Cover
Cover - shares | 3 Months Ended | |
Mar. 31, 2024 | Apr. 25, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2024 | |
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 $0.01 per share | |
Trading Symbol | FOLD | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 296,198,963 | |
Entity Central Index Key | 0001178879 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q1 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 209,761 | $ 246,994 |
Investments in marketable securities | 29,842 | 39,206 |
Accounts receivable | 76,433 | 87,632 |
Inventories | 60,759 | 59,696 |
Prepaid expenses and other current assets | 54,444 | 49,533 |
Total current assets | 431,239 | 483,061 |
Operating lease right-of-use assets, net | 23,003 | 26,312 |
Property and equipment, less accumulated depreciation of $26,563 and $25,429 at March 31, 2024 and December 31, 2023, respectively | 32,421 | 31,667 |
Intangible assets, less accumulated amortization of $3,328 and $2,510 at March 31, 2024 and December 31, 2023, respectively | 19,672 | 20,490 |
Goodwill | 197,797 | 197,797 |
Other non-current assets | 17,657 | 18,553 |
Total Assets | 721,789 | 777,880 |
Current liabilities: | ||
Accounts payable | 9,210 | 15,120 |
Accrued expenses and other current liabilities | 124,622 | 144,245 |
Operating lease liabilities | 8,270 | 8,324 |
Total current liabilities | 142,102 | 167,689 |
Long-term debt | 388,391 | 387,858 |
Operating lease liabilities | 47,831 | 48,877 |
Other non-current liabilities | 12,771 | 13,282 |
Total liabilities | 591,095 | 617,706 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Common stock, $0.01 par value, 500,000,000 shares authorized, 296,159,417 and 293,594,209 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively | 2,922 | 2,918 |
Additional paid-in capital | 2,853,550 | 2,836,018 |
Accumulated other comprehensive loss: | ||
Foreign currency translation adjustment | 6,847 | 5,429 |
Unrealized loss on available-for-sale securities | (203) | (188) |
Warrants | 71 | 71 |
Accumulated deficit | (2,732,493) | (2,684,074) |
Total stockholders’ equity | 130,694 | 160,174 |
Total Liabilities and Stockholders’ Equity | $ 721,789 | $ 777,880 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Statement of Financial Position [Abstract] | ||
Accumulated depreciation of property and equipment | $ 26,563 | $ 25,429 |
Accumulated amortization of intangible assets | $ 3,328 | $ 2,510 |
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) | 296,159,417 | 293,594,209 |
Common stock, shares outstanding (in shares) | 296,159,417 | 293,594,209 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Income Statement [Abstract] | ||
Net product sales | $ 110,403 | $ 86,270 |
Cost of goods sold | 13,567 | 6,942 |
Gross profit | 96,836 | 79,328 |
Operating expenses: | ||
Research and development | 28,329 | 41,499 |
Selling, general, and administrative | 88,029 | 73,957 |
Changes in fair value of contingent consideration payable | 0 | 251 |
Restructuring charges | 6,045 | 0 |
Depreciation and amortization | 2,154 | 1,257 |
Total operating expenses | 124,557 | 116,964 |
Loss from operations | (27,721) | (37,636) |
Other expense: | ||
Interest income | 1,540 | 2,199 |
Interest expense | (12,436) | (11,844) |
Other expense | (4,966) | (5,938) |
Loss before income tax | (43,583) | (53,219) |
Income tax (expense) benefit | (4,836) | 287 |
Net loss attributable to common stockholders | $ (48,419) | $ (52,932) |
Net loss attributable to common stockholders per common share - basic (in dollars per share) | $ (0.16) | $ (0.18) |
Net loss attributable to common stockholders per common share — diluted (in dollars per share) | $ (0.16) | $ (0.18) |
Weighted average common shares outstanding - basic (in shares) | 302,903,009 | 291,336,750 |
Weighted average common shares outstanding - diluted (in shares) | 302,903,009 | 291,336,750 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (48,419) | $ (52,932) |
Other comprehensive gain, net of tax: | ||
Foreign currency translation adjustment gain | 1,418 | 5,446 |
Unrealized loss on available-for-sale securities | (15) | (85) |
Other comprehensive gain | 1,403 | 5,361 |
Comprehensive loss | $ (47,016) | $ (47,571) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Warrants | Other Comprehensive Gain (Loss) | Accumulated Deficit |
Beginning balance (in shares) at Dec. 31, 2022 | 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) | 384,108 | |||||
Stock options exercised, net | 2,655 | $ 3 | 2,652 | |||
Vesting of restricted stock units, net of taxes (in shares) | 1,612,975 | |||||
Vesting of restricted stock units, net of taxes | (12,806) | (12,806) | ||||
Stock-based compensation | 34,894 | 34,894 | ||||
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares) | 195,229 | |||||
Issuance of shares in connection with at-the-market offering, net of issuance costs | 2,354 | $ 2 | 2,352 | |||
Unrealized loss on available-for-sale securities | (85) | (85) | ||||
Foreign currency translation adjustment | 5,446 | 5,446 | ||||
Net loss | (52,932) | (52,932) | ||||
Ending balance (in shares) at Mar. 31, 2023 | 283,300,585 | |||||
Ending balance at Mar. 31, 2023 | $ 102,573 | $ 2,820 | 2,691,836 | 83 | (6,744) | (2,585,422) |
