Cover Page
Cover Page - shares | 3 Months Ended | |
Mar. 31, 2022 | Apr. 27, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2022 | |
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 | 3675 Market Street, | |
Entity Address, City or Town | Philadelphia, | |
Entity Address, State or Province | PA | |
Entity Address, Postal Zip Code | 19104 | |
City Area Code | (215) | |
Local Phone Number | 921-7600 | |
Title of 12(b) Security | Common Stock, par value $0.01 per share | |
Trading Symbol | FOLD | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding (in shares) | 280,162,619 | |
Entity Central Index Key | 0001178879 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q1 |
Consolidated Balance Sheets (Un
Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 233,317 | $ 245,197 |
Investments in marketable securities | 177,878 | 237,299 |
Accounts receivable | 52,421 | 52,672 |
Inventories | 24,324 | 26,818 |
Prepaid expenses and other current assets | 30,960 | 34,848 |
Total current assets | 518,900 | 596,834 |
Operating lease right-of-use assets, net | 27,509 | 20,586 |
Property and equipment, less accumulated depreciation of $20,966 and $19,882 at March 31, 2022 and December 31, 2021, respectively | 34,544 | 42,496 |
In-process research & development | 23,000 | 23,000 |
Goodwill | 197,797 | 197,797 |
Other non-current assets | 25,188 | 24,427 |
Total Assets | 826,938 | 905,140 |
Current liabilities: | ||
Accounts payable | 22,914 | 21,513 |
Accrued expenses and other current liabilities | 80,379 | 98,153 |
Contingent consideration payable | 19,151 | 18,900 |
Operating lease liabilities | 7,255 | 7,409 |
Total current liabilities | 129,699 | 145,975 |
Deferred reimbursements | 5,906 | 5,906 |
Long-term debt | 389,994 | 389,357 |
Deferred income taxes | 4,930 | 4,930 |
Operating lease liabilities | 50,457 | 43,363 |
Other non-current liabilities | 7,335 | 8,240 |
Total liabilities | 588,321 | 597,771 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Common stock, $0.01 par value, 500,000,000 shares authorized, 280,133,856 and 278,912,800 shares issued and outstanding at March 31, 2022 and December 31, 2021, respectively | 2,809 | 2,808 |
Additional paid-in capital | 2,617,935 | 2,595,419 |
Accumulated other comprehensive (loss) gain: | ||
Foreign currency translation adjustment | (420) | 5,251 |
Unrealized loss on available-for-sale securities | (608) | (270) |
Warrants | 83 | 83 |
Accumulated deficit | (2,381,182) | (2,295,922) |
Total stockholders’ equity | 238,617 | 307,369 |
Total Liabilities and Stockholders’ Equity | $ 826,938 | $ 905,140 |
Consolidated Balance Sheets (_2
Consolidated Balance Sheets (Unaudited) (Parenthetical) $ in Thousands | Mar. 31, 2022USD ($)shares | Mar. 31, 2022$ / shares | Dec. 31, 2021USD ($)shares | Dec. 31, 2021$ / shares |
Statement of Financial Position [Abstract] | ||||
Accumulated depreciation of property and equipment | $ | $ 20,966 | $ 19,882 | ||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | ||
Common stock, shares authorized (in shares) | 500,000,000 | 500,000,000 | ||
Common stock, shares issued (in shares) | 280,133,856 | 278,912,800 | ||
Common stock, shares outstanding (in shares) | 280,133,856 | 278,912,800 |
Consolidated Statements of Oper
Consolidated Statements of Operations (Unaudited) $ in Thousands | 3 Months Ended | |||
Mar. 31, 2022USD ($)shares | Mar. 31, 2022$ / shares | Mar. 31, 2021USD ($)shares | Mar. 31, 2021$ / shares | |
Revenue: | ||||
Net product sales | $ 78,715 | $ 66,402 | ||
Cost of goods sold | 7,582 | 6,539 | ||
Gross profit | 71,133 | 59,863 | ||
Operating expenses: | ||||
Research and development | 81,517 | 64,117 | ||
Selling, general, and administrative | 58,116 | 46,726 | ||
Changes in fair value of contingent consideration payable | (1,188) | 471 | ||
Loss on impairment of assets | 6,616 | 0 | ||
Depreciation and amortization | 1,411 | 1,604 | ||
Total operating expenses | 146,472 | 112,918 | ||
Loss from operations | (75,339) | (53,055) | ||
Other (expense) income: | ||||
Interest income | 133 | 165 | ||
Interest expense | (8,147) | (7,992) | ||
Other income (expense) | 1,902 | (3,200) | ||
Loss before income tax | (81,451) | (64,082) | ||
Income tax expense | (3,809) | (1,582) | ||
Net loss attributable to common stockholders | $ (85,260) | $ (65,664) | ||
Net loss attributable to common stockholders per common share - basic (in dollars per share) | $ / shares | $ (0.30) | $ (0.25) | ||
Net loss attributable to common stockholders per common share — diluted (in dollars per share) | $ / shares | $ (0.30) | $ (0.25) | ||
Weighted average common shares outstanding - basic (in shares) | shares | 288,481,741 | 264,369,317 | ||
Weighted average number of shares outstanding - diluted (in shares) | shares | 288,481,741 | 264,369,317 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (85,260) | $ (65,664) |
Other comprehensive (loss) gain: | ||
Foreign currency translation adjustment (loss) gain | (5,671) | 608 |
Unrealized loss on available-for-sale securities | (338) | 0 |
Other comprehensive (loss) gain | (6,009) | 608 |
Comprehensive loss | $ (91,269) | $ (65,056) |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Warrants | Other Comprehensive Gain (Loss) | Accumulated Deficit |
Beginning balance (in shares) at Dec. 31, 2020 | 262,063,461 | |||||
Beginning balance at Dec. 31, 2020 | $ 286,380 | $ 2,650 | $ 2,308,578 | $ 12,387 | $ 8,227 | $ (2,045,462) |
Increase (Decrease) in Stockholders' Equity | ||||||
Stock options exercised, net (in shares) | 488,111 | |||||
Stock options exercised, net | 4,161 | $ 4 | 4,157 | |||
Vesting of restricted stock units, net of tax (in shares) | 897,063 | |||||
Vesting of restricted stock units, net of taxes | (14,194) | (14,194) | ||||
Stock-based compensation | 20,354 | 20,354 | ||||
