Cover
Cover - shares | 9 Months Ended | |
Sep. 30, 2023 | Oct. 25, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2023 | |
Document Transition Report | false | |
Entity File Number | 001-33497 | |
Entity Registrant Name | Amicus Therapeutics, Inc. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 71-0869350 | |
Entity Address, Address Line One | 47 Hulfish Street | |
Entity Address, City or Town | Princeton | |
Entity Address, State or Province | NJ | |
Entity Address, Postal Zip Code | 08542 | |
City Area Code | (609) | |
Local Phone Number | 662-2000 | |
Title of 12(b) Security | Common Stock, par value $0.01 per share | |
Trading Symbol | FOLD | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 293,245,738 | |
Entity Central Index Key | 0001178879 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q3 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 263,320 | $ 148,813 |
Investments in marketable securities | 16,980 | 144,782 |
Accounts receivable | 73,331 | 66,196 |
Inventories | 56,936 | 23,816 |
Prepaid expenses and other current assets | 52,689 | 40,209 |
Total current assets | 463,256 | 423,816 |
Operating lease right-of-use assets, net | 29,511 | 29,534 |
Property and equipment, less accumulated depreciation of $25,018 and $22,281 at September 30, 2023 and December 31, 2022, respectively | 31,072 | 30,778 |
Intangible assets, less accumulated amortization of $1,682 and $0 at September 30, 2023 and December 31, 2022, respectively | 21,318 | 23,000 |
Goodwill | 197,797 | 197,797 |
Other non-current assets | 21,130 | 19,242 |
Total Assets | 764,084 | 724,167 |
Current liabilities: | ||
Accounts payable | 23,154 | 15,413 |
Accrued expenses and other current liabilities | 138,535 | 93,636 |
Contingent consideration payable | 0 | 21,417 |
Operating lease liabilities | 7,765 | 8,552 |
Total current liabilities | 169,454 | 139,018 |
Long-term debt | 394,071 | 391,990 |
Operating lease liabilities | 52,454 | 51,578 |
Deferred reimbursements | 5,906 | 4,656 |
Deferred income taxes | 0 | 4,939 |
Other non-current liabilities | 8,962 | 8,939 |
Total liabilities | 630,847 | 601,120 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Common stock, $0.01 par value, 500,000,000 shares authorized, 290,667,041 and 281,108,273 shares issued and outstanding at September 30, 2023 and December 31, 2022, respectively | 2,890 | 2,815 |
Additional paid-in capital | 2,787,275 | 2,664,744 |
Accumulated other comprehensive loss: | ||
Foreign currency translation adjustment | (6,573) | (11,989) |
Unrealized loss on available-for-sale securities | (195) | (116) |
Warrants | 71 | 83 |
Accumulated deficit | (2,650,231) | (2,532,490) |
Total stockholders’ equity | 133,237 | 123,047 |
Total Liabilities and Stockholders’ Equity | $ 764,084 | $ 724,167 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Accumulated depreciation of property and equipment | $ 25,018 | $ 22,281 |
Accumulated amortization of intangible assets | $ 1,682 | $ 0 |
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Common stock, shares authorized (in shares) | 500,000,000 | 500,000,000 |
Common stock, shares issued (in shares) | 290,667,041 | 281,108,273 |
Common stock, shares outstanding (in shares) | 290,667,041 | 281,108,273 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Revenue: | ||||
Net product sales | $ 103,501 | $ 81,691 | $ 284,274 | $ 241,137 |
Cost of goods sold | 9,946 | 13,436 | 26,002 | 29,215 |
Gross profit | 93,555 | 68,255 | 258,272 | 211,922 |
Operating expenses: | ||||
Research and development | 40,704 | 52,970 | 117,352 | 212,806 |
Selling, general, and administrative | 65,651 | 47,272 | 205,031 | 158,767 |
Changes in fair value of contingent consideration payable | 1,995 | 567 | 2,583 | (506) |
Loss on impairment of assets | 0 | 0 | 1,134 | 6,616 |
Depreciation and amortization | 2,228 | 1,286 | 5,691 | 4,031 |
Total operating expenses | 110,578 | 102,095 | 331,791 | 381,714 |
Loss from operations | (17,023) | (33,840) | (73,519) | (169,792) |
Other income (expense): | ||||
Interest income | 1,471 | 563 | 5,407 | 1,052 |
Interest expense | (12,986) | (9,620) | (37,322) | (26,024) |
Other income (expense) | 3,833 | 13,634 | (13,007) | 22,804 |
Loss before income tax | (24,705) | (29,263) | (118,441) | (171,960) |
Income tax benefit (expense) | 3,128 | (4,023) | 700 | (8,743) |
Net loss attributable to common stockholders | $ (21,577) | $ (33,286) | $ (117,741) | $ (180,703) |
Net loss attributable to common stockholders per common share - basic (in dollars per share) | $ (0.07) | $ (0.12) | $ (0.40) | $ (0.63) |
Net loss attributable to common stockholders per common share — diluted (in dollars per share) | $ (0.07) | $ (0.12) | $ (0.40) | $ (0.63) |
Weighted average common shares outstanding - basic (in shares) | 295,759,435 | 289,223,709 | 293,314,167 | 288,841,092 |
Weighted average common shares outstanding - diluted (in shares) | 295,759,435 | 289,223,709 | 293,314,167 | 288,841,092 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (21,577) | $ (33,286) | $ (117,741) | $ (180,703) |
Other comprehensive (loss) gain: | ||||
Foreign currency translation adjustment (loss) gain | (10,910) | (22,121) | 5,416 | (43,975) |
Unrealized (loss) gain on available-for-sale securities | (18) | 283 | (79) | (84) |
Other comprehensive (loss) gain | (10,928) | (21,838) | 5,337 | (44,059) |
Comprehensive loss | $ (32,505) | $ (55,124) | $ (112,404) | $ (224,762) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands | Total | ATM | Common Stock | Common Stock ATM | Additional Paid-In Capital | Additional Paid-In Capital ATM | Warrants | Other Comprehensive Loss | Accumulated Deficit |
Beginning balance (in shares) at Dec. 31, 2021 | 278,912,800 | ||||||||
Beginning balance at Dec. 31, 2021 | $ 307,369 | $ 2,808 | $ 2,595,419 | $ 83 | $ 4,981 | $ (2,295,922) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock options exercised, net (in shares) | 506,823 | ||||||||
Stock options exercised, net | 3,191 | $ 5 | 3,186 | ||||||
Vesting of restricted stock units, net of taxes (in shares) | 1,467,513 | ||||||||
Vesting of restricted stock units, net of taxes | (11,119) | (11,119) | |||||||
Stock-based compensation | 57,886 | 57,886 | |||||||
Unrealized (loss) gain on available-for-sale securities | (84) | (84) | |||||||
Foreign currency translation adjustment | (43,975) | (43,975) | |||||||
Net loss | (180,703) | (180,703) | |||||||
Ending balance (in shares) at Sep. 30, 2022 | 280,887,136 | ||||||||
Ending balance at Sep. 30, 2022 | 132,565 | $ 2,813 | 2,645,372 | 83 | (39,078) | (2,476,625) | |||
Beginning balance (in shares) at Jun. 30, 2022 | 280,456,667 | ||||||||
Beginning balance at Jun. 30, 2022 | 173,425 | $ 2,811 | 2,631,110 | 83 | (17,240) | (2,443,339) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock options exercised, net (in shares) | 172,118 | ||||||||
Stock options exercised, net | 1,333 | $ 2 | 1,331 | ||||||
Vesting of restricted stock units, net of taxes (in shares) | 258,351 | ||||||||
Vesting of restricted stock units, net of taxes | (1,841) | (1,841) | |||||||
Stock-based compensation | 14,772 | 14,772 | |||||||
Unrealized (loss) gain on available-for-sale securities | 283 | 283 | |||||||
Foreign currency translation adjustment | (22,121) | (22,121) | |||||||
Net loss | (33,286) | (33,286) | |||||||
Ending balance (in shares) at Sep. 30, 2022 | 280,887,136 | ||||||||
Ending balance at Sep. 30, 2022 | $ 132,565 | $ 2,813 | 2,645,372 | 83 | (39,078) | (2,476,625) | |||
Beginning balance (in shares) at Dec. 31, 2022 | 281,108,273 | 281,108,273 | |||||||
Beginning balance at Dec. 31, 2022 | $ 123,047 | $ 2,815 | 2,664,744 | 83 | (12,105) | (2,532,490) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock options exercised, net (in shares) | 1,032,000 | 1,025,684 | |||||||
Stock options exercised, net | $ 7,847 | $ 11 | 7,836 | ||||||
