 38,820 | 36,350 | |
Royalties receivable | 3,054 | 1,442 | |
ERC credit | 8,711 | ||
Receivable from collaboration agreement | [1] | 15,000 | 20,000 |
Deposits and Other Current Assets | 4,354 | 6,505 | |
Total other current assets | $ 22,408 | $ 36,658 | |
[1] In January 2024 the Company waived the remaining reimbursement payment of $ 15.0 million in connection with the decision to discontinue the CAD program. |
Prepaid and Other Current Ass_4
Prepaid and Other Current Assets - Summary of Prepaid Assets and Other Current Assets (Parenthetical) (Details) $ in Millions | 1 Months Ended |
Jan. 31, 2024 USD ($) | |
Subsequent Event [Member] | |
Prepaid Expense and Other Current Assets [Line Items] | |
Development cost reimbursement received | $ 15 |
Prepaid and Other Current Ass_5
Prepaid and Other Current Assets - Additional Information (Details) | 1 Months Ended | 12 Months Ended | ||
Sep. 30, 2021 USD ($) | Nov. 30, 2022 USD ($) | Dec. 31, 2023 Employees | Dec. 31, 2021 | |
Prepaid Expense and Other Assets, Current [Abstract] | ||||
Maximum number of employees with employers eligible for refundable tax credit | Employees | 500 | |||
Percentage of qualified wages paid to employees under employee retention credit | 70% | |||
Maximum credit per employee for each calendar quarter | $ 7,000 | |||
Refund filed under CARES Act relating to employee retention credit | $ 8,700,000 |
Development Liability - Additio
Development Liability - Additional Information (Detail) $ in Thousands | 1 Months Ended | 8 Months Ended | 12 Months Ended | |||||||||||
Feb. 28, 2019 USD ($) Installment | Dec. 31, 2023 USD ($) | May 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | May 31, 2022 USD ($) | Jan. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) Installment | Dec. 31, 2021 USD ($) | Jun. 30, 2021 USD ($) | Jan. 31, 2020 USD ($) | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Jun. 30, 2019 USD ($) | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Interest expense | $ 29,581 | $ 32,626 | $ 13,241 | |||||||||||
SFJ Agreement [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Proceeds from SFJ agreement | $ 60,000 | |||||||||||||
Additional funding amount upon achievement of development milestones | $ 60,000 | |||||||||||||
Number of milestone payments | Installment | 3 | |||||||||||||
Development funding for minimum period of operating expense | 10 months | |||||||||||||
Increase in additional funding for development costs | $ 20,000 | |||||||||||||
Obligated to pay initial payment | $ 366,000 | $ 5,000 | $ 5,000 | 366,000 | 5,000 | |||||||||
Aggregate amount of additional annual payments | $ 225,000 | 225,000 | $ 225,000 | |||||||||||
Number of additional annual payments | Installment | 6 | |||||||||||||
Payment made under agreement | $ 5,000 | $ 4,000 | $ 80,000 | 94,000 | ||||||||||
First annual payment under agreement | $ 18,000 | $ 11,500 | ||||||||||||
Second annual payment under agreement | $ 31,000 | $ 24,500 | ||||||||||||
Interest expense | $ 1,200 | $ 26,000 | $ 26,900 | |||||||||||
SFJ Agreement [Member] | Regulatory Approval Granted US Food and Drug Administration [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Obligated to pay initial payment | $ 4,000 | |||||||||||||
Aggregate amount of additional annual payments | $ 226,000 | |||||||||||||
Number of additional annual payments | Installment | 6 |
Development Liability - Summary
Development Liability - Summary of Development Liability (Detail) - SFJ Agreement [Member] - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Development liability | $ 366,000 | $ 421,500 |
Less: Unamortized discount to development liability | (50,353) | (76,349) |
Less: Current portion of development liability, net of discount | (75,830) | (29,504) |
Total long term development liability | $ 239,817 | $ 315,647 |
Effective Interest Rate | 7.91% | 7.91% |
Development Liability - Schedul
Development Liability - Schedule of Future Minimum SFJ Payments (Details) - S F J Agreement [Member] - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2021 |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||
2024 | $ 98,750 | |
2025 | 103,000 | |
2026 | 109,000 | |
2027 | 55,250 | |
Total future minimum payments | $ 366,000 | $ 5,000 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Accrued research and development | $ 28,318 | $ 34,849 |
Accrued royalties | 10,197 | 907 |
Accrued payroll liabilities | 51,781 | 43,212 |
Product revenue reserves | 16,625 | 2,351 |
Other | 20,885 | 13,820 |
Total accrued expenses | $ 127,806 | $ 95,139 |
Long-term Debt - Additional Inf
Long-term Debt - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 4 Months Ended | 12 Months Ended | ||||||||||
May 12, 2020 USD ($) | Sep. 16, 2019 USD ($) | Jul. 31, 2022 USD ($) shares | Jul. 31, 2021 USD ($) shares | Jun. 30, 2021 USD ($) shares | Jan. 31, 2021 USD ($) shares | Sep. 30, 2021 USD ($) | Mar. 31, 2021 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2023 USD ($) Days $ / shares | Dec. 31, 2022 USD ($) shares | Dec. 31, 2021 USD ($) shares | Dec. 31, 2020 USD ($) | Sep. 11, 2019 USD ($) CounterParty $ / shares | |
Debt Instrument [Line Items] | ||||||||||||||
Aggregate principal amount converted | $ 98,086,000 | $ 328,017,000 | ||||||||||||
Loss on conversion of debt | 32,890,000 | 100,589,000 | ||||||||||||
Forfeiture of accrued interest in exchange of convertible notes | 1,287,000 | 4,171,000 | ||||||||||||