Unrealized holding loss on available-for-sale securities | 0 | |||||
Foreign currency translation adjustment | 608 | 608 | ||||
Net loss | (65,664) | (65,664) | ||||
Warrants exercised (in shares) | 2,554,999 | |||||
Warrants exercised | 19,230 | $ 26 | 31,591 | (12,387) | ||
Equity component of the convertible notes (in shares) | 4,084 | |||||
Equity component of the convertible notes | 21 | 21 | ||||
Ending balance (in shares) at Mar. 31, 2021 | 266,007,718 | |||||
Ending balance at Mar. 31, 2021 | $ 250,896 | $ 2,680 | 2,350,507 | 0 | 8,835 | (2,111,126) |
Beginning balance (in shares) at Dec. 31, 2021 | 278,912,800 | 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) | 149,000 | 145,449 | ||||
Stock options exercised, net | $ 859 | $ 1 | 858 | |||
Vesting of restricted stock units, net of tax (in shares) | 1,075,607 | |||||
Vesting of restricted stock units, net of taxes | (8,993) | (8,993) | ||||
Stock-based compensation | 30,651 | 30,651 | ||||
Unrealized holding loss on available-for-sale securities | (338) | (338) | ||||
Foreign currency translation adjustment | (5,671) | (5,671) | ||||
Net loss | $ (85,260) | (85,260) | ||||
Ending balance (in shares) at Mar. 31, 2022 | 280,133,856 | 280,133,856 | ||||
Ending balance at Mar. 31, 2022 | $ 238,617 | $ 2,809 | $ 2,617,935 | $ 83 | $ (1,028) | $ (2,381,182) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Operating activities | ||
Net loss | $ (85,260) | $ (65,664) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Amortization of debt discount and deferred financing | 637 | 555 |
Depreciation and amortization | 1,411 | 1,604 |
Stock-based compensation | 30,651 | 20,354 |
Non-cash changes in the fair value of contingent consideration payable | (1,188) | 471 |
Foreign currency remeasurement loss | 673 | 2,846 |
Loss on impairment of assets | 6,616 | 0 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (1,000) | 839 |
Inventories | 1,812 | 741 |
Prepaid expenses and other current assets | 3,332 | 7,669 |
Accounts payable, accrued expenses, and other current liabilities | (14,563) | (40,191) |
Other non-current assets and liabilities | (1,436) | (1,578) |
Net cash used in operating activities | (58,315) | (72,354) |
Investing activities | ||
Sale and redemption of marketable securities | 108,328 | 163,680 |
Purchases of marketable securities | (49,244) | (76,247) |
Capital expenditures | (871) | (868) |
Net cash provided by investing activities | 58,213 | 86,565 |
Financing activities | ||
Payment of finance leases | (20) | (368) |
Proceeds from warrants exercised | 0 | 19,230 |
Purchase of vested restricted stock units, net of taxes | (8,993) | (14,194) |
Proceeds from stock options exercised, net | 859 | 4,161 |
Net cash (used in) provided by financing activities | (8,154) | 8,829 |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | (3,377) | (1,153) |
Net (decrease) increase in cash, cash equivalents, and restricted cash at the end of the period | (11,633) | 21,887 |
Cash, cash equivalents, and restricted cash at the beginning of period | 249,456 | 166,162 |
Cash, cash equivalents, and restricted cash at the end of period | 237,823 | 188,049 |
Supplemental disclosures of cash flow information | ||
Cash paid during the period for interest | 7,509 | 7,513 |
Capital expenditures unpaid at the end of period | 72 | 188 |
Cash paid for taxes | $ 456 | $ 2,472 |
Description of Business
Description of Business | 3 Months Ended |
Mar. 31, 2022 | |
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 a portfolio of product opportunities including the first, oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease, that is under review with the U.S. Food and Drug Administration ("FDA") as well as the European Medicines Agency ("EMA"). We are 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 United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan, with multiple additional approvals granted and applications pending in several geographies around the world. The lead biologics program of the Company's pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, two-component, potential best-in-class treatment for Pompe disease. In February 2019, the FDA granted Breakthrough Therapy designation ("BTD") to AT-GAA for the treatment of late onset Pompe disease. In September 2021, the FDA set the Prescription Drug User Fee Act ("PDUFA") target action date of May 29, 2022 for the New Drug Application ("NDA") for miglustat and July 29, 2022 for the Biologics License Application ("BLA") for cipaglucosidase alfa. The EMA validated the Marketing Authorization Application (“MAA”) in the fourth quarter of 2021. On May 9, 2022, the FDA extended the review period for the NDA for miglustat and the BLA for cipaglucosidase alfa resulting in revised PDUFA action dates of August 29, 2022 and October 29, 2022, respectively. The Company's operations have not been significantly impacted by the novel coronavirus (“COVID-19”) pandemic to date. The Company continued to observe increased lag times between patient identification and Galafold ® initiation due to the resurgence of COVID-19 in certain markets. The Company has maintained operations in all geographies, secured its global supply chain for its commercial and clinical products, as well as maintained the operational integrity of its clinical trials, with minimal disruptions. Whether the Company will continue to operate without any significant disruptions will depend on the continued health of its employees, the ongoing demand for Galafold ® and the continued operation of its global supply chain. The Company has continued to provide uninterrupted access to medicines for those in need of treatment, while prioritizing the health and safety of its global workforce. However, the Company's results of operations in future periods may be negatively impacted by unknown future impacts from the COVID-19 pandemic. The Company had an accumulated deficit of $2.4 billion as of March 31, 2022 and anticipates incurring losses through the fiscal year ending December 31, 2022 and beyond. The Company has historically funded its operations through stock offerings, Galafold ® revenues, debt issuances, collaborations, and other financing arrangements. 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 to achieve self-sustainability. Potential impacts of the COVID-19 pandemic, 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 | 3 Months Ended |