Beginning balance (in shares) at Dec. 31, 2023 | 293,594,209 | 293,594,209 | ||||
Beginning balance at Dec. 31, 2023 | $ 160,174 | $ 2,918 | 2,836,018 | 71 | 5,241 | (2,684,074) |
Increase (Decrease) in Stockholders' Equity | ||||||
Stock options exercised, net (in shares) | 439,000 | 444,497 | ||||
Stock options exercised, net | $ 3,454 | $ 4 | 3,450 | |||
Vesting of restricted stock units, net of taxes (in shares) | 2,120,711 | |||||
Vesting of restricted stock units, net of taxes | (16,721) | (16,721) | ||||
Stock-based compensation | 30,803 | 30,803 | ||||
Unrealized loss on available-for-sale securities | (15) | (15) | ||||
Foreign currency translation adjustment | 1,418 | 1,418 | ||||
Net loss | $ (48,419) | (48,419) | ||||
Ending balance (in shares) at Mar. 31, 2024 | 296,159,417 | 296,159,417 | ||||
Ending balance at Mar. 31, 2024 | $ 130,694 | $ 2,922 | $ 2,853,550 | $ 71 | $ 6,644 | $ (2,732,493) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Operating activities | ||
Net loss | $ (48,419) | $ (52,932) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Amortization of debt discount and deferred financing | 531 | 667 |
Depreciation and amortization | 2,154 | 1,257 |
Stock-based compensation | 30,803 | 34,894 |
Non-cash changes in the fair value of contingent consideration payable | 0 | 251 |
Foreign currency remeasurement loss | 1,736 | 5,885 |
Deferred taxes | 0 | (4,939) |
Other | 5,818 | 0 |
Changes in operating assets and liabilities: | ||
Accounts receivable | 9,557 | (1,367) |
Inventories | (1,678) | (3,158) |
Prepaid expenses and other current assets | (5,340) | 1,839 |
Accounts payable, accrued expenses, and other current liabilities | (24,483) | (72) |
Other non-current assets and liabilities | (374) | (394) |
Net cash used in operating activities | (29,695) | (18,069) |
Investing activities | ||
Sale and redemption of marketable securities | 38,907 | 54,944 |
Purchases of marketable securities | (29,559) | (16,747) |
Capital expenditures | (1,811) | (1,942) |
Net cash provided by investing activities | 7,537 | 36,255 |
Financing activities | ||
Payment of finance leases | (42) | (28) |
Withholding taxes paid on vested restricted stock units | (16,721) | (12,806) |
Proceeds from stock options exercised, net | 3,454 | 2,655 |
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs | 0 | 2,354 |
Net cash used in financing activities | (13,309) | (7,825) |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | (1,816) | 1,507 |
Net (decrease) increase in cash, cash equivalents, and restricted cash at the end of the period | (37,283) | 11,868 |
Cash, cash equivalents, and restricted cash at the beginning of period | 250,077 | 153,115 |
Cash, cash equivalents, and restricted cash at the end of period | 212,794 | 164,983 |
Supplemental disclosures of cash flow information | ||
Cash paid during the period for interest | 11,801 | 11,361 |
Capital expenditures unpaid at the end of period | 1,110 | 1,260 |
Cash paid for taxes | 635 | 178 |
Tenant improvements paid through lease incentives | $ 52 | $ 0 |
Description of Business
Description of Business | 3 Months Ended |
Mar. 31, 2024 | |
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 seeks to deliver the highest quality therapies that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. The Company's two marketed therapies are Galafold ® , the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti ® + Opfolda ® , a novel treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease. Galafold ® (also referred to as "migalastat"), is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold ® has been granted orphan drug designation in the U.S., E.U., U.K., Japan and several other countries. Pombiliti ® + Opfolda ® (also referred to as "cipaglucosidase alfa-atga/miglustat"), was approved in 2023 in the three largest Pompe markets: the U.S., the E.U., and the U.K. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti ® + Opfolda ® has been granted orphan drug designation in the U.S., E.U., U.K., Japan and several other countries. The Company had an accumulated deficit of $2.7 billion as of March 31, 2024 and anticipates incurring losses through the fiscal year ending December 31, 2024. The Company has historically funded its operations through stock offerings, product revenues, debt issuances, collaborations, and other financing arrangements. Based on its current operating model, the Company believes 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 opportunities, pipeline expansion, and investment in manufacturing capabilities could impact the Company's long-term capital requirements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited Consolidated Financial Statements have been prepared 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, certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. 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. Management has determined that the Company operates in one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. 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, 2023. 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, 2023. 