Debt interest expense | 5,707,000 | $ 12,050,000 | $ 3,583,000 | |||||||||||
Amortization of debt issuance costs | $ 297,000 | $ 459,000 | ||||||||||||
Long-term debt | $ 93,033,000 | |||||||||||||
Common Stock [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Shares issued | shares | 3,073,000 | 10,065,000 | ||||||||||||
2019 Convertible Notes [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Premium paid for capped call transactions | $ 28,400,000 | |||||||||||||
2020 Convertible Notes [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Premium paid for capped call transactions | $ 43,100,000 | |||||||||||||
Capped Call Transactions [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Number of counterparties | CounterParty | 2 | |||||||||||||
Initial conversion price | $ / shares | $ 39.4625 | |||||||||||||
Initial cap price | $ / shares | $ 63.14 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Debt instrument, stated percentage | 3.50% | |||||||||||||
Debt instrument, due and payment description | The Convertible Notes are senior unsecured obligations of the Company and bear interest at a rate of 3.5% per year payable semiannually in arrears on March 15 and September 15 of each year, beginning on March 15, 2020. | |||||||||||||
Debt instrument, frequency of periodic payment | semiannually | |||||||||||||
Maturity date | Sep. 15, 2026 | |||||||||||||
Threshold trading days | Days | 20 | |||||||||||||
Threshold consecutive trading days | Days | 30 | |||||||||||||
Threshold percentage of stock price trigger | 130% | |||||||||||||
Redemption period, start date | Sep. 20, 2023 | |||||||||||||
Redemption price, percentage | 100% | |||||||||||||
Aggregate principal amount converted | $ 700,000 | $ 0 | ||||||||||||
Aggregate principal amount converted into shares | shares | 18,775 | |||||||||||||
Long-term debt | 92,736,000 | $ 93,033,000 | $ 92,736,000 | |||||||||||
Convertible Senior Notes Due 2026 [Member] | Prior to March 15, 2026 Convertible [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Threshold trading days | Days | 20 | |||||||||||||
Threshold consecutive trading days | Days | 30 | |||||||||||||
Threshold percentage of stock price trigger | 130% | |||||||||||||
Trading price per principal amount | $ 1,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | Prior to March 15, 2026 Convertible [Member] | Maximum [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Threshold percentage of stock price trigger | 98% | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | Common Stock [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Terms of conversion | The Convertible Notes are convertible into shares of the Company’s common stock at an initial conversion rate of 25.3405 shares per $1,000 principal amount of Convertible Notes (equivalent to an initial conversion price of approximately $39.4625 per share of common stock). | |||||||||||||
Conversion ratio | 0.0253405 | |||||||||||||
Conversion price | $ / shares | $ 39.4625 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2019 Convertible Notes [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Net proceeds from the sale of the notes | $ 212,900,000 | |||||||||||||
Payment of convertible debt discounts and commissions | 6,600,000 | |||||||||||||
Payment of convertible debt offering expenses | 500,000 | |||||||||||||
Payments of transactions cost | 28,400,000 | |||||||||||||
Debt instrument effective interest rate | 10.50% | |||||||||||||
Debt instrument convertible carrying amount of liability component | $ 145,100,000 | |||||||||||||
Debt discount | 74,900,000 | |||||||||||||
Debt issuance costs | 7,100,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2019 Convertible Notes [Member] | Exchange Agreements [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Conversion ratio | 0.0253405 | 0.0253405 | ||||||||||||
Debt issuance costs | $ 4,100,000 | $ 3,300,000 | ||||||||||||
Increase (decrease) in net debt | 197,000,000 | 122,800,000 | ||||||||||||
Aggregate principal amount converted | $ 201,100,000 | $ 126,100,000 | ||||||||||||
Increase in shares outstanding | shares | 5,992,217 | 3,906,869 | ||||||||||||
Shares issued | shares | 5,097,166 | 3,196,172 | ||||||||||||
Additional shares issued | shares | 895,051 | 710,697 | ||||||||||||
Additional shares issued for settlement of debt issuance cost paid | shares | 78,419 | 69,491 | ||||||||||||
Loss on conversion of debt | $ 61,100,000 | $ 39,500,000 | ||||||||||||
Loss on conversion of debt related to additional shares issued | 55,900,000 | 36,400,000 | ||||||||||||
Additional amount issued in payment of issuance costs | $ 5,200,000 | $ 3,100,000 | ||||||||||||
Forfeiture of accrued interest in exchange of convertible notes | $ 1,700,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2019 Convertible Notes [Member] | Common Stock [Member] | Exchange Agreements [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Aggregate principal amount converted into shares | shares | 3,906,869 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2019 Convertible Notes [Member] | Liability Component [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Debt issuance costs | 4,700,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2019 Convertible Notes [Member] | Equity Component [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Debt issuance costs | $ 2,400,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2020 Convertible Notes [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Net proceeds from the sale of the notes | $ 322,900,000 | |||||||||||||