Mar. 31, 2022 | |
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 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, 2021. 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, 2021. 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. Additionally, the Company assessed the impact COVID-19 pandemic has had on its operations and financial results as of March 31, 2022 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses. 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 current assets and 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 related to its product sales of Galafold ® . The Company's accounts receivable at March 31, 2022 have arisen from product sales primarily in Europe and the U.S. 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 March 31, 2022, the Company recorded an allowance for doubtful accounts of $0.2 million. Property and Equipment Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are charged to income in the period in which the costs are incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy. The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is 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 long-lived asset over its estimated fair value in the period at which such a determination is made. During the three months ended March 31, 2022, in connection with the strategic prioritization of its gene therapy portfolio, the Company performed an assessment of its fixed assets. As a result, the Company recognized an impairment charge of $6.6 million. Revenue Recognition The Company's net product sales consist of sales of Galafold ® for the treatment of Fabry disease. The Company has recorded revenue on sales where Galafold ® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold ® 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 of Galafold ® . 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 of Galafold ® 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 from Galafold ® disaggregated by geographic area: Three Months Ended March 31, (in thousands) 2022 2021 U.S. $ 24,178 $ 20,853 Ex-U.S. 54,537 45,549 Total net product sales $ 78,715 $ 66,402 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 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. 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. |
Cash, Cash Equivalents, Marketa
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash | 3 Months Ended |
Mar. 31, 2022 | |
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, 2022, the Company held $233.3 million in cash and cash equivalents and $177.9 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 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, 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 many of these securities are either government backed or of the highest 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, 2022 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 233,317 $ — $ — $ 233,317 Commercial paper 132,131 — (311) 131,820 U.S. government agency bonds 35,229 — (78) 35,151 Asset-backed securities 6,013 — (15) 5,998 Corporate debt securities 4,514 — (6) 4,508 Money market 350 — — 350 Certificates of deposit 51 — — 51 $ 411,605 $ — $ (410) $ 411,195 Included in cash and cash equivalents $ 233,317 $ — $ — $ 233,317 Included in marketable securities 178,288 — (410) 177,878 Total cash, cash equivalents, and marketable securities $ 411,605 $ — $ (410) $ 411,195 As of December 31, 2021 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 245,197 $ — $ — $ 245,197 Commercial paper 174,578 7 (54) 174,531 Corporate debt securities 32,322 — (11) 32,311 Asset-backed securities 30,070 — (14) 30,056 Money market 350 — — 350 Certificate of deposit 51 — — 51 $ 482,568 $ 7 $ (79) $ 482,496 Included in cash and cash equivalents $ 245,197 $ — $ — $ 245,197 Included in marketable securities 237,371 7 (79) 237,299 Total cash, cash equivalents, and marketable securities $ 482,568 $ 7 $ (79) $ 482,496 For both the three months ended March 31, 2022 and the fiscal year ended December 31, 2021, 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, 2022 and December 31, 2021 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 fair value of these marketable securities in unrealized loss positions was $173.5 million and $173.4 million as of March 31, 2022 and December 31, 2021, 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) 2022 2021 Cash and cash equivalents $ 233,317 $ 184,833 Restricted cash 4,506 3,216 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 237,823 $ 188,049 |
Inventories
Inventories | 3 Months Ended |
Mar. 31, 2022 | |
Inventory Disclosure [Abstract] | |
Inventories | Inventories Inventories consist of raw materials, work-in-process, and finished goods related to the manufacture of Galafold ® . The following table summarizes the components of inventories: (in thousands) March 31, 2022 December 31, 2021 Raw materials $ 12,504 $ 12,289 Work-in-process 8,526 10,699 Finished goods 3,294 3,830 Total inventories $ 24,324 $ 26,818 The Company recorded a reserve for inventory of $1.2 million and $1.1 million as of March 31, 2022 and December 31, 2021, respectively. |