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 weighted 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. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other expense. 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 gain 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 as a component of 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, marketable securities, and accounts receivable. 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's accounts receivable at March 31, 2024 have primarily arisen from Galafold ® sales in Europe, the U.S., and Japan. The Company periodically assesses 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 March 31, 2024, 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 obligation to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. 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 primarily consist of 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 March 31, (in thousands) 2024 2023 Galafold ® $ 99,359 $ 86,112 Pombiliti ® + Opfolda ® 11,044 158 Total net product sales $ 110,403 $ 86,270 The following table summarizes the Company's net product sales disaggregated by geographic area: Three Months Ended March 31, (in thousands) 2024 2023 U.S. $ 37,375 $ 28,831 Ex-U.S. 73,028 57,439 Total net product sales $ 110,403 $ 86,270 Inventories and Cost of Goods Sold 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 Pombiliti ® + Opfolda ® inventory 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. Recent Accounting Developments - Guidance Adopted in 2024 In November 2023, the Financial Accounting Standards Board ("FASB") issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures . The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. This ASU applies to disclosure requirements only, and the Company will provide required annual disclosures as part of the 2024 Annual Report on Form 10-K and required interim disclosures as part of 2025 Quarterly Reports on Form 10-Q. Recent Accounting Developments - Guidance Not Yet Adopted In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): I mprovements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements. |
Cash, Cash Equivalents, Marketa
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, 2024, the Company held $209.8 million in cash and cash equivalents and $29.8 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 gain in the 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 the Consolidated Statements of Operations 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 the Consolidated Statements of Operations. The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of 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 March 31, 2024 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 209,761 $ — $ — $ 209,761 Commercial paper 25,718 — (4) 25,714 U.S. government agency bonds 3,978 — (1) 3,977 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 239,608 $ — $ (5) $ 239,603 Included in cash and cash equivalents $ 209,761 $ — $ — $ 209,761 Included in marketable securities 29,847 — (5) 29,842 Total cash, cash equivalents, and marketable securities $ 239,608 $ — $ (5) $ 239,603 As of December 31, 2023 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Commercial paper 14,651 12 — 14,663 Treasury bill 12,944 2 — 12,946 U.S. government agency bonds 11,450 — (4) 11,446 Money market 100 — — 100 Certificate of deposit 51 — — 51 $ 286,190 $ 14 $ (4) $ 286,200 Included in cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Included in marketable securities 39,196 14 (4) 39,206 Total cash, cash equivalents, and marketable securities $ 286,190 $ 14 $ (4) $ 286,200 For both the three months ended March 31, 2024 and March 31, 2023, 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 March 31, 2024 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 as a component of other comprehensive gain. The fair value of these marketable securities in unrealized loss positions are $29.7 million and $11.4 million as of March 31, 2024 and December 31, 2023, respectively. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows. As of March 31, (in thousands) 2024 2023 Cash and cash equivalents $ 209,761 $ 160,602 Restricted cash 3,033 4,381 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 212,794 $ 164,983 |
Inventories
Inventories | 3 Months Ended |
Mar. 31, 2024 | |
Inventory Disclosure [Abstract] | |
Inventories | Inventories The following table summarizes the components of the Company’s inventories for each of the periods indicated: (in thousands) March 31, 2024 December 31, 2023 Raw materials $ 31,613 $ 30,230 Work-in-process 22,146 22,597 Finished goods 7,000 6,869 Total inventories $ 60,759 $ 59,696 The Company's reserve for inventory was $2.9 million and $0.5 million as of March 31, 2024 and December 31, 2023, respectively. |
Debt
Debt | 3 Months Ended |
Mar. 31, 2024 | |
Debt Disclosure [Abstract] | |
