Cover
Cover - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Feb. 19, 2021 | Jun. 30, 2020 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2020 | ||
Current Fiscal Year End Date | --12-31 | ||
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 Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
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 | ||
ICFR Auditor Attestation Flag | true | ||
Entity Shell Company | false | ||
Entity Public Float | $ 3,851,129,284 | ||
Entity Common Stock, Shares Outstanding | 264,316,074 | ||
Documents Incorporated by Reference | Portions of the Proxy Statement for the registrant's 2021 Annual Meeting of Stockholders which is to be filed subsequent to the date hereof are incorporated by reference into Part III of this Annual Report on Form 10-K. | ||
Entity Central Index Key | 0001178879 | ||
Amendment Flag | false | ||
Document Fiscal Year Focus | 2020 | ||
Document Fiscal Period Focus | FY |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 163,240 | $ 142,837 |
Investments in marketable securities | 320,029 | 309,903 |
Accounts receivable | 46,923 | 33,284 |
Inventories | 19,556 | 14,041 |
Prepaid expenses and other current assets | 29,721 | 20,008 |
Total current assets | 579,469 | 520,073 |
Operating lease right-of-use assets, less accumulated amortization of $7,574 and $5,342 at December 31, 2020 and December 31, 2019, respectively | 23,296 | 33,315 |
Property and equipment, less accumulated depreciation of $14,487 and $17,604 at December 31, 2020 and December 31, 2019, respectively | 43,863 | 47,705 |
In-process research & development | 23,000 | 23,000 |
Goodwill | 197,797 | 197,797 |
Other non-current assets | 19,095 | 28,317 |
Total Assets | 886,520 | 850,207 |
Current liabilities: | ||
Accounts payable | 17,063 | 21,722 |
Accrued expenses and other current liabilities | 96,841 | 99,901 |
Contingent consideration payable | 8,900 | 0 |
Operating lease liabilities | 6,872 | 7,189 |
Total current liabilities | 129,676 | 128,812 |
Deferred reimbursements | 7,406 | 8,906 |
Long-term debt | 389,254 | 149,505 |
Contingent consideration payable | 16,925 | 22,681 |
Deferred income taxes | 4,896 | 5,051 |
Operating lease liabilities | 45,604 | 53,531 |
Other non-current liabilities | 6,379 | 5,296 |
Total liabilities | 600,140 | 373,782 |
Commitments and contingencies | ||
Stockholders' equity: | ||
Common stock, $0.01 par value, 500,000,000 shares authorized, 262,063,461 and 255,417,869 shares issued and outstanding at December 31, 2020 and December 31, 2019, respectively | 2,650 | 2,598 |
Additional paid-in capital | 2,308,578 | 2,227,225 |
Accumulated other comprehensive loss: | ||
Foreign currency translation adjustment | 8,412 | 2,785 |
Unrealized (loss) gain on available-for securities | (185) | 40 |
Warrants | 12,387 | 12,387 |
Accumulated deficit | (2,045,462) | (1,768,610) |
Total stockholders' equity | 286,380 | 476,425 |
Total Liabilities and Stockholders' Equity | $ 886,520 | $ 850,207 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Statement of Financial Position [Abstract] | ||
Accumulated depreciation and amortization, operating lease right-of-use assets | $ 7,574 | $ 5,342 |
Accumulated depreciation of property and equipment | $ 14,487 | $ 17,604 |
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) | 262,063,461 | 255,417,869 |
Common stock, shares outstanding (in shares) | 262,063,461 | 255,417,869 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Income Statement [Abstract] | |||
Net product sales | $ 260,886 | $ 182,237 | $ 91,245 |
Cost of goods sold | 31,044 | 21,963 | 14,404 |
Gross profit | 229,842 | 160,274 | 76,841 |
Operating expenses: | |||
Research and development | 308,443 | 286,378 | 270,902 |
Selling, general, and administrative | 156,407 | 169,861 | 127,200 |
Changes in fair value of contingent consideration payable | 3,144 | 3,297 | 3,300 |
Depreciation and amortization | 8,846 | 4,775 | 4,216 |
Total operating expenses | 476,840 | 464,311 | 405,618 |
Loss from operations | (246,998) | (304,037) | (328,777) |
Other (expense) income: | |||
Interest income | 3,226 | 10,249 | 10,461 |
Interest expense | (22,425) | (18,872) | (22,402) |
Loss on exchange of convertible notes | 0 | (40,624) | 0 |
Loss on extinguishment of debt | (7,276) | 0 | 0 |
Change in fair value of derivatives | 0 | 0 | (2,739) |
Other expense | (781) | (2,626) | (5,632) |
Loss before income tax | (274,254) | (355,910) | (349,089) |
Income tax (expense) benefit | (2,598) | (478) | 94 |
Net loss attributable to common stockholders | $ (276,852) | $ (356,388) | $ (348,995) |
Net loss attributable to common stockholders per common share - basic and diluted (in dollars per share) | $ (1.07) | $ (1.48) | $ (1.88) |
Weighted-average common shares outstanding - basic and diluted (in shares) | 258,867,380 | 240,421,001 | 185,790,021 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Statement of Comprehensive Income [Abstract] | |||