Vesting of restricted stock units, net of taxes (in shares) | 2,068,048 | ||||||||
Vesting of restricted stock units, net of taxes | (16,355) | (16,355) | |||||||
Stock-based compensation | 67,982 | 67,982 | |||||||
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares) | 5,244,936 | ||||||||
Issuance of shares in connection with at-the-market offering, net of issuance costs | $ 63,108 | $ 52 | $ 63,056 | ||||||
Warrants exercised (in shares) | 1,220,100 | ||||||||
Warrants exercised | 12 | $ 12 | 12 | (12) | |||||
Unrealized (loss) gain on available-for-sale securities | (79) | (79) | |||||||
Foreign currency translation adjustment | 5,416 | 5,416 | |||||||
Net loss | $ (117,741) | (117,741) | |||||||
Ending balance (in shares) at Sep. 30, 2023 | 290,667,041 | 290,667,041 | |||||||
Ending balance at Sep. 30, 2023 | $ 133,237 | $ 2,890 | 2,787,275 | 71 | (6,768) | (2,650,231) | |||
Beginning balance (in shares) at Jun. 30, 2023 | 286,992,923 | ||||||||
Beginning balance at Jun. 30, 2023 | 111,581 | $ 2,856 | 2,733,148 | 71 | 4,160 | (2,628,654) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock options exercised, net (in shares) | 372,467 | ||||||||
Stock options exercised, net | 3,442 | $ 4 | 3,438 | ||||||
Vesting of restricted stock units, net of taxes (in shares) | 299,297 | ||||||||
Vesting of restricted stock units, net of taxes | (2,347) | (2,347) | |||||||
Stock-based compensation | 16,511 | 16,511 | |||||||
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares) | 3,002,354 | ||||||||
Issuance of shares in connection with at-the-market offering, net of issuance costs | $ 36,555 | $ 30 | $ 36,525 | ||||||
Unrealized (loss) gain on available-for-sale securities | (18) | (18) | |||||||
Foreign currency translation adjustment | (10,910) | (10,910) | |||||||
Net loss | $ (21,577) | (21,577) | |||||||
Ending balance (in shares) at Sep. 30, 2023 | 290,667,041 | 290,667,041 | |||||||
Ending balance at Sep. 30, 2023 | $ 133,237 | $ 2,890 | $ 2,787,275 | $ 71 | $ (6,768) | $ (2,650,231) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Operating activities | ||
Net loss | $ (117,741) | $ (180,703) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Amortization of debt discount and deferred financing | 2,080 | 1,963 |
Depreciation and amortization | 5,691 | 4,031 |
Stock-based compensation | 67,982 | 57,886 |
Non-cash changes in the fair value of contingent consideration payable | 2,583 | (506) |
Foreign currency remeasurement loss | 18,121 | 2,828 |
Deferred taxes | (4,939) | 0 |
Asset impairment charges and other asset write-offs | 2,360 | 17,271 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (8,614) | (7,426) |
Inventories | (42,233) | 4,913 |
Prepaid expenses and other current assets | (26,010) | (5,583) |
Accounts payable, accrued expenses, and other current liabilities | 41,101 | 25,465 |
Other non-current assets and liabilities | (4,993) | (5,942) |
Payment of contingent consideration | (7,937) | 0 |
Net cash used in operating activities | (72,549) | (85,803) |
Investing activities | ||
Sale and redemption of marketable securities | 180,828 | 259,920 |
Purchases of marketable securities | (53,098) | (99,811) |
Capital expenditures | (5,709) | (1,089) |
Net cash provided by investing activities | 122,021 | 159,020 |
Financing activities | ||
Payment of finance leases | (82) | (92) |
Withholding taxes paid on vested restricted stock units | (16,355) | (11,119) |
Proceeds from stock options exercised, net | 7,847 | 3,191 |
Proceeds from warrants exercised, net | 12 | 0 |
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs | 63,108 | 0 |
Payment of contingent consideration | (1,063) | 0 |
Net cash provided by (used in) financing activities | 53,467 | (8,020) |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | 10,218 | (33,120) |
Net increase in cash, cash equivalents, and restricted cash at the end of the period | 113,157 | 32,077 |
Cash, cash equivalents, and restricted cash at the beginning of period | 153,115 | 249,456 |
Cash, cash equivalents, and restricted cash at the end of period | 266,272 | 281,533 |
Supplemental disclosures of cash flow information | ||
Cash paid during the period for interest | 35,448 | 24,058 |
Cash paid for taxes | 6,473 | 935 |
Capital expenditures unpaid at the end of period | $ 877 | $ 53 |
Description of Business
Description of Business | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Description of Business | Description of Business Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company has developed and commercialized the first oral monotherapy for Fabry disease that has achieved widespread global approval and the first two-component therapy for late-onset Pompe disease that has been approved in the European Union (“E.U.”), the United Kingdom (“U.K.”), and in the United States (“U.S.”). The Company is committed to discovering and developing next generation therapies in Fabry and Pompe diseases. The cornerstone of the Company's portfolio is Galafold ® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold ® in the U.S., E.U., U.K., and Japan, with multiple additional approvals granted and applications pending in several geographies around the world. Pombiliti ™ + Opfolda ™ (also referred to as AT-GAA, ATB200/AT2221, or cipaglucosidase alfa-atga/miglustat), is a novel, two-component treatment for late-onset Pompe disease that was approved by the European Commission ("EC") in June 2023, the U.K. Medicines and Healthcare products Regulatory Agency ("MHRA") in August 2023, and the U.S. Food and Drug Administration ("FDA") in September 2023. The Company began launch activities for Pombiliti ™ + Opfolda ™ in these markets and have commenced the reimbursement processes with healthcare authorities in additional European countries. The Company had an accumulated deficit of $2.7 billion as of September 30, 2023 and anticipates incurring losses through the fiscal year ending December 31, 2023. The Company has historically funded its operations through stock offerings, Galafold ® revenues, debt issuances, collaborations, and other financing arrangements. In October 2023, the Company entered into a $400 million loan agreement (the “Senior Secured Term Loan due 2029”) with Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C. (collectively, “Blackstone”) with an interest rate equal to 3-month Term SOFR, subject to a 2.5% floor, plus a Term SOFR adjustment of 0.26161% and a margin of 6.25% that requires interest-only payments until early-2027 and matures in six years in 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and estimated expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029. Simultaneously, the Company also entered into a securities purchase agreement with funds managed by Blackstone, for the private placement of an aggregate of 2,467,104 shares of the Company’s common stock, at a purchase price of $12.16 per share. Proceeds from the private placement, net of offering costs, were $29.8 million. The Company used proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, accrued interest and prepayment premium. The remaining proceeds will be used to fund ongoing operations. Based on its current operating model, the Company believes that the current cash position, which includes expected revenues, is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company's future capital requirements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Basis of Presentation The Company has prepared the accompanying unaudited Consolidated Financial Statements in accordance with the U.S. generally accepted accounting principles ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information. The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2022. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2022. Consolidation The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation. Foreign Currency Transactions The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. 