Debt | Debt The following table summarizes the Company's debt for each of the periods indicated: (in thousands) March 31, 2024 December 31, 2023 Senior Secured Term Loan due 2029: Principal $ 400,000 $ 400,000 Less: debt discount (1) (9,230) (9,652) Less: deferred financing (1) (2,379) (2,490) Net carrying value of Long-term debt $ 388,391 $ 387,858 ______________________________ (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 2029 using the effective interest rate method. Interest Expense The following table sets forth interest expense recognized related to the Company's debt for the three months ended March 31, 2024 and 2023, respectively: Three Months Ended March 31, (in thousands) 2024 2023 Contractual interest expense $ 11,974 $ 11,230 Amortization of debt discount $ 422 $ 381 Amortization of deferred financing $ 109 $ 286 |
Restructuring
Restructuring | 3 Months Ended |
Mar. 31, 2024 | |
Restructuring and Related Activities [Abstract] | |
Restructuring | Restructuring In the first quarter of 2024, restructuring charges are primarily related to an initiative to reduce operating costs by abandoning one of the Company's leases that it no longer believes is necessary to conduct its operations. The associated liabilities are recorded in accrued expenses and other current liabilities in the Company’s Consolidated Balance Sheets. Total charges incurred are summarized as follows: (in thousands) Other Facility and Non-Lease Costs Accelerated Depreciation Total Balance as of December 31, 2023 $ — $ — $ — Restructuring charges 3,201 2,844 6,045 Non-cash items — (2,844) (2,844) Cash settled (310) — (310) Balance as of March 31, 2024 $ 2,891 $ — $ 2,891 |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation | Stock-Based Compensation The 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 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 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 were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Three Months Ended March 31, 2024 2023 Expected stock price volatility 57.2 % 59.3 % Risk free interest rate 4.0 % 3.9 % Expected life of options (years) 5.6 5.5 Expected annual dividend per share $ — $ — A summary of the Company's stock options for the three months ended March 31, 2024 were as follows: Number of Weighted Average Exercise Weighted Average Remaining Aggregate (in thousands) (in millions) Options outstanding, December 31, 2023 23,002 $ 11.69 Granted 4,033 $ 14.23 Exercised (439) $ 7.86 Forfeited (271) $ 12.56 Expired (20) $ 17.28 Options outstanding, March 31, 2024 26,305 $ 12.13 6.4 $ 23.9 Vested and unvested expected to vest, March 31, 2024 24,409 $ 12.08 6.2 $ 23.5 Exercisable at March 31, 2024 15,979 $ 11.60 4.9 $ 22.3 As of March 31, 2024, the total unrecognized compensation cost related to non-vested stock options granted was $52.5 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 three months ended March 31, 2024 is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Non-vested units as of December 31, 2023 10,033 $ 13.37 Granted 3,251 $ 15.35 Vested (3,332) $ 13.49 Forfeited (393) $ 14.66 Non-vested units as of March 31, 2024 9,559 $ 13.92 2.5 $ 112.6 As of March 31, 2024, there was $77.7 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 three 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 March 31, (in thousands) 2024 2023 Research and development expense $ 4,871 $ 8,490 Selling, general, and administrative expense 25,932 26,404 Total equity compensation expense $ 30,803 $ 34,894 |
Assets and Liabilities Measured
Assets and Liabilities Measured at Fair Value | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, 2024 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 25,714 $ 25,714 U.S. government agency bonds — 3,977 3,977 Money market 7,201 — 7,201 $ 7,201 $ 29,691 $ 36,892 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability $ 7,101 $ — $ 7,101 $ 7,101 $ — $ 7,101 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, 2023 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 14,663 $ 14,663 Treasury bill — 12,946 12,946 U.S. government agency bonds — 11,446 11,446 Money market 7,631 — 7,631 $ 7,631 $ 39,055 $ 46,686 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability 7,531 — 7,531 $ 7,531 $ — $ 7,531 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 or liabilities as of March 31, 2024 or December 31, 2023. Cash, Money Market Funds, and Marketable Securities |
Basic and Diluted Net Loss per
Basic and Diluted Net Loss per Common Share | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, (in thousands, except per share amounts) 2024 2023 Numerator: Net loss attributable to common stockholders $ (48,419) $ (52,932) Denominator: Weighted average common shares outstanding — basic and diluted 302,903,009 291,336,750 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 $0.01. The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method: As of March 31, (in thousands) 2024 2023 Options to purchase common stock 26,305 23,437 Unvested restricted stock units 9,559 9,430 Total number of potentially issuable shares 35,864 32,867 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Pay vs Performance Disclosure | ||
Net loss | $ (48,419) | $ (52,932) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Mar. 31, 2024 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited Consolidated Financial Statements have been prepared 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, certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. 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. Management has determined that the Company operates in one segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. |