Debt
Debt | 3 Months Ended |
Mar. 31, 2022 | |
Debt Disclosure [Abstract] | |
Debt | Debt The Company's debt consists of the following: (in thousands) March 31, 2022 December 31, 2021 Senior Secured Term Loan due 2026: Principal $ 400,000 $ 400,000 Less: debt discount (1) (5,710) (6,074) Less: deferred financing (1) (4,296) (4,569) Net carrying value of Long-term debt $ 389,994 $ 389,357 ______________________________ (1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan 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, 2022 and 2021, respectively: Three Months Ended March 31, (in thousands) 2022 2021 Contractual interest expense $ 7,500 $ 7,521 Amortization of debt discount $ 364 $ 332 Amortization of deferred financing $ 273 $ 223 |
Share-Based Compensation
Share-Based Compensation | 3 Months Ended |
Mar. 31, 2022 | |
Share-based Payment Arrangement [Abstract] | |
Share-Based Compensation | Share-Based CompensationThe 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 March 31, 2022 2021 Expected stock price volatility 62.3 % 66.7 % Risk free interest rate 1.5 % 0.4 % Expected life of options (years) 5.3 5.4 Expected annual dividend per share $ — $ — A summary of the Company's stock options for the three months ended March 31, 2022 were as follows: Number of Weighted Average Exercise Weighted Average Remaining Aggregate (in thousands) (in millions) Options outstanding, December 31, 2021 14,731 $ 11.08 Granted 4,892 $ 11.89 Exercised (149) $ 6.00 Forfeited (127) $ 12.57 Expired (43) $ 11.74 Options outstanding, March 31, 2022 19,304 $ 11.31 7.1 $ 12.3 Vested and unvested expected to vest, March 31, 2022 17,174 $ 11.16 6.8 $ 12.2 Exercisable at March 31, 2022 9,932 $ 10.20 5.1 $ 12.0 As of March 31, 2022, the total unrecognized compensation cost related to non-vested stock options granted was $42.3 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, 2022 is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Non-vested units as of December 31, 2021 7,341 $ 13.90 Granted 4,633 $ 12.12 Vested (1,538) $ 12.70 Forfeited (85) $ 12.57 Non-vested units as of March 31, 2022 10,351 $ 13.16 2.7 $ 98.0 All non-vested units are expected to vest over their normal term. As of March 31, 2022, there was $73.0 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 Consolidated Statements of Operations related to the equity awards: Three Months Ended March 31, (in thousands) 2022 2021 Research and development expense $ 9,365 $ 6,305 Selling, general, and administrative expense 21,286 14,049 Total equity compensation expense $ 30,651 $ 20,354 |
Assets and Liabilities Measured
Assets and Liabilities Measured at Fair Value | 3 Months Ended |
Mar. 31, 2022 | |
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, 2022 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 131,820 $ 131,820 U.S. government agency bonds 35,151 35,151 Asset-backed securities 5,998 5,998 Money market 5,755 5,755 Corporate debt securities 4,508 4,508 $ 183,232 $ 183,232 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 19,151 $ 19,151 Deferred compensation plan liability 5,405 — 5,405 $ 5,405 $ 19,151 $ 24,556 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, 2021 are identified in the following tables: (in thousands) Level 2 Total Assets: Corporate debt securities $ 174,531 $ 174,531 Commercial paper 32,311 32,311 Asset-backed securities 30,056 30,056 Money market funds 5,150 5,150 $ 242,048 $ 242,048 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 20,339 $ 20,339 Deferred compensation plan liability 4,800 — 4,800 $ 4,800 $ 20,339 $ 25,139 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. The Company did not have any Level 3 assets as of March 31, 2022 or December 31, 2021. 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 most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Gains and losses are included in the Consolidated Statements of Operations. The contingent consideration payable for Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approach, the estimated fair value could be significantly higher or lower than the fair value the Company determined. The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program: Contingent Consideration Liability Fair Value as of March 31, 2022 Valuation Technique Unobservable Input Range (in thousands) Discount rate 7.5% Clinical and regulatory milestones $ 19,151 Probability weighted discounted cash flow Probability of achievement of milestones 75% - 88% Projected year of payments 2022 - 2023 Contingent consideration liabilities are remeasured to fair value each reporting period using discount rates, probabilities of payment, and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met. The following table shows the change in the balance of contingent consideration payable for the three months ended March 31, 2022 and 2021, respectively: Three Months Ended March 31, (in thousands) 2022 2021 Balance, beginning of the period $ 20,339 $ 25,825 Changes in fair value during the period, included in the Consolidated Statements of Operations (1,188) 471 Balance, end of the period (1) $ 19,151 $ 26,296 ______________________________ (1) As certain milestones are expected to be reached within the next twelve months, the March 31, 2022 balance was recorded as a current liability in the Consolidated Balance Sheets. |
Basic and Diluted Net Loss per
Basic and Diluted Net Loss per Common Share | 3 Months Ended |
Mar. 31, 2022 | |