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, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive loss in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs. Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in other non-current assets on the Company's Consolidated Balance Sheets. Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities. The Company is subject to credit risk from its accounts receivable primarily related to its product sales of Galafold ® . The Company's accounts receivable at September 30, 2023 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of September 30, 2023, the Company's allowance for doubtful accounts was $0.1 million. Revenue Recognition The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies, with the ultimate payor often a government authority. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes. The following table summarizes the Company's net product sales disaggregated by product: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Galafold ® $ 100,733 $ 81,631 $ 281,177 $ 241,056 Pombiliti ™ + Opfolda ™ 2,768 60 3,097 81 Total net product sales $ 103,501 $ 81,691 $ 284,274 $ 241,137 The following table summarizes the Company's net product sales disaggregated by geographic area: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 U.S. $ 37,801 $ 30,222 $ 103,760 $ 81,940 Ex-U.S. 65,700 51,469 180,514 159,197 Total net product sales $ 103,501 $ 81,691 $ 284,274 $ 241,137 Inventories and Cost of Goods Sold Until regulatory approval of Pombiliti ™ + Opfolda ™ , the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Pombiliti ™ + Opfolda ™ . Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations. Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of inventory available for sale was expensed as research and development costs prior to regulatory approval and as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin. Intangible Assets and Goodwill The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is conducted. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference. Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made. No indicators of impairment were noted during the nine months ended September 30, 2023. Recent Accounting Developments The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures. |
Intangible Assets
Intangible Assets | 9 Months Ended |
Sep. 30, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible Assets | Intangible Assets As of September 30, 2023, the Company's intangible assets consisted of lead enzyme replacement therapy assets acquired with the Callidus Biopharma, Inc. acquisition in 2013, previously accounted for as in-process research and development. In March 2023, as a result of the EC's approval of Pombiliti ™ , the Company began amortizing the assets over the initial regulatory exclusivity period of 7 years. The Company completed an impairment assessment before changing the classification to definite-lived intangible asset noting no impairment. Amortization expense for the three and nine months ended September 30, 2023 was $0.8 million and $1.7 million, respectively. Total estimated amortization for the finite-lived intangible assets is estimated to be $2.5 million for the year ending December 31, 2023 and $3.3 million for the next four years thereafter. |
Cash, Cash Equivalents, Marketa
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash | 9 Months Ended |
Sep. 30, 2023 | |
Cash, Cash Equivalents, and Short-Term Investments [Abstract] | |
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash | Cash, Cash Equivalents, Marketable Securities, and Restricted Cash As of September 30, 2023, the Company held $263.3 million in cash and cash equivalents and $17.0 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive (loss) gain in the Company's Consolidated Statements of Comprehensive Loss. If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in earnings as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss, the Company would recognize an allowance and the corresponding credit loss would be included in earnings. The Company regularly invests excess operating cash in deposits with major financial institutions and money market funds, as well as fixed income investments which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current. Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following: As of September 30, 2023 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 263,320 $ — $ — $ 263,320 Commercial paper 16,826 3 — 16,829 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 280,297 $ 3 $ — $ 280,300 Included in cash and cash equivalents $ 263,320 $ — $ — $ 263,320 Included in marketable securities 16,977 3 — 16,980 Total cash, cash equivalents, and marketable securities $ 280,297 $ 3 $ — $ 280,300 As of December 31, 2022 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 148,813 $ — $ — $ 148,813 Commercial paper 144,299 82 — 144,381 Money market 350 — — 350 Certificate of deposit 51 — — 51 $ 293,513 $ 82 $ — $ 293,595 Included in cash and cash equivalents $ 148,813 $ — $ — $ 148,813 Included in marketable securities 144,700 82 — 144,782 Total cash, cash equivalents, and marketable securities $ 293,513 $ 82 $ — $ 293,595 For both the nine months ended September 30, 2023 and the fiscal year ended December 31, 2022, there were no realized gains or losses. The cost of securities sold is based on the specific identification method. Unrealized loss positions in the marketable securities as of September 30, 2023 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized in other comprehensive (loss) gain. The Company had no securities in an unrealized loss position as of both September 30, 2023 and December 31, 2022. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company's Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Company's Consolidated Statements of Cash Flows. As of September 30, (in thousands) 2023 2022 Cash and cash equivalents $ 263,320 $ 277,592 Restricted cash 2,952 3,941 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 266,272 $ 281,533 |
Inventories
Inventories | 9 Months Ended |
Sep. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Inventories | Inventories Inventories as of September 30, 2023 and December 31, 2022 consisted of the following: (in thousands) September 30, 2023 December 31, 2022 Raw materials $ 36,062 $ 10,054 Work-in-process 14,316 9,615 Finished goods 6,558 4,147 Total inventories $ 56,936 $ 23,816 The Company's reserve for inventory was $0.3 million and $0.4 million as of September 30, 2023 and December 31, 2022, respectively. |
Debt
Debt | 9 Months Ended |
Sep. 30, 2023 | |
Debt Disclosure [Abstract] | |