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 weighted 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. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other expense. |
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 gain 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, marketable securities, and accounts receivable. 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's accounts receivable at March 31, 2024 have primarily arisen from 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 obligation to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. 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 primarily consist of 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 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 Pombiliti ® + Opfolda ® |
Recent Accounting Developments | Recent Accounting Developments - Guidance Adopted in 2024 In November 2023, the Financial Accounting Standards Board ("FASB") issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures . The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. This ASU applies to disclosure requirements only, and the Company will provide required annual disclosures as part of the 2024 Annual Report on Form 10-K and required interim disclosures as part of 2025 Quarterly Reports on Form 10-Q. Recent Accounting Developments - Guidance Not Yet Adopted In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): I mprovements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, (in thousands) 2024 2023 Galafold ® $ 99,359 $ 86,112 Pombiliti ® + Opfolda ® 11,044 158 Total net product sales $ 110,403 $ 86,270 The following table summarizes the Company's net product sales disaggregated by geographic area: Three Months Ended March 31, (in thousands) 2024 2023 U.S. $ 37,375 $ 28,831 Ex-U.S. 73,028 57,439 Total net product sales $ 110,403 $ 86,270 |
Cash, Cash Equivalents, Marke_2
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, 2024 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 209,761 $ — $ — $ 209,761 Commercial paper 25,718 — (4) 25,714 U.S. government agency bonds 3,978 — (1) 3,977 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 239,608 $ — $ (5) $ 239,603 Included in cash and cash equivalents $ 209,761 $ — $ — $ 209,761 Included in marketable securities 29,847 — (5) 29,842 Total cash, cash equivalents, and marketable securities $ 239,608 $ — $ (5) $ 239,603 As of December 31, 2023 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Commercial paper 14,651 12 — 14,663 Treasury bill 12,944 2 — 12,946 U.S. government agency bonds 11,450 — (4) 11,446 Money market 100 — — 100 Certificate of deposit 51 — — 51 $ 286,190 $ 14 $ (4) $ 286,200 Included in cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Included in marketable securities 39,196 14 (4) 39,206 Total cash, cash equivalents, and marketable securities $ 286,190 $ 14 $ (4) $ 286,200 |
Schedule of Cash, Cash Equivalents, and Restricted Cash | The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows. As of March 31, (in thousands) 2024 2023 Cash and cash equivalents $ 209,761 $ 160,602 Restricted cash 3,033 4,381 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 212,794 $ 164,983 |
Inventories (Tables)
Inventories (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventories for the Period | The following table summarizes the components of the Company’s inventories for each of the periods indicated: (in thousands) March 31, 2024 December 31, 2023 Raw materials $ 31,613 $ 30,230 Work-in-process 22,146 22,597 Finished goods 7,000 6,869 Total inventories $ 60,759 $ 59,696 |
Debt (Tables)
Debt (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Debt Disclosure [Abstract] | |
Schedule of Liability Components of Long-Term Debt | The following table summarizes the Company's debt for each of the periods indicated: (in thousands) March 31, 2024 December 31, 2023 Senior Secured Term Loan due 2029: Principal $ 400,000 $ 400,000 Less: debt discount (1) (9,230) (9,652) Less: deferred financing (1) (2,379) (2,490) Net carrying value of Long-term debt $ 388,391 $ 387,858 ______________________________ (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 2029 using the effective interest rate method. |
Schedule of Components of Total Interest Expense | The following table sets forth interest expense recognized related to the Company's debt for the three months ended March 31, 2024 and 2023, respectively: Three Months Ended March 31, (in thousands) 2024 2023 Contractual interest expense $ 11,974 $ 11,230 Amortization of debt discount $ 422 $ 381 Amortization of deferred financing $ 109 $ 286 |
Restructuring (Tables)
Restructuring (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Restructuring and Related Activities [Abstract] | |