Payment of convertible debt discounts and commissions | 5,700,000 | |||||||||||||
Payment of convertible debt offering expenses | 300,000 | |||||||||||||
Payments of transactions cost | $ 43,100,000 | |||||||||||||
Debt instrument effective interest rate | 10.50% | |||||||||||||
Debt instrument convertible carrying amount of liability component | $ 204,500,000 | |||||||||||||
Debt discount | 95,500,000 | |||||||||||||
Debt issuance costs | 6,000,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2020 Convertible Notes [Member] | Exchange Agreements [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Conversion ratio | 0.0253405 | |||||||||||||
Debt issuance costs | $ 1,300,000 | |||||||||||||
Increase (decrease) in net debt | 96,800,000 | |||||||||||||
Aggregate principal amount converted | $ 98,100,000 | |||||||||||||
Shares issued | shares | 2,485,548 | |||||||||||||
Additional shares issued | shares | 541,470 | |||||||||||||
Additional shares issued for settlement of debt issuance cost paid | shares | 46,132 | |||||||||||||
Loss on conversion of debt | $ 32,900,000 | |||||||||||||
Loss on conversion of debt related to additional shares issued | 30,400,000 | |||||||||||||
Additional amount issued in payment of issuance costs | $ 2,500,000 | |||||||||||||
Convertible notes held in treasury | $ 425,400,000 | $ 425,400,000 | $ 425,400,000 | |||||||||||
Convertible Senior Notes Due 2026 [Member] | 2020 Convertible Notes [Member] | Common Stock [Member] | Exchange Agreements [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Aggregate principal amount converted into shares | shares | 3,027,018 | 5,992,217 | ||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2020 Convertible Notes [Member] | Liability Component [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Debt issuance costs | 3,700,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2020 Convertible Notes [Member] | Equity Component [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Debt issuance costs | 2,300,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | 2019 and 2020 Convertible Notes [Member] | Exchange Agreements [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Aggregate principal amount converted | $ 201,100,000 | |||||||||||||
Forfeiture of accrued interest in exchange of convertible notes | $ 2,500,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | Convertible Senior Notes [Member] | Exchange Agreements [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Increase in shares outstanding | shares | 3,027,018 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | Private Offering [Member] | 2019 Convertible Notes [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Principal amount | $ 220,000,000 | |||||||||||||
Convertible Senior Notes Due 2026 [Member] | Private Offering [Member] | 2020 Convertible Notes [Member] | ||||||||||||||
Debt Instrument [Line Items] | ||||||||||||||
Principal amount | $ 300,000,000 |
Long-term Debt - Schedule of Ou
Long-term Debt - Schedule of Outstanding Balance of Convertible Notes (Detail) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Debt Instrument [Line Items] | ||
Principal | $ 102,797 | |
Less: debit discount and issuance costs, net | (864) | |
Net carrying amount | 93,033 | |
Convertible Senior Notes Due 2026 [Member] | ||
Debt Instrument [Line Items] | ||
Principal | 93,897 | $ 93,897 |
Less: debit discount and issuance costs, net | 864 | (1,161) |
Net carrying amount | $ 93,033 | $ 92,736 |
Long-term Debt - Schedule of In
Long-term Debt - Schedule of Interest Expense Recognized Related to Convertible Notes (Detail) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Interest Expense, Debt [Abstract] | ||||
Amortization of debt discount | $ 297 | $ 459 | ||
Contractual interest expense | $ 3,286 | 5,248 | $ 11,086 | |
Total interest expense | $ 5,707 | $ 12,050 | $ 3,583 |
Long-term Debt - Schedule of Fu
Long-term Debt - Schedule of Future Minimum Payments on Convertible Notes Payable (Detail) $ in Thousands | Dec. 31, 2023 USD ($) |
Debt Disclosure [Abstract] | |
2024 | $ 3,286 |
2025 | 3,286 |
2026 | 96,225 |
Total future minimum payments | 102,797 |
Less: interest | (8,900) |
Less: debit discount and issuance costs, net | (864) |
Net carrying amount | $ 93,033 |
Leases - Additional Information
Leases - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Lessee Lease Description [Line Items] | |||
Operating lease description | The Company enters into lease agreements with terms generally ranging from 2-7 years. Some of the Company’s lease agreements include Company options to extend the lease on a month-to-month basis or for set periods for up to five years. Many leases also include options to terminate the leases within one year or per other contractual terms. Renewal and termination options were generally not included in the lease term for the Company’s existing operating leases. | ||
Operating lease, existence of option to extend | true | ||
Operating lease maximum term of options to terminate lease | 1 year | ||
Operating lease expense | $ 7 | $ 6.2 | $ 5.6 |
Minimum [Member] | |||
Lessee Lease Description [Line Items] | |||
Operating lease, term of contract | 2 years | ||
Maximum [Member] | |||
Lessee Lease Description [Line Items] | |||
Operating lease, term of contract | 7 years | ||
Operating lease options to extend lease | 5 years |