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) 2022 2021 Numerator: Net loss attributable to common stockholders $ (85,260) $ (65,664) Denominator: Weighted average common shares outstanding — basic and diluted 288,481,741 264,369,317 Dilutive common stock equivalents would include the dilutive effect of common stock options, convertible debt units, RSUs, and warrants for common stock equivalents. 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) 2022 2021 Options to purchase common stock 19,304 15,084 Unvested restricted stock units 10,351 7,466 Convertible notes — 458 Total number of potentially issuable shares 29,655 23,008 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2022 | |
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 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. Additionally, the Company assessed the impact COVID-19 pandemic has had on its operations and financial results as of March 31, 2022 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses. |
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 related to its product sales of Galafold ® |
Property and Equipment | Property and Equipment Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are charged to income in the period in which the costs are incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy. The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is 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 long-lived asset over its estimated fair value in the period at which such a determination is made. |
Revenue Recognition | Revenue Recognition The Company's net product sales consist of sales of Galafold ® for the treatment of Fabry disease. The Company has recorded revenue on sales where Galafold ® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold ® 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 of Galafold ® . 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 of Galafold ® 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 Consolidated Statements of Operations. |
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) | 3 Months Ended |
Mar. 31, 2022 | |
Accounting Policies [Abstract] | |
Disaggregation of Revenue | The following table summarizes the Company's net product sales from Galafold ® disaggregated by geographic area: Three Months Ended March 31, (in thousands) 2022 2021 U.S. $ 24,178 $ 20,853 Ex-U.S. 54,537 45,549 Total net product sales $ 78,715 $ 66,402 |
Cash, Cash Equivalents, Marke_2
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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, 2022 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 233,317 $ — $ — $ 233,317 Commercial paper 132,131 — (311) 131,820 U.S. government agency bonds 35,229 — (78) 35,151 Asset-backed securities 6,013 — (15) 5,998 Corporate debt securities 4,514 — (6) 4,508 Money market 350 — — 350 Certificates of deposit 51 — — 51 $ 411,605 $ — $ (410) $ 411,195 Included in cash and cash equivalents $ 233,317 $ — $ — $ 233,317 Included in marketable securities 178,288 — (410) 177,878 Total cash, cash equivalents, and marketable securities $ 411,605 $ — $ (410) $ 411,195 As of December 31, 2021 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 245,197 $ — $ — $ 245,197 Commercial paper 174,578 7 (54) 174,531 Corporate debt securities 32,322 — (11) 32,311 Asset-backed securities 30,070 — (14) 30,056 Money market 350 — — 350 Certificate of deposit 51 — — 51 $ 482,568 $ 7 $ (79) $ 482,496 Included in cash and cash equivalents $ 245,197 $ — $ — $ 245,197 Included in marketable securities 237,371 7 (79) 237,299 Total cash, cash equivalents, and marketable securities $ 482,568 $ 7 $ (79) $ 482,496 |
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) 2022 2021 Cash and cash equivalents $ 233,317 $ 184,833 Restricted cash 4,506 3,216 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 237,823 $ 188,049 |
Inventories (Tables)
Inventories (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventories for the Period | The following table summarizes the components of inventories: (in thousands) March 31, 2022 December 31, 2021 Raw materials $ 12,504 $ 12,289 Work-in-process 8,526 10,699 Finished goods 3,294 3,830 Total inventories $ 24,324 $ 26,818 |
Debt (Tables)
Debt (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Debt Disclosure [Abstract] | |
Schedule of Liability Components of Long-Term Debt | The Company's debt consists of the following: (in thousands) March 31, 2022 December 31, 2021 Senior Secured Term Loan due 2026: Principal $ 400,000 $ 400,000 Less: debt discount (1) (5,710) (6,074) Less: deferred financing (1) (4,296) (4,569) Net carrying value of Long-term debt $ 389,994 $ 389,357 ______________________________ (1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan using the effective interest rate method. |
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, 2022 and 2021, respectively: Three Months Ended March 31, (in thousands) 2022 2021 Contractual interest expense $ 7,500 $ 7,521 Amortization of debt discount $ 364 $ 332 Amortization of deferred financing $ 273 $ 223 |
Share-Based Compensation (Table
Share-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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 March 31, 2022 2021 Expected stock price volatility 62.3 % 66.7 % Risk free interest rate 1.5 % 0.4 % Expected life of options (years) 5.3 5.4 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, 2022 were as follows: Number of Weighted Average Exercise Weighted Average Remaining Aggregate (in thousands) (in millions) Options outstanding, December 31, 2021 14,731 $ 11.08 Granted 4,892 $ 11.89 Exercised (149) $ 6.00 Forfeited (127) $ 12.57 Expired (43) $ 11.74 Options outstanding, March 31, 2022 19,304 $ 11.31 7.1 $ 12.3 Vested and unvested expected to vest, March 31, 2022 17,174 $ 11.16 6.8 $ 12.2 Exercisable at March 31, 2022 9,932 $ 10.20 5.1 $ 12.0 |
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, 2022 is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Non-vested units as of December 31, 2021 7,341 $ 13.90 Granted 4,633 $ 12.12 Vested (1,538) $ 12.70 Forfeited (85) $ 12.57 Non-vested units as of March 31, 2022 10,351 $ 13.16 2.7 $ 98.0 |