Schedule of Total Charges Incurred | Total charges incurred are summarized as follows: (in thousands) Other Facility and Non-Lease Costs Accelerated Depreciation Total Balance as of December 31, 2023 $ — $ — $ — Restructuring charges 3,201 2,844 6,045 Non-cash items — (2,844) (2,844) Cash settled (310) — (310) Balance as of March 31, 2024 $ 2,891 $ — $ 2,891 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Schedule of Fair Value of Options | The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Three Months Ended March 31, 2024 2023 Expected stock price volatility 57.2 % 59.3 % Risk free interest rate 4.0 % 3.9 % Expected life of options (years) 5.6 5.5 Expected annual dividend per share $ — $ — |
Schedule of Stock Options Activity | A summary of the Company's stock options for the three months ended March 31, 2024 were as follows: Number of Weighted Average Exercise Weighted Average Remaining Aggregate (in thousands) (in millions) Options outstanding, December 31, 2023 23,002 $ 11.69 Granted 4,033 $ 14.23 Exercised (439) $ 7.86 Forfeited (271) $ 12.56 Expired (20) $ 17.28 Options outstanding, March 31, 2024 26,305 $ 12.13 6.4 $ 23.9 Vested and unvested expected to vest, March 31, 2024 24,409 $ 12.08 6.2 $ 23.5 Exercisable at March 31, 2024 15,979 $ 11.60 4.9 $ 22.3 |
Schedule of Non-Vested RSU Activity under the Plan | A summary of non-vested RSU activity under the Plan for the three months ended March 31, 2024 is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Non-vested units as of December 31, 2023 10,033 $ 13.37 Granted 3,251 $ 15.35 Vested (3,332) $ 13.49 Forfeited (393) $ 14.66 Non-vested units as of March 31, 2024 9,559 $ 13.92 2.5 $ 112.6 |
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 March 31, (in thousands) 2024 2023 Research and development expense $ 4,871 $ 8,490 Selling, general, and administrative expense 25,932 26,404 Total equity compensation expense $ 30,803 $ 34,894 |
Assets and Liabilities Measur_2
Assets and Liabilities Measured at Fair Value (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, 2024 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 25,714 $ 25,714 U.S. government agency bonds — 3,977 3,977 Money market 7,201 — 7,201 $ 7,201 $ 29,691 $ 36,892 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability $ 7,101 $ — $ 7,101 $ 7,101 $ — $ 7,101 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, 2023 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 14,663 $ 14,663 Treasury bill — 12,946 12,946 U.S. government agency bonds — 11,446 11,446 Money market 7,631 — 7,631 $ 7,631 $ 39,055 $ 46,686 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability 7,531 — 7,531 $ 7,531 $ — $ 7,531 |
Basic and Diluted Net Loss pe_2
Basic and Diluted Net Loss per Common Share (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
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 March 31, (in thousands, except per share amounts) 2024 2023 Numerator: Net loss attributable to common stockholders $ (48,419) $ (52,932) Denominator: Weighted average common shares outstanding — basic and diluted 302,903,009 291,336,750 |
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 March 31, (in thousands) 2024 2023 Options to purchase common stock 26,305 23,437 Unvested restricted stock units 9,559 9,430 Total number of potentially issuable shares 35,864 32,867 |
Description of Business (Detail
Description of Business (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Accumulated deficit | $ 2,732,493 | $ 2,684,074 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Narrative (Details) $ in Millions | 3 Months Ended |
Mar. 31, 2024 USD ($) segment | |
Accounting Policies [Abstract] | |
Number of reportable segments | 1 |
Number of operating segments | 1 |
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 | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Disaggregation of Revenue [Line Items] | ||
Net product sales | $ 110,403 | $ 86,270 |
U.S. | ||
Disaggregation of Revenue [Line Items] | ||
Net product sales | 37,375 | 28,831 |
Ex-U.S. | ||
Disaggregation of Revenue [Line Items] | ||
Net product sales | 73,028 | 57,439 |
Galafold | ||
Disaggregation of Revenue [Line Items] | ||
Net product sales | 99,359 | 86,112 |
Pombiliti + Opfolda | ||
Disaggregation of Revenue [Line Items] | ||
Net product sales | $ 11,044 | $ 158 |
Cash, Cash Equivalents, Marke_3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($) | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Cash, Cash Equivalents, and Short-Term Investments [Abstract] | |||
Cash and cash equivalents | $ 209,761,000 | $ 160,602,000 | $ 246,994,000 |
Available-for-sale debt securities | 29,842,000 | 39,206,000 | |
Debt securities, realized gain (loss) | 0 | $ 0 | |
Fair value of available-for-sale debt securities in unrealized loss positions | $ 29,700,000 | $ 11,400,000 |
Cash, Cash Equivalents, Marke_4
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash and Available for Sale Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 |
Cash, Money Market Funds, and Marketable Securities | |||
Cash and cash equivalents | $ 209,761 | $ 246,994 | $ 160,602 |
Fair value, cash balances | 209,761 | 246,994 | |
Cost, available-for-sale securities | 29,847 | 39,196 | |
Gross unrealized gain, available-for-sale securities | 0 | 14 | |
Gross unrealized loss, available-for-sale securities | (5) | (4) | |
Fair value, available-for-sale debt securities | 29,842 | 39,206 | |