Leases - Schedule of Additional
Leases - Schedule of Additional Information Related to Operating Lease Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Leases [Abstract] | ||
Operating lease assets | $ 16,745 | $ 18,747 |
Operating lease liabilities | $ 17,895 | $ 19,977 |
Weighted average remaining term in years | 2 years 9 months 29 days | 3 years 6 months 25 days |
Weighted average discount rate used to measure outstanding lease liabilities | 7.20% | 7.26% |
Leases - Supplemental Cash Flow
Leases - Supplemental Cash Flow Information Related to Operating Leases (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Leases [Abstract] | |||
Operating cash flows from operating leases | $ 7,939 | $ 7,375 | $ 5,989 |
Operating lease assets obtained in exchange for lease obligations | $ 2,700 | $ 5,675 |
Leases - Maturity of Operating
Leases - Maturity of Operating Lease Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Leases [Abstract] | ||
2024 | $ 7,393 | |
2025 | 6,238 | |
2026 | 5,299 | |
2027 | 859 | |
Total future minimum lease payments | 19,789 | |
Less imputed interest | (1,894) | |
Total operating lease liabilities | $ 17,895 | $ 19,977 |
Other Comprehensive Income an_3
Other Comprehensive Income and Accumulated Other Comprehensive Income - Summary of Changes in Accumulated Other Comprehensive Income/(Loss), by Component (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Accumulated Other Comprehensive Income Loss [Line Items] | ||
Beginning balance | $ 169,872 | $ 198,662 |
Ending balance | 194,521 | 169,872 |
Unrealized Gains (Losses) from Marketable Securities [Member] | ||
Accumulated Other Comprehensive Income Loss [Line Items] | ||
Beginning balance | 1 | |
Net other comprehensive income (loss) | (1) | |
Foreign Currency Translation Adjustment [Member] | ||
Accumulated Other Comprehensive Income Loss [Line Items] | ||
Beginning balance | (2,521) | (2,091) |
Net other comprehensive income (loss) | (49) | (430) |
Ending balance | (2,570) | (2,521) |
Unrealized Gains (Losses) from Pension Plan [Member] | ||
Accumulated Other Comprehensive Income Loss [Line Items] | ||
Beginning balance | 1,646 | |
Net other comprehensive income (loss) | (2,618) | 1,646 |
Ending balance | (972) | 1,646 |
Total Accumulated Other Comprehensive Income (Loss) [Member] | ||
Accumulated Other Comprehensive Income Loss [Line Items] | ||
Beginning balance | (875) | (2,090) |
Net other comprehensive income (loss) | (2,667) | 1,215 |
Ending balance | $ (3,542) | $ (875) |
Fair Value Measurements - Sched
Fair Value Measurements - Schedule of Financial Instruments and the Related Fair Value Hierarchy of the Valuation Techniques Utilized (Detail) - Fair Value, Nonrecurring [Member] - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Total Financial Assets | $ 276,391 | $ 527,728 |
Money Market Funds [Member] | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Total Financial Assets | 276,391 | 527,728 |
Level 1 [Member] | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Total Financial Assets | 276,391 | 527,728 |
Level 1 [Member] | Money Market Funds [Member] | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Total Financial Assets | $ 276,391 | $ 527,728 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) $ in Millions | Dec. 31, 2023 | Dec. 31, 2022 |
Level 1 [Member] | Convertible Senior Notes [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Convertible notes | $ 140.8 | $ 143.9 |
Convertible notes, interest rate | 3.50% | 3.50% |
Level 2 [Member] | Development Liability [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Fair value | $ 306.9 | $ 315.8 |
License and Collaboration Agr_2
License and Collaboration Agreements - Additional Information (Detail) | 1 Months Ended | 12 Months Ended | ||||||||||||||||
Nov. 30, 2020 USD ($) | Oct. 27, 2020 USD ($) | Jan. 31, 2024 USD ($) | Nov. 30, 2023 USD ($) | Oct. 31, 2023 USD ($) | Apr. 30, 2023 USD ($) | Jan. 31, 2023 USD ($) | Jun. 30, 2022 USD ($) | Apr. 30, 2022 USD ($) | Jan. 31, 2022 USD ($) | Aug. 31, 2021 USD ($) | Jun. 30, 2021 Program | Jan. 31, 2021 USD ($) | Dec. 31, 2023 USD ($) License | Dec. 31, 2022 USD ($) | Dec. 31, 2020 USD ($) | Jul. 31, 2021 USD ($) | Oct. 31, 2020 USD ($) | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Royalties receivable | $ 3,054,000 | $ 1,442,000 | ||||||||||||||||
Accrued expenses | 127,806,000 | 95,139,000 | ||||||||||||||||
Subsequent Event [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Development cost reimbursement received | $ 15,000,000 | |||||||||||||||||
SYFOVRE [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Royalty expense incurred | 8,900,000 | |||||||||||||||||
Regulatory milestone payments | $ 2,000,000 | $ 2,300,000 | ||||||||||||||||
Regulatory milestone incurred | 5,000,000 | |||||||||||||||||
Regulatory milestone accrued | 3,000,000 | |||||||||||||||||
Empaveli And Aspaveli [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Royalty expense incurred | 7,900,000 | |||||||||||||||||
Accrued expenses | 1,500,000 | |||||||||||||||||
Swedish Orphan Biovitrum AB (Publ) [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Development cost reimbursement | $ 80,000,000 | |||||||||||||||||
Non-refundable Upfront Payment | 250,000,000 | |||||||||||||||||