Debt | Debt The Company's debt consists of the following: (in thousands) September 30, 2023 December 31, 2022 Senior Secured Term Loan due 2026: Principal $ 400,000 $ 400,000 Less: debt discount (1) (3,384) (4,571) Less: deferred financing (1) (2,545) (3,439) Net carrying value of Long-term debt (2) $ 394,071 $ 391,990 ______________________________ (1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan due 2026 using the effective interest rate method. (2) The Company classifies the current portion of long-term debt as non-current liabilities on the Consolidated Balance Sheets when it has the intent and ability to refinance the obligations on a long-term basis, in accordance with ASC 470-50 “Debt.” Accordingly, as of September 30, 2023, the debt was recorded as a long-term liability in the Company’s Consolidated Balance Sheet. In October 2023, the Company entered into a $400 million loan agreement (the “Senior Secured Term Loan due 2029”) with Blackstone with an interest rate equal to 3-month Term SOFR, subject to a 2.5% floor, plus a Term SOFR adjustment of 0.26161% and a margin of 6.25% that requires interest-only payments until early-2027 and matures in six years in 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and estimated expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029. The Company used proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, accrued interest and prepayment premium. In connection with the prepayment, the Company expects to record a loss from early extinguishment of debt of approximately $13.9 million in the fourth quarter of 2023, primarily related to the prepayment premium and write-off of unamortized debt discount and deferred financing costs. Interest Expense The following table sets forth interest expense recognized related to the Company's debt for the three and nine months ended September 30, 2023 and 2022, respectively: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Contractual interest expense $ 12,270 $ 8,945 $ 35,289 $ 24,034 Amortization of debt discount $ 411 $ 382 $ 1,187 $ 1,121 Amortization of deferred financing $ 310 $ 286 $ 893 $ 842 |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2023 | |
Equity [Abstract] | |
Stockholders' Equity | Stockholder's Equity During the three and nine months ended September 30, 2023, the Company issued and sold an aggregate of 3,002,354 and 5,244,936 shares, respectively, through its at-the-market equity program ("ATM program") at weighted-average public offering prices of $12.68 and $12.50 per share, resulting in net proceeds of $36.6 million and $63.1 million, respectively. As of September 30, 2023, an aggregate of $184.4 million worth of shares remain available to be issued and sold under the ATM program. In October 2023, in connection with the Senior Secured Term Loan due 2029, the Company entered into a securities purchase agreement with funds managed by Blackstone, for the private placement of an aggregate of 2,467,104 shares of the Company’s common stock, at a purchase price of $12.16 per share. Proceeds from the private placement, net of offering costs, were $29.8 million. |
Stock-Based Compensation
Stock-Based Compensation | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation | Stock-Based Compensation The Company's Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option. Stock Option Grants The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Expected stock price volatility 58.4 % 61.0 % 59.2 % 62.2 % Risk free interest rate 4.3 % 3.1 % 3.9 % 1.7 % Expected life of options (years) 5.5 5.3 5.5 5.3 Expected annual dividend per share $ — $ — $ — $ — A summary of the Company's stock options for the nine months ended September 30, 2023 were as follows: Number of Weighted Average Exercise Weighted Average Remaining Aggregate (in thousands) (in millions) Options outstanding, December 31, 2022 19,064 $ 11.31 Granted 5,522 $ 12.03 Exercised (1,032) $ 7.61 Forfeited (248) $ 11.51 Expired (50) $ 14.39 Options outstanding, September 30, 2023 23,256 $ 11.64 6.7 $ 32.1 Vested and unvested expected to vest, September 30, 2023 21,389 $ 11.58 6.5 $ 31.1 Exercisable at September 30, 2023 13,246 $ 11.12 5.1 $ 27.9 As of September 30, 2023, the total unrecognized compensation cost related to non-vested stock options granted was $39.8 million and is expected to be recognized over a weighted average period of three years. Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs") RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2023 is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Non-vested units as of December 31, 2022 9,717 $ 13.07 Granted 4,514 $ 13.08 Vested (3,252) $ 12.25 Forfeited (648) $ 10.07 Non-vested units as of September 30, 2023 10,331 $ 13.56 2.2 $ 125.6 As of September 30, 2023, there was $63.1 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years. Compensation Expense Related to Equity Awards The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Research and development expense $ 4,380 $ 5,428 $ 16,987 $ 19,172 Selling, general, and administrative expense 12,131 9,344 50,995 38,714 Total equity compensation expense $ 16,511 $ 14,772 $ 67,982 $ 57,886 |
Assets and Liabilities Measured
Assets and Liabilities Measured at Fair Value | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured at Fair Value | Assets and Liabilities Measured at Fair Value The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows: Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly. Level 3 — Inputs that are unobservable for the asset or liability. A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2023 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 16,829 $ 16,829 Money market 6,818 6,818 $ 23,647 $ 23,647 (in thousands) Level 2 Total Liabilities: Deferred compensation plan liability $ 6,718 $ 6,718 $ 6,718 $ 6,718 A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2022 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 144,381 $ 144,381 Money market 5,808 5,808 $ 150,189 $ 150,189 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 21,417 $ 21,417 Deferred compensation plan liability 5,458 — 5,458 $ 5,458 $ 21,417 $ 26,875 The Company's Senior Secured Term Loan due 2026 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. The carrying value of the Senior Secured Term Loan due 2026 approximates the fair value. Deferred compensation plan liability is recorded as a component of other non-current liabilities on the Company's Consolidated Balance Sheets. The Company did not have any Level 3 assets as of September 30, 2023 or December 31, 2022. Liabilities measured at fair value using Level 3 inputs consisted of contingent consideration. Cash, Money Market Funds, and Marketable Securities The Company classifies its cash within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets and the money market funds within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. Contingent Consideration Payable The contingent consideration payable resulted from the acquisition of Callidus Biopharma, Inc. ("Callidus") in November 2013. The Company reached regulatory milestones of $9.0 million in March 2023 and $15.0 million in September 2023 associated with the approval of Pombiliti ™ by the EC and FDA, respectively. The $9.0 million milestone payment was paid in the second quarter of 2023 and the $15.0 million milestone payment, which is payable in cash, is recorded as a component of accounts payable on the Company's Consolidated Balance Sheets as of September 30, 2023. The following table shows the change in the balance of contingent consideration payable for the three and nine months ended September 30, 2023 and 2022, respectively: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Balance, beginning of the period $ 13,005 $ 19,266 $ 21,417 $ 20,339 Changes in fair value during the period, included in the Consolidated Statements of Operations 1,995 567 2,583 (506) Milestone paid or payable in cash (15,000) — (24,000) — Balance, end of the period $ — $ 19,833 $ — $ 19,833 |
Basic and Diluted Net Loss per
Basic and Diluted Net Loss per Common Share | 9 Months Ended |
Sep. 30, 2023 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss per Common Share | Basic and Diluted Net Loss per Common Share The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share: Three Months Ended September 30, Nine Months Ended September 30, (in thousands, except per share amounts) 2023 2022 2023 2022 Numerator: Net loss attributable to common stockholders $ (21,577) $ (33,286) $ (117,741) $ (180,703) Denominator: Weighted average common shares outstanding — basic and diluted 295,759,435 289,223,709 293,314,167 288,841,092 Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and unvested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01. The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method: As of September 30, (in thousands) 2023 2022 Options to purchase common stock 23,256 19,236 Unvested restricted stock units 10,331 9,839 Total number of potentially issuable shares 33,587 29,075 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Pay vs Performance Disclosure | ||||