Cost, cash balances and available-for-sale securities | 239,608 | 286,190 | |
Gross unrealized gain, cash balances and available-for-sale securities | 0 | 14 | |
Gross unrealized loss, cash balances and available-for-sale securities | (5) | (4) | |
Fair value, cash balances and available-for-sale securities | 239,603 | 286,200 | |
Commercial paper | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 25,718 | 14,651 | |
Gross unrealized gain, available-for-sale securities | 0 | 12 | |
Gross unrealized loss, available-for-sale securities | (4) | 0 | |
Fair value, available-for-sale debt securities | 25,714 | 14,663 | |
Treasury bill | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 12,944 | ||
Gross unrealized gain, available-for-sale securities | 2 | ||
Gross unrealized loss, available-for-sale securities | 0 | ||
Fair value, available-for-sale debt securities | 12,946 | ||
U.S. government agency bonds | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 3,978 | 11,450 | |
Gross unrealized gain, available-for-sale securities | 0 | 0 | |
Gross unrealized loss, available-for-sale securities | (1) | (4) | |
Fair value, available-for-sale debt securities | 3,977 | 11,446 | |
Money market | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 100 | 100 | |
Gross unrealized gain, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | 100 | 100 | |
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 - Reconciliation of Cash and Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 | Dec. 31, 2022 |
Cash, Cash Equivalents, and Short-Term Investments [Abstract] | ||||
Cash and cash equivalents | $ 209,761 | $ 246,994 | $ 160,602 | |
Restricted cash | 3,033 | 4,381 | ||
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows | $ 212,794 | $ 250,077 | $ 164,983 | $ 153,115 |
Inventories (Details)
Inventories (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 31,613 | $ 30,230 |
Work-in-process | 22,146 | 22,597 |
Finished goods | 7,000 | 6,869 |
Total inventories | $ 60,759 | $ 59,696 |
Inventories - Narrative (Detail
Inventories - Narrative (Details) - USD ($) $ in Millions | Mar. 31, 2024 | Dec. 31, 2023 |
Inventory Disclosure [Abstract] | ||
Reserve for inventory | $ 2.9 | $ 0.5 |
Debt - Summary of Long Term Deb
Debt - Summary of Long Term Debt (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Debt Instrument [Line Items] | ||
Net carrying value of Long-term debt | $ 388,391 | $ 387,858 |
Senior Loans | Senior Secured Term Loan due 2029 | ||
Debt Instrument [Line Items] | ||
Principal | 400,000 | 400,000 |
Less: debt discount | (9,230) | (9,652) |
Less: deferred financing | $ (2,379) | $ (2,490) |
Debt - Interest Expense (Detail
Debt - Interest Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Debt Disclosure [Abstract] | ||
Contractual interest expense | $ 11,974 | $ 11,230 |
Amortization of debt discount | 422 | 381 |
Amortization of deferred financing | $ 109 | $ 286 |
Restructuring (Details)
Restructuring (Details) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 USD ($) lease | Mar. 31, 2023 USD ($) | |
Restructuring Cost and Reserve [Line Items] | ||
Number of leases abandoned | lease | 1 | |
Restructuring Reserve [Roll Forward] | ||
Balance | $ 0 | |
Restructuring charges | 6,045 | $ 0 |
Non-cash items | (2,844) | |
Cash settled | (310) | |
Balance | 2,891 | |
Other Facility and Non-Lease Costs | ||
Restructuring Reserve [Roll Forward] | ||
Balance | 0 | |
Restructuring charges | 3,201 | |
Non-cash items | 0 | |
Cash settled | (310) | |
Balance | 2,891 | |
Accelerated Depreciation | ||
Restructuring Reserve [Roll Forward] | ||
Balance | 0 | |
Restructuring charges | 2,844 | |
Non-cash items | (2,844) | |
Cash settled | 0 | |
Balance | $ 0 |
Stock-Based Compensation - Weig
Stock-Based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected stock price volatility | 57.20% | 59.30% |
Risk free interest rate | 4% | 3.90% |
Expected life of options (years) | 5 years 7 months 6 days | 5 years 6 months |
Expected annual dividend per share (in dollars per share) | $ 0 | $ 0 |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock Option Activity (Details) $ / shares in Units, shares in Thousands, $ in Millions | 3 Months Ended |
Mar. 31, 2024 USD ($) $ / shares shares | |
Number of Shares | |
Balance at the beginning of the period (in shares) | shares | 23,002 |
Options, granted (in shares) | shares | 4,033 |
Options, exercised (in shares) | shares | (439) |
Options, forfeited (in shares) | shares | (271) |
Options, expired (in shares) | shares | (20) |
Balance at the end of the period (in shares) | shares | 26,305 |
Vested and unvested expected to vest as of the end of the period (in shares) | shares | 24,409 |
Exercisable at the end of the period (in shares) | shares | 15,979 |
Weighted Average Exercise Price | |
Balance at the beginning of the period (in dollars per share) | $ / shares | $ 11.69 |
Options, granted (in dollars per share) | $ / shares | 14.23 |
Options, exercised (in dollars per share) | $ / shares | 7.86 |
Options, forfeited (in dollars per share) | $ / shares | 12.56 |
Options, expired (in dollars per share) | $ / shares | 17.28 |
Balance at the end of the period (in dollars per share) | $ / shares | 12.13 |