Schedule of Equity Compensation Expenses | The following table summarizes information related to compensation expense recognized in the Consolidated Statements of Operations related to the equity awards: Three Months Ended March 31, (in thousands) 2022 2021 Research and development expense $ 9,365 $ 6,305 Selling, general, and administrative expense 21,286 14,049 Total equity compensation expense $ 30,651 $ 20,354 |
Assets and Liabilities Measur_2
Assets and Liabilities Measured at Fair Value (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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, 2022 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 131,820 $ 131,820 U.S. government agency bonds 35,151 35,151 Asset-backed securities 5,998 5,998 Money market 5,755 5,755 Corporate debt securities 4,508 4,508 $ 183,232 $ 183,232 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 19,151 $ 19,151 Deferred compensation plan liability 5,405 — 5,405 $ 5,405 $ 19,151 $ 24,556 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, 2021 are identified in the following tables: (in thousands) Level 2 Total Assets: Corporate debt securities $ 174,531 $ 174,531 Commercial paper 32,311 32,311 Asset-backed securities 30,056 30,056 Money market funds 5,150 5,150 $ 242,048 $ 242,048 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 20,339 $ 20,339 Deferred compensation plan liability 4,800 — 4,800 $ 4,800 $ 20,339 $ 25,139 |
Schedule of Significant Unobservable Inputs Used in the Valuation of the Contingent Consideration Payable | The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program: Contingent Consideration Liability Fair Value as of March 31, 2022 Valuation Technique Unobservable Input Range (in thousands) Discount rate 7.5% Clinical and regulatory milestones $ 19,151 Probability weighted discounted cash flow Probability of achievement of milestones 75% - 88% Projected year of payments 2022 - 2023 |
Schedule of Changes in Continent Consideration Payable | The following table shows the change in the balance of contingent consideration payable for the three months ended March 31, 2022 and 2021, respectively: Three Months Ended March 31, (in thousands) 2022 2021 Balance, beginning of the period $ 20,339 $ 25,825 Changes in fair value during the period, included in the Consolidated Statements of Operations (1,188) 471 Balance, end of the period (1) $ 19,151 $ 26,296 ______________________________ (1) As certain milestones are expected to be reached within the next twelve months, the March 31, 2022 balance was recorded as a current liability in the Consolidated Balance Sheets. |
Basic and Diluted Net Loss pe_2
Basic and Diluted Net Loss per Common Share (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
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) 2022 2021 Numerator: Net loss attributable to common stockholders $ (85,260) $ (65,664) Denominator: Weighted average common shares outstanding — basic and diluted 288,481,741 264,369,317 |
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) 2022 2021 Options to purchase common stock 19,304 15,084 Unvested restricted stock units 10,351 7,466 Convertible notes — 458 Total number of potentially issuable shares 29,655 23,008 |
Description of Business (Detail
Description of Business (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Accumulated deficit | $ 2,381,182 | $ 2,295,922 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Allowance for doubtful accounts receivable | $ 200 | |
Loss on impairment of assets | 6,616 | $ 0 |
Net product sales | $ 78,715 | 66,402 |
Minimum | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Estimated useful life | 3 years | |
Maximum | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Estimated useful life | 5 years | |
U.S. | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Net product sales | $ 24,178 | 20,853 |
Ex-U.S. | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Net product sales | $ 54,537 | $ 45,549 |
Cash, Cash Equivalents, Marke_3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2021 | |
Cash, Money Market Funds, and Marketable Securities | |||
Cash and cash equivalents | $ 233,317,000 | $ 245,197,000 | $ 184,833,000 |
Available-for-sale debt securities | 177,878,000 | 237,299,000 | |
Realized gain (loss) on debt securities | 0 | 0 | |
Fair value of available-for-sale debt securities in unrealized loss positions | 173,500,000 | $ 173,400,000 | |
Marketable Securities | |||
Cash, Money Market Funds, and Marketable Securities | |||
Available-for-sale debt securities | $ 177,900,000 |
Cash, Cash Equivalents, Marke_4
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Components (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2021 |
Cash, Money Market Funds, and Marketable Securities | |||
Cash and cash equivalents | $ 233,317 | $ 245,197 | $ 184,833 |
Fair value, cash balances | 233,317 | 245,197 | |
Cost, available-for-sale securities | 178,288 | 237,371 | |
Gross unrealized gain, available-for-sale securities | 0 | 7 | |
Gross unrealized loss, available-for-sale securities | (410) | (79) | |
Fair value, available-for-sale debt securities | 177,878 | 237,299 | |
Cost, cash balances and available-for-sale securities | 411,605 | 482,568 | |
Gross unrealized gain, cash balances and available-for-sale securities | 0 | 7 | |
Gross unrealized loss, cash balances and available-for-sale securities | (410) | (79) | |
Fair value, cash balances and available-for-sale securities | 411,195 | 482,496 | |
Commercial paper | |||
Cash, Money Market Funds, and Marketable Securities | |||
Corporate debt securities, cost | 132,131 | ||
Corporate debt securities, gross unrealized gain | 0 | ||