Royalty revenue | $ 10,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Development cost reimbursement | $ 80,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | License [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Upfront payment | $ 250,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | Swedish Orphan Biovitrum AB (Publ) [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Upfront payment | $ 250,000,000 | |||||||||||||||||
Milestone payment received for achievement of regulatory development milestone | $ 5,000,000 | $ 50,000,000 | ||||||||||||||||
Development cost reimbursement | $ 80,000,000 | |||||||||||||||||
Development cost reimbursement received | $ 20,000,000 | $ 20,000,000 | 25,000,000 | |||||||||||||||
Royalties entitled to receive after first commercial sale of applicable licensed product period | 10 years | |||||||||||||||||
Royalty revenue | 3,000,000 | |||||||||||||||||
Royalties received | $ 10,000,000 | |||||||||||||||||
Contract research and development expense | 0 | 5,000,000 | ||||||||||||||||
Remaining reimbursement payment | 15,000,000 | |||||||||||||||||
Contract research and development reimbursement commitment | 80,000,000 | |||||||||||||||||
Contract research and development current | 15,000,000 | |||||||||||||||||
Contract research and development receivable | 35,000,000 | |||||||||||||||||
Contract research and development receivable other current assets | 20,000,000 | |||||||||||||||||
Contract research and development receivable other assets | $ 15,000,000 | |||||||||||||||||
Proceeds from contract research and development | 20,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | Swedish Orphan Biovitrum AB (Publ) [Member] | Subsequent Event [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Remaining reimbursement payment | $ 15,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | Swedish Orphan Biovitrum AB (Publ) [Member] | ASC Topic 808 | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Upfront payment | $ 250,000,000 | |||||||||||||||||
Development cost reimbursement | 80,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | Swedish Orphan Biovitrum AB (Publ) [Member] | Maximum [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Aggregate milestone payments upon achievement of specified one time regulatory development and commercial milestone events | $ 915,000,000 | |||||||||||||||||
Collaboration and License Agreement [Member] | Swedish Orphan Biovitrum AB (Publ) [Member] | Maximum [Member] | ASC Topic 808 | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Aggregate milestone payments upon achievement of specified one-time regulatory and commercial milestone events | $ 915,000,000 | |||||||||||||||||
University of Pennsylvania [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Annual maintenance fees | 100,000 | |||||||||||||||||
Development milestone achievement | $ 5,000,000 | $ 1,000,000 | ||||||||||||||||
University of Pennsylvania [Member] | Empaveli And Aspaveli [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Milestone payments based on annual sales milestones | $ 1,000,000 | |||||||||||||||||
University of Pennsylvania [Member] | Maximum [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Aggregate milestones payments | 3,200,000 | |||||||||||||||||
Milestone payments based on annual sales milestones | 5,000,000 | |||||||||||||||||
2010 License Agreement [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Annual maintenance fees | $ 100,000 | |||||||||||||||||
Number of licensed products | License | 2 | |||||||||||||||||
2010 License Agreement [Member] | Maximum [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Aggregate milestones payments | $ 1,700,000 | |||||||||||||||||
Milestone payments based on annual sales milestones | 2,500,000 | |||||||||||||||||
Sobi Agreement and Another Licensing Transaction [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Sublicense fee owed | $ 25,000,000 | $ 500,000 | ||||||||||||||||
Research Collaboration Agreement [Member] | Beam [Member] | ||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||
Collaboration agreement term | 5 years | |||||||||||||||||
Number of research programs | Program | 6 | |||||||||||||||||
Up-front non-refundable payment | $ 50,000,000 | |||||||||||||||||
Up-front payment on first anniversary of agreement on June 30, 2022 | $ 25,000,000 | |||||||||||||||||
Collaboration agreement maximum extendable term | 2 years |
Employee Retirement Plans - Add
Employee Retirement Plans - Additional Information (Detail) - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Defined Benefit Plan Disclosure [Line Items] | |||
Defined Contribution Plan, Tax Status [Extensible Enumeration] | us-gaap:QualifiedPlanMember | us-gaap:QualifiedPlanMember | us-gaap:QualifiedPlanMember |
Minimum age of full-time employees eligible to participate in the plan | 21 years | ||
Employer contributions made to the 401(k) Plan | $ 5,700,000 | $ 4,300,000 | $ 3,100,000 |
Swiss Pension Plan [Member] | |||
Defined Benefit Plan Disclosure [Line Items] | |||
Funded status | 2,200,000 | 18,000 | |
Fair value of plan assets | 16,200,000 | 12,900,000 | |
Projected benefit obligation | 18,400,000 | 12,900,000 | |
Accumulated benefit obligation | 2,600,000 | 1,600,000 | |
Net periodic benefit cost | $ 900,000 | $ 600,000 |
Income Taxes - Components of Lo
Income Taxes - Components of Loss From Continuing Operation Before Provision for Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Components of Income Tax Expense (Benefit), Continuing Operations [Abstract] | |||
United States | $ (48,495) | $ (82,815) | $ (314,673) |
Foreign | (478,001) | (568,688) | (431,329) |