Net loss | $ (21,577) | $ (33,286) | $ (117,741) | $ (180,703) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended | 9 Months Ended |
Sep. 30, 2023 shares | Sep. 30, 2023 shares | |
Trading Arrangements, by Individual | ||
Material Terms of Trading Arrangement | The following table describes, for the quarterly period covered by this report, each director and officer (as defined in Rule 16a-1(f) under the Exchange Act who has adopted, modified, or terminated a trading plan intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act (each plan, a “Rule 10b5-1 Trading Plan”). Each Rule 10b5-1 Trading Plan described below was adopted during an open insider trading window and in accordance with the Company’s policies regarding both insider trading and transactions relating to Company securities. Name Action Taken Rule 10b5-1 Trading Plan Provides for Purchase/Sale Duration of the Trading Plan (1) Aggregate Number of Securities Bradley Campbell (President and Chief Executive Officer) Adoption (August 23, 2023) Sale December 31, 2024 Indeterminable (2) Jeffrey Castelli (Chief Development Officer) Adoption (August 25, 2023) Sale May 15, 2024 52,264 David Clark (Chief People Officer) Adoption (August 25, 2023) Sale August 15, 2024 Indeterminable (3) John F. Crowley (Executive Chairman) Adoption (September 6, 2023) Sale April 15, 2024 Indeterminable (3) Ellen Rosenberg (Chief Legal Officer and Corporate Secretary) Adoption (September 8, 2023) Sale December 7, 2024 95,000 ____________________________ (1) The dates in this column represent the scheduled expiration date of each director or officer’s Rule 10b5-1 Trading Plan. Each Rule 10b5-1 Trading Plan may terminate earlier than the date provided should all transactions contemplated thereunder occur prior to such date. (2) Mr. Campbell’s Rule 10b5-1 Trading Plan provides for the (i) exercise of up to 190,000 stock options and the sale of up to 190,000 underlying shares of common stock and (ii) sale of an indeterminable number of shares of common stock. The shares of common stock in clause (ii) will be obtained from the settlement of Mr. Campbell’s 2021 performance restricted stock unit (“PRSU”) awards. The number of shares of common stock obtained and available for sale will be subject to the (a) level of achievement of each performance goal contained within the 2021 PRSU awards and (b) shares of common stock withheld to satisfy applicable tax withholding obligations. (3) The shares of common stock to be sold under this Rule 10b5-1 Trading Plan will be obtained from the settlement of the 2021 PRSU awards and the respective vesting of the 2020, 2021, 2022 and 2023 annual restricted stock unit awards. The number of shares of common stock obtained and available for sale will be subject to the (i) level of achievement of each performance goal contained within the 2021 PRSU awards and (ii) shares of common stock withheld to satisfy applicable tax withholding obligations. | |
Non-Rule 10b5-1 Arrangement Adopted | false | |
Rule 10b5-1 Arrangement Terminated | false | |
Non-Rule 10b5-1 Arrangement Terminated | false | |
Bradley Campbell [Member] | ||
Trading Arrangements, by Individual | ||
Name | Bradley Campbell | |
Title | President and Chief Executive Officer | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | August 23, 2023 | |
Arrangement Duration | 135 days | |
Jeffrey Castelli [Member] | ||
Trading Arrangements, by Individual | ||
Name | Jeffrey Castelli | |
Title | Chief Development Officer | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | August 25, 2023 | |
Arrangement Duration | 264 days | |
Aggregate Available | 52,264 | 52,264 |
David Clark [Member] | ||
Trading Arrangements, by Individual | ||
Name | David Clark | |
Title | Chief People Officer | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | August 25, 2023 | |
Arrangement Duration | 356 days | |
John F. Crowley [Member] | ||
Trading Arrangements, by Individual | ||
Name | John F. Crowley | |
Title | Executive Chairman | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | September 6, 2023 | |
Arrangement Duration | 222 days | |
Ellen Rosenberg [Member] | ||
Trading Arrangements, by Individual | ||
Name | Ellen Rosenberg | |
Title | Chief Legal Officer and Corporate Secretary | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | September 8, 2023 | |
Arrangement Duration | 456 days | |
Aggregate Available | 95,000 | 95,000 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The Company has prepared the accompanying unaudited Consolidated Financial Statements in accordance with the U.S. generally accepted accounting principles ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information. |
Consolidation | Consolidation The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation. |
Foreign Currency Transactions | Foreign Currency Transactions The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. |
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, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. |
Cash, Cash Equivalents, Marketable Securities and Restricted Cash | Cash, Cash Equivalents, Marketable Securities, and Restricted Cash The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive loss in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs. |
Concentration of Credit Risk | Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities. The Company is subject to credit risk from its accounts receivable primarily related to its product sales of Galafold ® |
Revenue Recognition | Revenue Recognition The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies, with the ultimate payor often a government authority. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes. |
Inventories and Cost of Goods Sold | Inventories and Cost of Goods Sold Until regulatory approval of Pombiliti ™ + Opfolda ™ , the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Pombiliti ™ + Opfolda ™ . |
Intangible Assets and Goodwill | Intangible Assets and Goodwill The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is conducted. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference. Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made. No indicators of impairment were noted during the nine months ended September 30, 2023. |
Recent Accounting Developments | Recent Accounting Developments The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Schedule of Revenue Disaggregated by Geographical Area | The following table summarizes the Company's net product sales disaggregated by product: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Galafold ® $ 100,733 $ 81,631 $ 281,177 $ 241,056 Pombiliti ™ + Opfolda ™ 2,768 60 3,097 81 Total net product sales $ 103,501 $ 81,691 $ 284,274 $ 241,137 The following table summarizes the Company's net product sales disaggregated by geographic area: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 U.S. $ 37,801 $ 30,222 $ 103,760 $ 81,940 Ex-U.S. 65,700 51,469 180,514 159,197 Total net product sales $ 103,501 $ 81,691 $ 284,274 $ 241,137 |
Cash, Cash Equivalents, Marke_2
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Cash, Cash Equivalents, and Short-Term Investments [Abstract] | |