Corporate debt securities, gross unrealized loss | (311) | ||
Corporate debt securities, fair value | 131,820 | ||
Cost, available-for-sale securities | 174,578 | ||
Gross unrealized gain, available-for-sale securities | 7 | ||
Gross unrealized loss, available-for-sale securities | (54) | ||
Fair value, available-for-sale debt securities | 174,531 | ||
U.S. government agency bonds | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 35,229 | ||
Gross unrealized gain, available-for-sale securities | 0 | ||
Gross unrealized loss, available-for-sale securities | (78) | ||
Fair value, available-for-sale debt securities | 35,151 | ||
Asset-backed securities | |||
Cash, Money Market Funds, and Marketable Securities | |||
Corporate debt securities, cost | 6,013 | 30,070 | |
Corporate debt securities, gross unrealized gain | 0 | 0 | |
Corporate debt securities, gross unrealized loss | (15) | (14) | |
Corporate debt securities, fair value | 5,998 | 30,056 | |
Corporate debt securities | |||
Cash, Money Market Funds, and Marketable Securities | |||
Corporate debt securities, cost | 4,514 | 32,322 | |
Corporate debt securities, gross unrealized gain | 0 | 0 | |
Corporate debt securities, gross unrealized loss | (6) | (11) | |
Corporate debt securities, fair value | 4,508 | 32,311 | |
Money market | |||
Cash, Money Market Funds, and Marketable Securities | |||
Cost, available-for-sale securities | 350 | 350 | |
Gross unrealized gain, available-for-sale securities | 0 | 0 | |
Gross unrealized loss, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | 350 | 350 | |
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 | |
Gross unrealized loss, available-for-sale securities | 0 | 0 | |
Fair value, available-for-sale debt securities | $ 51 | $ 51 |
Cash, Cash Equivalents, Marke_5
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash, Cash Equivalents, And Restricted Cash (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2021 | Dec. 31, 2020 |
Cash, Cash Equivalents, and Short-term Investments [Abstract] | ||||
Cash and cash equivalents | $ 233,317 | $ 245,197 | $ 184,833 | |
Restricted cash | 4,506 | 3,216 | ||
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows | $ 237,823 | $ 249,456 | $ 188,049 | $ 166,162 |
Inventories (Details)
Inventories (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 12,504 | $ 12,289 |
Work-in-process | 8,526 | 10,699 |
Finished goods | 3,294 | 3,830 |
Total inventories | 24,324 | 26,818 |
Reserve for inventory | $ 1,200 | $ 1,100 |
Debt - Summary of Long Term Deb
Debt - Summary of Long Term Debt (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Debt Instrument [Line Items] | ||
Net carrying value | $ 389,994 | $ 389,357 |
Senior Loans | Senior Secured Term Loan due 2026 | ||
Debt Instrument [Line Items] | ||
Principal | 400,000 | 400,000 |
Less: debt discount | (5,710) | (6,074) |
Less: deferred financing | $ (4,296) | $ (4,569) |
Debt - Interest Expense (Detail
Debt - Interest Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Debt Disclosure [Abstract] | ||
Contractual interest expense | $ 7,500 | $ 7,521 |
Amortization of debt discount | 364 | 332 |
Amortization of deferred financing | $ 273 | $ 223 |
Share-Based Compensation - Weig
Share-Based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected stock price volatility | 62.30% | 66.70% |
Risk free interest rate | 1.50% | 0.40% |
Expected life of options (years) | 5 years 3 months 18 days | 5 years 4 months 24 days |
Expected annual dividend per share (in dollars per share) | $ 0 | $ 0 |
Share-Based Compensation - Stoc
Share-Based Compensation - Stock Option Activity (Details) - 3 months ended Mar. 31, 2022 shares in Thousands, $ in Millions | $ / sharesshares | USD ($)shares |
Number of Shares | ||
Options outstanding (in shares) | shares | 14,731 | |
Options, granted (in shares) | shares | 4,892 | |
Options, exercised (in shares) | shares | (149) | |
Options, forfeited (in shares) | shares | (127) | |
Options, expired (in shares) | shares | (43) | |
Options outstanding (in shares) | shares | 19,304 | |
Options, vested and unvested expected to vest (in shares) | shares | 17,174 | |
Options, exercisable (in shares) | shares | 9,932 | |
Weighted Average Exercise Price | ||
Options outstanding (in dollars per share) | $ / shares | $ 11.08 | |
Options, granted (in dollars per share) | $ / shares | 11.89 | |
Options, exercised (in dollars per share) | $ / shares | 6 | |
Options, forfeited (in dollars per share) | $ / shares | 12.57 | |
Options, expired (in dollars per share) | $ / shares | 11.74 | |
Options outstanding (in dollars per share) | $ / shares | 11.31 | |
Options, vested and unvested expected to vest (in dollars per share) | $ / shares | 11.16 | |
Options, exercisable (in dollars per share) | $ / shares | $ 10.20 | |
Additional Disclosures | ||
Weighted average remaining contractual life, options outstanding | 7 years 1 month 6 days | |
Weighted average remaining contractual life, options vested and unvested expected to vest | 6 years 9 months 18 days | |
Weighted average remaining contractual life, options exercisable | 5 years 1 month 6 days | |
Aggregate intrinsic value, options outstanding | $ | $ 12.3 | |
Aggregate intrinsic value, vested and unvested expected to vest | $ | 12.2 | |
Aggregate intrinsic value, exercisable | $ | $ 12 |
Share-Based Compensation - Narr
Share-Based Compensation - Narrative (Details) $ in Millions | 3 Months Ended |
Mar. 31, 2022USD ($) | |
Options to purchase common stock | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Total unrecognized compensation costs | $ 42.3 |
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 | $ 73 |
Unrecognized compensation costs, period for recognition (in years) | 3 years |
Share-Based Compensation - RSUs