Net loss before taxes | $ (526,496) | $ (651,503) | $ (746,002) |
Income Taxes - Components of Pr
Income Taxes - Components of Provision for Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Current income tax expense: | |||
U.S. State and Local | $ 1,869 | $ 520 | $ 205 |
Foreign | 263 | 149 | 147 |
Total current income tax expense | 2,132 | 669 | 352 |
Deferred income tax expense: | |||
Total tax expense | $ 2,132 | $ 669 | $ 352 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation Between U.S. Federal Statutory Rate and Effective Tax rate, Amount (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Expense (Benefit), Continuing Operations [Abstract] | |||
Statutory U.S. federal income tax, Amount | $ (110,564) | $ (136,816) | $ (156,660) |
Foreign tax rate differential | 42,100 | 50,219 | 38,677 |
State income taxes, net of federal benefit, Amount | (13,438) | 9,051 | (14,145) |
Change in valuation allowances, Amount | 119,592 | 94,668 | 133,668 |
Tax credits, Amount | (11,566) | (19,966) | (20,005) |
Share Based Compensation | (26,881) | ||
Change in state apportionment, Amount | (35) | ||
Loss on debt conversion | 6,626 | 19,548 | |
Permanent and other | 2,889 | (3,078) | (731) |
Total tax expense | $ 2,132 | $ 669 | $ 352 |
Income Taxes - Schedule of Re_2
Income Taxes - Schedule of Reconciliation Between U.S. Federal Statutory Rate and Effective Tax rate (Detail) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Expense (Benefit), Continuing Operations [Abstract] | |||
Statutory U.S. federal income tax, Percentage of income before income taxes | 21% | 21% | 21% |
Foreign tax rate differential | (8.00%) | (7.70%) | (5.20%) |
State income taxes, net of federal benefit, Percentage of income before income taxes | 2.60% | (1.40%) | 1.90% |
Change in valuation allowances, Percentage of income before income taxes | (23.20%) | (14.50%) | (17.90%) |
Tax credits, Percentage of income before income taxes | 2.20% | 3% | 2.60% |
Share Based Compensation | 5.10% | ||
Loss on debt conversion | (1.00%) | (2.60%) | |
Permanent and other | (0.10%) | 0.50% | 0.10% |
Effective income tax provision, Percentage of income before income taxes | (0.40%) | (0.10%) | (0.10%) |
Income Taxes - Schedule of Defe
Income Taxes - Schedule of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Deferred tax assets: | ||
Intangible assets | $ 192,444 | $ 187,839 |
Share-based compensation | 31,712 | 29,673 |
Net operating loss carryforwards | 329,135 | 228,985 |
Research and development credits | 67,667 | 57,419 |
Orphan drug credits | 34,023 | 30,160 |
Development derivative liability | 75,190 | 79,374 |
Convertible debt | 5,582 | 6,850 |
Fixed Assets | 18 | |
Lease liability | 2,888 | 4,304 |
Accruals | 17,401 | 16,272 |
Total deferred tax assets | 756,060 | 640,876 |
Deferred tax liabilities: | ||
Fixed assets | (91) | |
Right-of-use asset | (2,672) | (4,032) |
481(a) adjustment | (233) | |
Total deferred tax liabilities | (2,672) | (4,356) |
Net deferred tax assets before allowance: | 753,388 | 636,520 |
Less valuation allowance | $ (753,388) | $ (636,520) |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Income Taxes [Line Items] | ||
Deferred tax asset, increase in valuation allowance | $ 116,900,000 | |
Capitalization of R&D costs | $ 42,300,000 | $ 47,600,000 |
Net operating loss carryforward period | 3 years | |
Interest and penalties | $ 0 | 0 |
Accrued for interest and penalties | $ 0 | 0 |
Income tax examination description | Generally, the tax years 2020 through 2022 remain open and subject to examination by the major taxing jurisdictions to which the Company is subject. | |
U.S [Member] | ||
Income Taxes [Line Items] | ||
R&D costs amortization period | 5 years | |
Non - U.S [Member] | ||
Income Taxes [Line Items] | ||
R&D costs amortization period | 15 years | |
Federal [Member] | ||
Income Taxes [Line Items] | ||
Net operating loss carryforwards | $ 494,100,000 | 312,800,000 |
Research and development tax credit carryforwards | 85,700,000 | |
Net operating loss carryforward indefinitely | $ 449,800,000 | |
Net operating loss carryforward expiration year | 2025 | |
Research and development tax credit carryforward expiration year | 2025 | |
State [Member] | ||
Income Taxes [Line Items] | ||
Net operating loss carryforwards | $ 524,000,000 | 395,400,000 |
Research and development tax credit carryforwards | $ 20,200,000 | |
Net operating loss carryforward expiration year | 2025 | |
Research and development tax credit carryforward expiration year | 2025 | |
Foreign [Member] | ||
Income Taxes [Line Items] | ||
Net operating loss carryforwards | $ 1,663,300,000 | $ 1,204,700,000 |
Net operating loss carryforward expiration year | 2027 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 12 Months Ended |
Dec. 31, 2023 USD ($) | |
Commitments And Contingencies [Line Items] | |
Incurred any cost to defend lawsuits or settle claims | $ 0 |
Vehicles [Member] | |
Commitments And Contingencies [Line Items] | |
Operating lease, term of contract | 36 months |
Bachem Americas, Inc [Member] | |
Commitments And Contingencies [Line Items] | |
Non-cancellable purchase obligation substance over period | 5 years |
Non-cancellable purchase commitments | $ 94,600,000 |
Other non-cancellable purchase commitments | $ 16,100,000 |
Equity Incentive Plans - Additi
Equity Incentive Plans - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||||||