Schedule of Cash, Cash Equivalents and Marketable Securities | Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following: As of September 30, 2023 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 263,320 $ — $ — $ 263,320 Commercial paper 16,826 3 — 16,829 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 280,297 $ 3 $ — $ 280,300 Included in cash and cash equivalents $ 263,320 $ — $ — $ 263,320 Included in marketable securities 16,977 3 — 16,980 Total cash, cash equivalents, and marketable securities $ 280,297 $ 3 $ — $ 280,300 As of December 31, 2022 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 148,813 $ — $ — $ 148,813 Commercial paper 144,299 82 — 144,381 Money market 350 — — 350 Certificate of deposit 51 — — 51 $ 293,513 $ 82 $ — $ 293,595 Included in cash and cash equivalents $ 148,813 $ — $ — $ 148,813 Included in marketable securities 144,700 82 — 144,782 Total cash, cash equivalents, and marketable securities $ 293,513 $ 82 $ — $ 293,595 |
Schedule of Cash, Cash Equivalents, and Restricted Cash | The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company's Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Company's Consolidated Statements of Cash Flows. As of September 30, (in thousands) 2023 2022 Cash and cash equivalents $ 263,320 $ 277,592 Restricted cash 2,952 3,941 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 266,272 $ 281,533 |
Inventories (Tables)
Inventories (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventories for the Period | Inventories as of September 30, 2023 and December 31, 2022 consisted of the following: (in thousands) September 30, 2023 December 31, 2022 Raw materials $ 36,062 $ 10,054 Work-in-process 14,316 9,615 Finished goods 6,558 4,147 Total inventories $ 56,936 $ 23,816 |
Debt (Tables)
Debt (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Debt Disclosure [Abstract] | |
Schedule of Liability Components of Long-Term Debt | The Company's debt consists of the following: (in thousands) September 30, 2023 December 31, 2022 Senior Secured Term Loan due 2026: Principal $ 400,000 $ 400,000 Less: debt discount (1) (3,384) (4,571) Less: deferred financing (1) (2,545) (3,439) Net carrying value of Long-term debt (2) $ 394,071 $ 391,990 ______________________________ (1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan due 2026 using the effective interest rate method. (2) The Company classifies the current portion of long-term debt as non-current liabilities on the Consolidated Balance Sheets when it has the intent and ability to refinance the obligations on a long-term basis, in accordance with ASC 470-50 “Debt.” Accordingly, as of September 30, 2023, the debt was recorded as a long-term liability in the Company’s Consolidated Balance Sheet. In October 2023, the Company entered into a $400 million loan agreement (the “Senior Secured Term Loan due 2029”) with Blackstone with an interest rate equal to 3-month Term SOFR, subject to a 2.5% floor, plus a Term SOFR adjustment of 0.26161% and a margin of 6.25% that requires interest-only payments until early-2027 and matures in six years in 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and estimated expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029. The Company used proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, accrued interest and prepayment premium. In connection with the prepayment, the Company expects to record a loss from early extinguishment of debt of approximately $13.9 million in the fourth quarter of 2023, primarily related to the prepayment premium and write-off of unamortized debt discount and deferred financing costs. |
Schedule of Components of Total Interest Expense | The following table sets forth interest expense recognized related to the Company's debt for the three and nine months ended September 30, 2023 and 2022, respectively: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Contractual interest expense $ 12,270 $ 8,945 $ 35,289 $ 24,034 Amortization of debt discount $ 411 $ 382 $ 1,187 $ 1,121 Amortization of deferred financing $ 310 $ 286 $ 893 $ 842 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Schedule of Fair Value of Options | The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Expected stock price volatility 58.4 % 61.0 % 59.2 % 62.2 % Risk free interest rate 4.3 % 3.1 % 3.9 % 1.7 % Expected life of options (years) 5.5 5.3 5.5 5.3 Expected annual dividend per share $ — $ — $ — $ — |
Schedule of Stock Options Activity | A summary of the Company's stock options for the nine months ended September 30, 2023 were as follows: Number of Weighted Average Exercise Weighted Average Remaining Aggregate (in thousands) (in millions) Options outstanding, December 31, 2022 19,064 $ 11.31 Granted 5,522 $ 12.03 Exercised (1,032) $ 7.61 Forfeited (248) $ 11.51 Expired (50) $ 14.39 Options outstanding, September 30, 2023 23,256 $ 11.64 6.7 $ 32.1 Vested and unvested expected to vest, September 30, 2023 21,389 $ 11.58 6.5 $ 31.1 Exercisable at September 30, 2023 13,246 $ 11.12 5.1 $ 27.9 |
Schedule of Non-Vested RSU Activity under the Plan | A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2023 is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Non-vested units as of December 31, 2022 9,717 $ 13.07 Granted 4,514 $ 13.08 Vested (3,252) $ 12.25 Forfeited (648) $ 10.07 Non-vested units as of September 30, 2023 10,331 $ 13.56 2.2 $ 125.6 |
Schedule of Equity Compensation Expenses | The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Research and development expense $ 4,380 $ 5,428 $ 16,987 $ 19,172 Selling, general, and administrative expense 12,131 9,344 50,995 38,714 Total equity compensation expense $ 16,511 $ 14,772 $ 67,982 $ 57,886 |
Assets and Liabilities Measur_2
Assets and Liabilities Measured at Fair Value (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Summary of Assets and Liabilities Subject to Fair Value Measurements | A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2023 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 16,829 $ 16,829 Money market 6,818 6,818 $ 23,647 $ 23,647 (in thousands) Level 2 Total Liabilities: Deferred compensation plan liability $ 6,718 $ 6,718 $ 6,718 $ 6,718 A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2022 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 144,381 $ 144,381 Money market 5,808 5,808 $ 150,189 $ 150,189 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 21,417 $ 21,417 Deferred compensation plan liability 5,458 — 5,458 $ 5,458 $ 21,417 $ 26,875 |
Schedule of Changes in Continent Consideration Payable | The following table shows the change in the balance of contingent consideration payable for the three and nine months ended September 30, 2023 and 2022, respectively: Three Months Ended September 30, Nine Months Ended September 30, (in thousands) 2023 2022 2023 2022 Balance, beginning of the period $ 13,005 $ 19,266 $ 21,417 $ 20,339 Changes in fair value during the period, included in the Consolidated Statements of Operations 1,995 567 2,583 (506) Milestone paid or payable in cash (15,000) — (24,000) — Balance, end of the period $ — $ 19,833 $ — $ 19,833 |
Basic and Diluted Net Loss pe_2
Basic and Diluted Net Loss per Common Share (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Earnings Per Share [Abstract] | |
Schedule of Reconciliation of the Numerator and Denominator Used in Computing Basic and Diluted Net Loss per Common Share | The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share: Three Months Ended September 30, Nine Months Ended September 30, (in thousands, except per share amounts) 2023 2022 2023 2022 Numerator: Net loss attributable to common stockholders $ (21,577) $ (33,286) $ (117,741) $ (180,703) Denominator: Weighted average common shares outstanding — basic and diluted 295,759,435 289,223,709 293,314,167 288,841,092 |
Schedule of Potential Shares of Common Stock that were Excluded from the Computation as they were Anti-Dilutive Using the Treasury Stock Method | The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method: As of September 30, (in thousands) 2023 2022 Options to purchase common stock 23,256 19,236 Unvested restricted stock units 10,331 9,839 Total number of potentially issuable shares 33,587 29,075 |
Description of Business (Detail
Description of Business (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | ||