Share-Based Compensation - RSUs and PBRSUs Summary (Details) - Unvested restricted stock units shares in Thousands, $ in Millions | 3 Months Ended |
Mar. 31, 2022USD ($)$ / sharesshares | |
Number of Shares | |
Non-vested units (in shares) | shares | 7,341 |
Granted (in shares) | shares | 4,633 |
Vested (in shares) | shares | (1,538) |
Forfeited (in shares) | shares | (85) |
Non-vested units (in shares) | shares | 10,351 |
Weighted Average Grant Date Fair Value | |
Non-vested units (in dollars per share) | $ / shares | $ 13.90 |
Granted (in dollars per share) | $ / shares | 12.12 |
Vested (in dollars per share) | $ / shares | 12.70 |
Forfeited (in dollars per share) | $ / shares | 12.57 |
Non-vested units (in dollars per share) | $ / shares | $ 13.16 |
Additional Disclosures | |
Non-vested units, weighted average remaining years | 2 years 8 months 12 days |
Non-vested units, aggregate intrinsic value | $ | $ 98 |
Share-Based Compensation - Expe
Share-Based Compensation - Expense Summary (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total equity compensation expense | $ 30,651 | $ 20,354 |
Research and development expense | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total equity compensation expense | 9,365 | 6,305 |
Selling, general, and administrative expense | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Total equity compensation expense | $ 21,286 | $ 14,049 |
Assets and Liabilities Measur_3
Assets and Liabilities Measured at Fair Value - Components by Hierarchy Level (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Assets: | ||
Fair value of assets | $ 183,232 | $ 242,048 |
Liabilities: | ||
Contingent consideration payable | 19,151 | 20,339 |
Deferred compensation plan liability | 5,405 | 4,800 |
Fair value of liabilities | 24,556 | 25,139 |
Commercial paper | ||
Assets: | ||
Fair value of assets | 131,820 | 32,311 |
U.S. government agency bonds | ||
Assets: | ||
Fair value of assets | 35,151 | |
Asset-backed securities | ||
Assets: | ||
Fair value of assets | 5,998 | 30,056 |
Money market | ||
Assets: | ||
Fair value of assets | 5,755 | 5,150 |
Corporate debt securities | ||
Assets: | ||
Fair value of assets | 4,508 | 174,531 |
Level 2 | ||
Assets: | ||
Fair value of assets | 183,232 | 242,048 |
Liabilities: | ||
Contingent consideration payable | 0 | 0 |
Deferred compensation plan liability | 5,405 | 4,800 |
Fair value of liabilities | 5,405 | 4,800 |
Level 2 | Commercial paper | ||
Assets: | ||
Fair value of assets | 131,820 | 32,311 |
Level 2 | U.S. government agency bonds | ||
Assets: | ||
Fair value of assets | 35,151 | |
Level 2 | Asset-backed securities | ||
Assets: | ||
Fair value of assets | 5,998 | 30,056 |
Level 2 | Money market | ||
Assets: | ||
Fair value of assets | 5,755 | 5,150 |
Level 2 | Corporate debt securities | ||
Assets: | ||
Fair value of assets | 4,508 | 174,531 |
Level 3 | ||
Liabilities: | ||
Contingent consideration payable | 19,151 | 20,339 |
Deferred compensation plan liability | 0 | 0 |
Fair value of liabilities | $ 19,151 | $ 20,339 |
Assets and Liabilities Measur_4
Assets and Liabilities Measured at Fair Value - Significant Unobservable Inputs (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Contingent consideration payable | $ 19,151 | $ 20,339 |
Discount rate | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Contingent consideration payable | 19,151 | $ 20,339 |
Callidus Biopharma Inc | Clinical and regulatory milestones | ATB200 Pompe Program | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Contingent consideration payable | $ 19,151 | |
Callidus Biopharma Inc | Clinical and regulatory milestones | Probability of achievement of milestones | ATB200 Pompe Program | Minimum | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Contingent consideration, measurement input | 75.00% | |
Callidus Biopharma Inc | Clinical and regulatory milestones | Probability of achievement of milestones | ATB200 Pompe Program | Maximum | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Contingent consideration, measurement input | 88.00% | |
Callidus Biopharma Inc | Clinical and regulatory milestones | Discount rate | Discount rate | ATB200 Pompe Program | Probability weighted discounted cash flow | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Contingent consideration, measurement input | 7.50% |
Assets and Liabilities Measur_5
Assets and Liabilities Measured at Fair Value - Contingent Consideration Roll Forward (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | ||
Balance, beginning of the period | $ 20,339 | $ 25,825 |
Changes in fair value during the period, included in the Consolidated Statements of Operations | (1,188) | 471 |
Balance, end of the period | $ 19,151 | $ 26,296 |
Basic and Diluted Net Loss pe_3
Basic and Diluted Net Loss per Common Share (Details) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2022USD ($)shares | Mar. 31, 2021USD ($)shares | Mar. 31, 2022$ / shares | |
Numerator: | |||
Net loss attributable to common stockholders | $ | $ (85,260) | $ (65,664) | |
Denominator: | |||
Weighted average common shares outstanding - basic (in shares) | 288,481,741 | 264,369,317 | |
Weighted average number of shares outstanding - diluted (in shares) | 288,481,741 | 264,369,317 | |
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 29,655,000 | 23,008,000 | |
Private Placement | |||
Denominator: | |||
Warrants exercise price (in dollars per share) | $ / shares | $ 0.01 | ||
Options to purchase common stock | |||
Denominator: | |||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 19,304,000 | 15,084,000 | |
Unvested restricted stock units | |||
Denominator: | |||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 10,351,000 | 7,466,000 | |
Convertible notes | |||
Denominator: | |||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 0 | 458,000 |