Jan. 01, 2024 | Oct. 31, 2017 | Aug. 31, 2017 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Jan. 31, 2024 | Feb. 29, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Common stock reserved for future issuance | 19,180,000 | 16,793,000 | 13,576,000 | |||||
Stock options vesting period | 48 months | |||||||
Stock options vesting description | Options granted to employees on or after December 5, 2013 generally vest in installments of (i) 25% at the one-year anniversary and (ii) in either 36 equal monthly or 12 equal quarterly installments beginning in the thirteenth month after the initial vesting commencement date (as defined) subject to the employee’s continuous service with the Company. | |||||||
Aggregate number of stock options granted to purchase common stock | 838,000 | 1,300,000 | 2,700,000 | |||||
Weighted - Average Grant Date Fair Value Per Option, Granted | $ 34.26 | $ 23.62 | $ 30.72 | |||||
Aggregate intrinsic value of options exercised in period | $ 181,000 | $ 44,800 | $ 39,900 | |||||
Fair market value of options vested in period | 46,800 | $ 59,000 | 53,200 | |||||
Unrecognized compensation expense | $ 46,100 | |||||||
Estimated weighted-average period to recognize | 2 years 2 months 4 days | |||||||
Common stock, issued | 119,556,000 | 110,772,000 | ||||||
Common stock issued at average per share price | $ 20.11 | |||||||
Share-based compensation expense | $ 105,945 | $ 91,085 | $ 70,667 | |||||
Restricted Stock Units [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Unrecognized compensation expense | $ 155,500 | |||||||
Estimated weighted-average period to recognize | 2 years 7 months 6 days | |||||||
Aggregate intrinsic value of restricted grants vested | $ 55,300 | |||||||
Fair market value of restricted stock units vested | $ 42,200 | |||||||
Frist Anniversary [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Stock options vesting percentage | 25% | |||||||
Second Anniversary [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Stock options vesting percentage | 25% | |||||||
Third Anniversary [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Stock options vesting percentage | 25% | |||||||
Fourth Anniversary [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Stock options vesting percentage | 25% | |||||||
Subsequent Event [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Shares issuable increased during the period | 200,000 | |||||||
Equity Incentive Plan 2010 [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Common stock shares reserved for issuance | 6,188,466 | |||||||
Percentage of voting shares | 10% | |||||||
Options expire from issuance date | 10 years | |||||||
Equity Incentive Plan 2010 [Member] | Maximum [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage of estimated fair value per share of common stock on the date of grant | 100% | |||||||
Equity Incentive Plan 2010 [Member] | Minimum [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage price of each share of the fair value on date of grant | 110% | |||||||
2017 Stock Incentive Plan [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Common stock shares reserved for issuance | 1,359,587 | |||||||
Percentage of voting shares | 4% | |||||||
Number of shares available for future grant | 299,568 | 6,497,120 | ||||||
Shares issuable increased during the period | 4,219,409 | |||||||
Common Stock Issuance Description | equal to the lowest of 4,219,409 shares of common stock, 4.0% of the number of shares of common stock outstanding on the first day of the fiscal year and an amount determined by the board of directors. | |||||||
Common stock reserved for future issuance | 16,989,000 | 14,271,000 | 11,014,000 | |||||
2017 Stock Incentive Plan [Member] | Subsequent Event [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Number of shares available for future grant | 4,219,409 | |||||||
2020 Inducement Stock Incentive Plan [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Number of shares available for future grant | 354,466 | |||||||
Common stock reserved for future issuance | 1,638,000 | 1,857,000 | 1,761,000 | 750,000 | ||||
2020 Inducement Stock Incentive Plan [Member] | Subsequent Event [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Number of shares available for future grant | 1,950,000 | |||||||
2017 Employee Stock Purchase Plan [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Common stock shares reserved for issuance | 468,823 | |||||||
Percentage of voting shares | 1% | |||||||
2017 Employee Stock Purchase Plan [Member] | Maximum [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Common stock reserved for future issuance | 937,646 | |||||||
Employee Stock Purchase Plan [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Percentage price of each share of the fair value on date of grant | 85% | |||||||
Number of shares available for future grant | 552,881 | |||||||
Common stock reserved for future issuance | 0 | |||||||
Percentage of earnings withheld to purchase shares of common stock | 15% | |||||||
Common stock, issued | 112,064 | |||||||
Common stock issued at average per share price | $ 48 | |||||||
Cash received from issuance of purchase rights | $ 5,400 | |||||||
Share-based compensation expense | $ 2,100 |
Equity Incentive Plans - Summar
Equity Incentive Plans - Summary of Shares of Common Stock Reserved for Future Issuance (Detail) - shares | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Feb. 29, 2020 |
Class Of Stock [Line Items] | ||||