Oct. 31, 2023 | Sep. 30, 2023 | Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Accumulated deficit | $ 2,650,231 | $ 2,532,490 | |
Subsequent Event | |||
Debt Instrument [Line Items] | |||
Private placement net of offering cost | $ 29,800 | ||
Subsequent Event | Private Placement | |||
Debt Instrument [Line Items] | |||
Purchase price (in dollars per share) | $ 12.16 | ||
Subsequent Event | Common Stock | |||
Debt Instrument [Line Items] | |||
Stock issued during period, shares, new issues (in shares) | 2,467,104 | ||
Stock issued from equity financing (in shares) | 2,467,104 | ||
Secured Debt | Senior Loans | Subsequent Event | |||
Debt Instrument [Line Items] | |||
Maximum borrowing capacity | $ 400,000 | ||
Basis spread on variable rate | 6.25% | ||
Maturity term | 6 years | ||
Proceeds from secured lines of credit | $ 387,400 | ||
Secured Debt | Senior Loans | Subsequent Event | Secured Overnight Financing Rate Floor | |||
Debt Instrument [Line Items] | |||
Basis spread on variable rate | 2.50% | ||
Secured Debt | Senior Loans | Subsequent Event | Secured Overnight Financing Rate Adjustment | |||
Debt Instrument [Line Items] | |||
Basis spread on variable rate | 0.26161% |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Narrative (Details) $ in Millions | Sep. 30, 2023 USD ($) |
Accounting Policies [Abstract] | |
Allowance for doubtful accounts receivable | $ 0.1 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Revenue by Product and Geographical Area (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Net product sales | $ 103,501 | $ 81,691 | $ 284,274 | $ 241,137 |
Galafold | ||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Net product sales | 100,733 | 81,631 | 281,177 | 241,056 |
Pombiliti + Opfolda | ||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Net product sales | 2,768 | 60 | 3,097 | 81 |
U.S. | ||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Net product sales | 37,801 | 30,222 | 103,760 | 81,940 |
Ex-U.S. | ||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||
Net product sales | $ 65,700 | $ 51,469 | $ 180,514 | $ 159,197 |
Intangible Assets (Details)
Intangible Assets (Details) $ in Millions | 3 Months Ended | 9 Months Ended |
Sep. 30, 2023 USD ($) | Sep. 30, 2023 USD ($) | |
Goodwill and Intangible Assets Disclosure [Abstract] | ||
Initial regulatory exclusivity period | 7 years | |
Amortization expenses of intangible assets | $ 0.8 | $ 1.7 |
Remainder of fiscal year 2023 | 2.5 | 2.5 |
2024 | $ 3.3 | $ 3.3 |
Cash, Cash Equivalents, Marke_3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($) | 9 Months Ended | 12 Months Ended | |
Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | |
Cash, Cash Equivalents, and Short-Term Investments [Abstract] | |||
Cash and cash equivalents | $ 263,320,000 | $ 148,813,000 | $ 277,592,000 |
Available-for-sale debt securities | 16,980,000 | 144,782,000 | |
Debt securities, realized gain (loss) | $ 0 | 0 | |
Fair value of available-for-sale debt securities in unrealized loss positions | $ 0 |
Cash, Cash Equivalents, Marke_4
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Components (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 |
Cash, Money Market Funds, and Marketable Securities | |||
Cash and cash equivalents | $ 263,320 | $ 148,813 | $ 277,592 |
Fair value, cash balances | 263,320 | 148,813 | |
Cost, available-for-sale securities | 16,977 | 144,700 | |
Gross unrealized gain, available-for-sale securities | 3 | 82 | |
Gross unrealized loss, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | 16,980 | 144,782 | |
Cost, cash balances and available-for-sale securities | 280,297 | 293,513 | |
Gross unrealized gain, cash balances and available-for-sale securities | 3 | 82 | |
Gross unrealized loss, cash balances and available-for-sale securities | 0 | 0 | |
Fair value, cash balances and available-for-sale securities | 280,300 | 293,595 | |
Commercial paper | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 16,826 | 144,299 | |
Gross unrealized gain, available-for-sale securities | 3 | 82 | |
Gross unrealized loss, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | 16,829 | 144,381 | |
Money market | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 100 | 350 | |
Gross unrealized gain, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | 100 | 350 | |
Gross unrealized loss, cash balances and available-for-sale securities | 0 | 0 | |
Certificates of deposit | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 51 | 51 | |
Gross unrealized gain, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | 51 | 51 | |
Gross unrealized loss, cash balances and available-for-sale securities | $ 0 | $ 0 |
Cash, Cash Equivalents, Marke_5
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash, Cash Equivalents, And Restricted Cash (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | Dec. 31, 2021 |
Cash, Cash Equivalents, and Short-Term Investments [Abstract] | ||||
Cash and cash equivalents | $ 263,320 | $ 148,813 | $ 277,592 | |
Restricted cash | 2,952 | 3,941 | ||
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows | $ 266,272 | $ 153,115 | $ 281,533 | $ 249,456 |
Inventories (Details)
Inventories (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 36,062 | $ 10,054 |
Work-in-process | 14,316 | 9,615 |
Finished goods | 6,558 | 4,147 |
Total inventories | $ 56,936 | $ 23,816 |
Inventories - Narrative (Detail
Inventories - Narrative (Details) - USD ($) $ in Millions | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Reserve for inventory | $ 0.3 | $ 0.4 |
Debt - Summary of Long Term Deb
Debt - Summary of Long Term Debt (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Debt Instrument [Line Items] | ||
Net carrying value of Long-term debt(2) | $ 394,071 | $ 391,990 |
Senior Loans | Senior Secured Term Loan due 2026 | ||
Debt Instrument [Line Items] | ||
Principal | 400,000 | 400,000 |
Less: debt discount | (3,384) | (4,571) |
Less: deferred financing | $ (2,545) | $ (3,439) |
Debt - Narrative (Details)
Debt - Narrative (Details) - Secured Debt - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended |
Oct. 31, 2023 | Dec. 31, 2023 | |
Senior Loans | Subsequent Event | ||
Debt Instrument [Line Items] | ||
Maximum borrowing capacity | $ 400 | |
Basis spread on variable rate | 6.25% | |
Maturity term | 6 years | |
Proceeds from secured lines of credit | $ 387.4 | |
Senior Loans | Subsequent Event | Secured Overnight Financing Rate Floor | ||
Debt Instrument [Line Items] | ||
Basis spread on variable rate | 2.50% | |
Senior Loans | Subsequent Event | Secured Overnight Financing Rate Adjustment | ||
Debt Instrument [Line Items] | ||
Basis spread on variable rate | 0.26161% | |
2029 Senior Secured Term Loan | Forecast | ||
Debt Instrument [Line Items] | ||
Loss on extinguishment of debt | $ 13.9 |
Debt - Interest Expense (Detail
Debt - Interest Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Debt Disclosure [Abstract] | ||||
Contractual interest expense | $ 12,270 | $ 8,945 | $ 35,289 | $ 24,034 |
Amortization of debt discount | 411 | 382 | 1,187 | 1,121 |
Amortization of deferred financing | $ 310 | $ 286 | $ 893 | $ 842 |
Stockholders' Equity (Details)
Stockholders' Equity (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended |
Oct. 31, 2023 | Sep. 30, 2023 | Sep. 30, 2023 | |
Subsidiary, Sale of Stock [Line Items] | |||
Offering price per share (in dollars per share) | $ 12.50 | ||
Subsequent Event | |||
Subsidiary, Sale of Stock [Line Items] | |||
Private placement net of offering cost | $ 29.8 | ||
Common Stock | Subsequent Event | |||
Subsidiary, Sale of Stock [Line Items] | |||
Stock issued from equity financing (in shares) | 2,467,104 | ||
ATM | |||
Subsidiary, Sale of Stock [Line Items] | |||
Number of shares issued, private placement (in shares) | 3,002,354 | 5,244,936 | |