Vested and unvested expected to vest as of the end of the period (in dollars per share) | $ / shares | 12.08 |
Exercisable at the end of the period (in dollars per share) | $ / shares | $ 11.60 |
Weighted Average Remaining Years and Aggregate Intrinsic Value | |
Balance at the end of the period | 6 years 4 months 24 days |
Vested and unvested expected to vest at the end of the period | 6 years 2 months 12 days |
Exercisable at the end of the period | 4 years 10 months 24 days |
Aggregate intrinsic value, options outstanding | $ | $ 23.9 |
Aggregate intrinsic value, vested and unvested expected to vest | $ | 23.5 |
Aggregate intrinsic value of options exercisable | $ | $ 22.3 |
Stock-Based Compensation - Narr
Stock-Based Compensation - Narrative (Details) $ in Millions | 3 Months Ended |
Mar. 31, 2024 USD ($) | |
Options to purchase common stock | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Total unrecognized compensation costs | $ 52.5 |
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 | $ 77.7 |
Unrecognized compensation costs, period for recognition (in years) | 3 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 | 3 Months Ended |
Mar. 31, 2024 USD ($) $ / shares shares | |
Number of Shares | |
Non-vested units as of the beginning of the period (in shares) | shares | 10,033 |
Granted (in shares) | shares | 3,251 |
Vested (in shares) | shares | (3,332) |
Forfeited (in shares) | shares | (393) |
Non-vested units as of the end of the period (in shares) | shares | 9,559 |
Weighted Average Grant Date Fair Value | |
Non-vested units as of the beginning of the period (in dollars per share) | $ / shares | $ 13.37 |
Granted (in dollars per share) | $ / shares | 15.35 |
Vested (in dollars per share) | $ / shares | 13.49 |
Forfeited (in dollars per share) | $ / shares | 14.66 |
Non-vested units as of the end of the period (in dollars per share) | $ / shares | $ 13.92 |
Weighted Average Remaining Years and Aggregate Intrinsic Value | |
Non-vested units, weighted average remaining years | 2 years 6 months |
Non-vested units, aggregate intrinsic value | $ | $ 112.6 |
Stock-Based Compensation - Expe
Stock-Based Compensation - Expense Summary (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total equity compensation expense | $ 30,803 | $ 34,894 |
Research and development expense | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total equity compensation expense | 4,871 | 8,490 |
Selling, general, and administrative expense | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total equity compensation expense | $ 25,932 | $ 26,404 |
Assets and Liabilities Measur_3
Assets and Liabilities Measured at Fair Value (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Assets: | ||
Fair value of assets | $ 36,892 | $ 46,686 |
Liabilities: | ||
Deferred compensation plan liability | 7,101 | 7,531 |
Fair value of liabilities | 7,101 | 7,531 |
Commercial paper | ||
Assets: | ||
Fair value of assets | 25,714 | 14,663 |
Treasury bill | ||
Assets: | ||
Fair value of assets | 12,946 | |
U.S. government agency bonds | ||
Assets: | ||
Fair value of assets | 3,977 | 11,446 |
Money market | ||
Assets: | ||
Fair value of assets | 7,201 | 7,631 |
Level 1 | ||
Assets: | ||
Fair value of assets | 7,201 | 7,631 |
Liabilities: | ||
Deferred compensation plan liability | 7,101 | 7,531 |
Fair value of liabilities | 7,101 | 7,531 |
Level 1 | Commercial paper | ||
Assets: | ||
Fair value of assets | 0 | 0 |
Level 1 | Treasury bill | ||
Assets: | ||
Fair value of assets | 0 | |
Level 1 | U.S. government agency bonds | ||
Assets: | ||
Fair value of assets | 0 | 0 |
Level 1 | Money market | ||
Assets: | ||
Fair value of assets | 7,201 | 7,631 |
Level 2 | ||
Assets: | ||
Fair value of assets | 29,691 | 39,055 |
Liabilities: | ||
Deferred compensation plan liability | 0 | 0 |
Fair value of liabilities | 0 | 0 |
Level 2 | Commercial paper | ||
Assets: | ||
Fair value of assets | 25,714 | 14,663 |
Level 2 | Treasury bill | ||
Assets: | ||
Fair value of assets | 12,946 | |
Level 2 | U.S. government agency bonds | ||
Assets: | ||
Fair value of assets | 3,977 | 11,446 |
Level 2 | Money market | ||
Assets: | ||
Fair value of assets | $ 0 | $ 0 |
Basic and Diluted Net Loss pe_3
Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Numerator: | ||
Net loss attributable to common stockholders | $ (48,419) | $ (52,932) |
Denominator: | ||
Weighted average common shares outstanding - basic (in shares) | 302,903,009 | 291,336,750 |
Weighted average common shares outstanding - diluted (in shares) | 302,903,009 | 291,336,750 |
Antidilutive securities | ||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 35,864,000 | 32,867,000 |
Options to purchase common stock | ||
Antidilutive securities | ||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 26,305,000 | 23,437,000 |
Unvested restricted stock units | ||
Antidilutive securities | ||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 9,559,000 | 9,430,000 |
Basic and Diluted Net Loss pe_4
Basic and Diluted Net Loss per Common Share - Narrative (Details) | Mar. 31, 2024 $ / shares |
Private Placement | |
Antidilutive securities | |
Warrants exercise price (in dollars per share) | $ 0.01 |