Common stock reserved for future issuance | 19,180,000 | 16,793,000 | 13,576,000 | |
2017 Stock Incentive Plan [Member] | ||||
Class Of Stock [Line Items] | ||||
Common stock reserved for future issuance | 16,989,000 | 14,271,000 | 11,014,000 | |
2017 Employee Stock Purchase Plan [Member] | ||||
Class Of Stock [Line Items] | ||||
Common stock reserved for future issuance | 553,000 | 665,000 | 801,000 | |
2020 Inducement Stock Incentive Plan [Member] | ||||
Class Of Stock [Line Items] | ||||
Common stock reserved for future issuance | 1,638,000 | 1,857,000 | 1,761,000 | 750,000 |
Equity Incentive Plans - Summ_2
Equity Incentive Plans - Summary of Share-based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Share-based compensation expense | $ 105,945 | $ 91,085 | $ 70,667 |
Research and Development Expense [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Share-based compensation expense | 45,644 | 42,052 | 30,586 |
General and Administrative [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Share-based compensation expense | $ 60,301 | $ 49,033 | $ 40,081 |
Equity Incentive Plans - Summ_3
Equity Incentive Plans - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward] | |||
Shares Outstanding, Beginning balance | 12,295 | ||
Shares, Granted | 838 | 1,300 | 2,700 |
Shares, Exercised | (3,950) | ||
Shares, Forfeited | (519) | ||
Shares Outstanding, Ending balance | 8,664 | 12,295 | |
Shares, Options exercisable | 6,712 | ||
Shares, Expected to vest | 1,952 | ||
Weighted -Average Exercise Price Per Share Outstanding, Beginning balance | $ 26.31 | ||
Weighted -Average Exercise Price Per Share, Granted | 52.48 | ||
Weighted -Average Exercise Price Per Share, Exercised | 20.11 | ||
Weighted -Average Exercise Price Per Share, Forfeited | 43.65 | ||
Weighted -Average Exercise Price Per Share, Outstanding, Ending balance | 30.65 | $ 26.31 | |
Weighted -Average Exercise Price Per Share, Options exercisable | 26.2 | ||
Weighted -Average Exercise Price Per Share, Expected to vest | $ 45.97 | ||
Weighted - Average Contractual Life Outstanding, Balance | 6 years 2 months 26 days | ||
Weighted - Average Contractual Life, Options exercisable | 5 years 4 months 20 days | ||
Weighted - Average Contractual Life, Expected to vest | 8 years 1 month 17 days | ||
Aggregate Intrinsic Value, Outstanding | $ 317,031 | ||
Aggregate Intrinsic Value, Options exercisable | $ 226,419 | ||
Aggregate Intrinsic Value, Expected to vest | $ 27,515 |
Equity Incentive Plans - Summ_4
Equity Incentive Plans - Summary of Assumption Used to Estimate the Grant Date Fair Value (Detail) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award, Fair Value Assumptions and Methodology [Abstract] | |||
Risk-free interest rate, minimum | 3.50% | 1.15% | 0.41% |
Risk-free interest rate, maximum | 4.01% | 3.37% | 1.34% |
Dividend yield | 0% | 0% | 0% |
Volatility, minimum | 68.40% | 68.30% | 71.70% |
Volatility, maximum | 71% | 70.40% | 74.40% |
Minimum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Fair Value Assumptions and Methodology [Abstract] | |||
Expected terms (years) | 3 years 9 months 21 days | 3 years 9 months 21 days | 5 years 3 months 21 days |
Maximum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Fair Value Assumptions and Methodology [Abstract] | |||
Expected terms (years) | 6 years 29 days | 6 years 29 days | 6 years 29 days |
Equity Incentive Plans - Schedu
Equity Incentive Plans - Schedule of Unvested Restricted Stock Units Activity (Detail) - Restricted Stock Units [Member] shares in Thousands | 12 Months Ended |
Dec. 31, 2023 $ / shares shares | |
Number of Stock Units | |
Unvested Beginning Balance | shares | 3,572 |
Granted | shares | 2,916 |
Vested | shares | (1,015) |
Forfeited | shares | (1,172) |
Unvested Ending Balance | shares | 4,301 |
Weighted Average Grant Date Fair Value Per Share | |
Unvested Balance at December 31, 2022 | $ / shares | $ 42.23 |
Granted | $ / shares | 52.19 |
Vested | $ / shares | 41.52 |
Forfeited | $ / shares | 48.43 |
Unvested Balance at December 31, 2023 | $ / shares | $ 47.48 |
Net Loss per Common Share - Sum
Net Loss per Common Share - Summary of Basic and Diluted Net Loss per Common Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Numerator: | |||
Net Income (Loss) | $ (528,628) | $ (652,172) | $ (746,354) |
Denominator: | |||
Weighted-average number of common shares used in net loss per common share, basic | 118,678 | 106,114 | 84,421 |
Weighted-average number of common shares used in net loss per common share, diluted | 118,678 | 106,114 | 84,421 |
Net loss per common share, basic | $ (4.45) | $ (6.15) | $ (8.84) |
Net loss per common share, diluted | $ (4.45) | $ (6.15) | $ (8.84) |
Net Loss per Common Share - S_2
Net Loss per Common Share - Summary of Shares Outstanding that were Excluded from Calculation of Diluted Net Loss Per Share (Detail) - shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from computation of earnings per share, amount | 15,344 | 18,246 | 18,651 |
Convertible Notes [Member] | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from computation of earnings per share, amount | 2,379 | 2,379 | 4,865 |
Common Stock Options [Member] | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from computation of earnings per share, amount | 8,664 | 12,295 | 12,562 |
Restricted Stock Units [Member] | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Antidilutive securities excluded from computation of earnings per share, amount | 4,301 | 3,572 | 1,224 |