Offering price per share (in dollars per share) | $ 12.68 | ||
Aggregate net proceeds from stock offering | $ 36.6 | $ 63.1 | |
Common stock, capital share reserved for future issuance | 184,400,000 | 184,400,000 | |
ATM | Common Stock | |||
Subsidiary, Sale of Stock [Line Items] | |||
Stock issued from equity financing (in shares) | 3,002,354 | 5,244,936 | |
Private Placement | Subsequent Event | |||
Subsidiary, Sale of Stock [Line Items] | |||
Purchase price (in dollars per share) | $ 12.16 |
Stock-Based Compensation - Weig
Stock-Based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected stock price volatility | 58.40% | 61% | 59.20% | 62.20% |
Risk free interest rate | 4.30% | 3.10% | 3.90% | 1.70% |
Expected life of options (years) | 5 years 6 months | 5 years 3 months 18 days | 5 years 6 months | 5 years 3 months 18 days |
Expected annual dividend per share (in dollars per share) | $ 0 | $ 0 | $ 0 | $ 0 |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock Option Activity (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Millions | 9 Months Ended |
Sep. 30, 2023 | |
Number of Shares | |
Balance at the beginning of the period (in shares) | 19,064 |
Options, granted (in shares) | 5,522 |
Options, exercised (in shares) | (1,032) |
Options, forfeited (in shares) | (248) |
Options, expired (in shares) | (50) |
Balance at the end of the period (in shares) | 23,256 |
Vested and unvested expected to vest as of the end of the period (in shares) | 21,389 |
Exercisable at the end of the period (in shares) | 13,246 |
Weighted Average Exercise Price | |
Balance at the beginning of the period (in dollars per share) | $ 11.31 |
Options, granted (in dollars per share) | 12.03 |
Options, exercised (in dollars per share) | 7.61 |
Options, forfeited (in dollars per share) | 11.51 |
Options, expired (in dollars per share) | 14.39 |
Balance at the end of the period (in dollars per share) | 11.64 |
Vested and unvested expected to vest as of the end of the period (in dollars per share) | 11.58 |
Exercisable at the end of the period (in dollars per share) | $ 11.12 |
Weighted Average Remaining Years and Aggregate Intrinsic Value | |
Balance at the end of the period | 6 years 8 months 12 days |
Vested and unvested expected to vest at the end of the period | 6 years 6 months |
Exercisable at the end of the period | 5 years 1 month 6 days |
Aggregate intrinsic value, options outstanding | $ 32.1 |
Aggregate intrinsic value, vested and unvested expected to vest | 31.1 |
Aggregate intrinsic value of options exercisable | $ 27.9 |
Stock-Based Compensation - Narr
Stock-Based Compensation - Narrative (Details) $ in Millions | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Options to purchase common stock | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Total unrecognized compensation costs | $ 39.8 |
Unrecognized compensation costs, period for recognition (in years) | 3 years |
Unvested restricted stock units | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Total unrecognized compensation costs | $ 63.1 |
Unrecognized compensation costs, period for recognition (in years) | 2 years |
Stock-Based Compensation - RSUs
Stock-Based Compensation - RSUs and PBRSUs Summary (Details) - Unvested restricted stock units $ / shares in Units, shares in Thousands, $ in Millions | 9 Months Ended |
Sep. 30, 2023 USD ($) $ / shares shares | |
Number of Shares | |
Non-vested units as of the beginning of the period (in shares) | shares | 9,717 |
Granted (in shares) | shares | 4,514 |
Vested (in shares) | shares | (3,252) |
Forfeited (in shares) | shares | (648) |
Non-vested units as of the end of the period (in shares) | shares | 10,331 |
Weighted Average Grant Date Fair Value | |
Non-vested units as of the beginning of the period (in dollars per share) | $ / shares | $ 13.07 |
Granted (in dollars per share) | $ / shares | 13.08 |
Vested (in dollars per share) | $ / shares | 12.25 |
Forfeited (in dollars per share) | $ / shares | 10.07 |
Non-vested units as of the end of the period (in dollars per share) | $ / shares | $ 13.56 |
Weighted Average Remaining Years and Aggregate Intrinsic Value | |
Non-vested units, weighted average remaining years | 2 years 2 months 12 days |
Non-vested units, aggregate intrinsic value | $ | $ 125.6 |
Stock-Based Compensation - Expe
Stock-Based Compensation - Expense Summary (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total equity compensation expense | $ 16,511 | $ 14,772 | $ 67,982 | $ 57,886 |
Research and development expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total equity compensation expense | 4,380 | 5,428 | 16,987 | 19,172 |
Selling, general, and administrative expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total equity compensation expense | $ 12,131 | $ 9,344 | $ 50,995 | $ 38,714 |
Assets and Liabilities Measur_3
Assets and Liabilities Measured at Fair Value - Summary of Fair Value of Assets and Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Assets: | ||
Fair value of assets | $ 23,647 | $ 150,189 |
Liabilities: | ||
Contingent consideration payable | 21,417 | |
Deferred compensation plan liability | 6,718 | 5,458 |
Fair value of liabilities | 6,718 | 26,875 |
Commercial paper | ||
Assets: | ||
Fair value of assets | 16,829 | 144,381 |
Money market | ||
Assets: | ||
Fair value of assets | 6,818 | 5,808 |
Level 2 | ||
Assets: | ||
Fair value of assets | 23,647 | 150,189 |
Liabilities: | ||
Contingent consideration payable | 0 | |
Deferred compensation plan liability | 6,718 | 5,458 |
Fair value of liabilities | 6,718 | 5,458 |
Level 2 | Commercial paper | ||
Assets: | ||
Fair value of assets | 16,829 | |
Level 2 | Money market | ||
Assets: | ||
Fair value of assets | $ 6,818 | 5,808 |
Level 3 | ||
Liabilities: | ||
Contingent consideration payable | 21,417 | |
Deferred compensation plan liability | 0 | |
Fair value of liabilities | $ 21,417 |
Assets and Liabilities Measur_4
Assets and Liabilities Measured at Fair Value - Narrative (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Mar. 31, 2023 | Dec. 31, 2022 |
Business Acquisition [Line Items] | |||
Contingent consideration | $ 21,417 | ||
Callidus Biopharma Inc | |||
Business Acquisition [Line Items] | |||
Contingent consideration | $ 15,000 | $ 9,000 |
Assets and Liabilities Measur_5
Assets and Liabilities Measured at Fair Value - Level 3 Roll Forward (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | ||||
Balance, beginning of the period | $ 13,005 | $ 19,266 | $ 21,417 | $ 20,339 |
Changes in fair value during the period, included in the Consolidated Statements of Operations | (1,995) | (567) | (2,583) | 506 |
Milestone paid or payable in cash | (15,000) | 0 | (24,000) | 0 |
Balance, end of the period | $ 0 | $ 19,833 | $ 0 | $ 19,833 |
Basic and Diluted Net Loss pe_3
Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Numerator: | ||||
Net loss attributable to common stockholders | $ (21,577) | $ (33,286) | $ (117,741) | $ (180,703) |
Denominator: | ||||
Weighted average common shares outstanding - basic (in shares) | 295,759,435 | 289,223,709 | 293,314,167 | 288,841,092 |
Weighted average common shares outstanding - diluted (in shares) | 295,759,435 | 289,223,709 | 293,314,167 | 288,841,092 |
Antidilutive securities | ||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 33,587,000 | 29,075,000 | ||
Options to purchase common stock | ||||
Antidilutive securities | ||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 23,256,000 | 19,236,000 | ||
Unvested restricted stock units | ||||
Antidilutive securities | ||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 10,331,000 | 9,839,000 |
Basic and Diluted Net Loss pe_4
Basic and Diluted Net Loss per Common Share - Narrative (Details) | Sep. 30, 2023 $ / shares |
Private Placement | |
Antidilutive securities | |
Warrants exercise price (in dollars per share) | $ 0.01 |