Net loss | $ (276,852) | $ (356,388) | $ (348,995) |
Other comprehensive gain: | |||
Foreign currency translation adjustment gain, net of tax impact of $1,552, $889, and $0, respectively | 5,627 | 2,290 | 2,537 |
Unrealized (loss) gain on available-for-sale securities, net of tax impact of $(50), $182, and $0, respectively | (225) | 467 | 9 |
Other comprehensive income | 5,402 | 2,757 | 2,546 |
Comprehensive loss | $ (271,450) | $ (353,631) | $ (346,449) |
Consolidated Statements of Co_2
Consolidated Statements of Comprehensive Loss (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Statement of Comprehensive Income [Abstract] | |||
Foreign currency translation gain (loss), tax impact | $ 1,552 | $ 889 | $ 0 |
Available-for-sale securities, tax expense (benefit) | $ (50) | $ 182 | $ 0 |
Consolidated Statements of Chan
Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands | Total | Cumulative Effect, Period of Adoption, Adjustment | Common Stock | Additional Paid-In Capital | Warrants | Other Comprehensive Gain (Loss) | Other Comprehensive Gain (Loss)Cumulative Effect, Period of Adoption, Adjustment | Accumulated Deficit | Accumulated DeficitCumulative Effect, Period of Adoption, Adjustment |
Balance (in shares) at Dec. 31, 2017 | 166,989,790 | ||||||||
Balance at Dec. 31, 2017 | $ 352,850 | $ 0 | $ 1,721 | $ 1,400,758 | $ 16,076 | $ (2,095) | $ (383) | $ (1,063,610) | $ 383 |
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock issued from exercise of stock options, net (in shares) | 1,397,908 | ||||||||
Stock issued from exercise of stock options, net | 9,144 | $ 14 | 9,130 | ||||||
Stock issued from equity financing (in shares) | 20,239,839 | ||||||||
Stock issued from equity financing | 294,583 | $ 202 | 294,381 | ||||||
Restricted stock tax vesting (in shares) | 303,173 | ||||||||
Restricted stock tax vesting | (2,832) | (2,832) | |||||||
Stock-based compensation | 29,260 | 29,260 | |||||||
Warrants exercised (in shares) | 453,214 | ||||||||
Warrants exercised | 3,617 | $ 5 | 6,625 | (3,013) | |||||
Change in fair value of derivatives | 2,739 | 2,739 | |||||||
Unrealized holding gain on available-for-sale securities | 9 | 9 | |||||||
Foreign currency translation adjustment | 2,537 | 2,537 | |||||||
Net loss | (348,995) | (348,995) | |||||||
Balance (in shares) at Dec. 31, 2018 | 189,383,924 | ||||||||
Balance at Dec. 31, 2018 | 342,912 | $ 1,942 | 1,740,061 | 13,063 | 68 | (1,412,222) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock issued from exercise of stock options, net (in shares) | 1,967,087 | ||||||||
Stock issued from exercise of stock options, net | 11,476 | $ 20 | 11,456 | ||||||
Stock issued from equity financing (in shares) | 18,720,930 | ||||||||
Stock issued from equity financing | 188,994 | $ 187 | 188,807 | ||||||
Restricted stock tax vesting (in shares) | 477,198 | ||||||||
Restricted stock tax vesting | (3,235) | (3,235) | |||||||
Stock issued for contingent consideration (in shares) | 771,804 | ||||||||
Stock issued for contingent consideration | 9,316 | $ 8 | 9,308 | ||||||
Stock-based compensation | 44,430 | 44,430 | |||||||
Warrants exercised (in shares) | 101,787 | ||||||||
Warrants exercised | 812 | $ 1 | 1,487 | (676) | |||||
Equity component of the convertible notes (in shares) | 43,995,139 | ||||||||
Equity component of the convertible notes | 215,476 | $ 440 | 215,036 | ||||||
Termination of capped call confirmations | 19,875 | 19,875 | |||||||
Unrealized holding gain on available-for-sale securities | 467 | 467 | |||||||
Foreign currency translation adjustment | 2,290 | 2,290 | |||||||
Net loss | $ (356,388) | (356,388) | |||||||
Balance (in shares) at Dec. 31, 2019 | 255,417,869 | 255,417,869 | |||||||
Balance at Dec. 31, 2019 | $ 476,425 | $ 2,598 | 2,227,225 | 12,387 | 2,825 | (1,768,610) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Stock issued from exercise of stock options, net (in shares) | 5,233,672 | ||||||||
Stock issued from exercise of stock options, net | 42,282 | $ 52 | 42,230 | ||||||
Restricted stock tax vesting (in shares) | 1,411,920 | ||||||||
Restricted stock tax vesting | (10,028) | (10,028) | |||||||
Stock-based compensation | 49,151 | 49,151 | |||||||
Unrealized holding gain on available-for-sale securities | (225) | (225) | |||||||
Foreign currency translation adjustment | 5,627 | 5,627 | |||||||
Net loss | $ (276,852) | (276,852) | |||||||
Balance (in shares) at Dec. 31, 2020 | 262,063,461 | 262,063,461 | |||||||
Balance at Dec. 31, 2020 | $ 286,380 | $ 2,650 | $ 2,308,578 | $ 12,387 | $ 8,227 | $ (2,045,462) |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Operating activities | |||
Net loss | $ (276,852) | $ (356,388) | $ (348,995) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Amortization of debt discount and deferred financing | 1,794 | 2,546 | 10,976 |
Depreciation and amortization | 8,846 | 4,775 | 4,216 |
Stock-based compensation | 49,151 | 44,430 | 29,260 |
Loss on exchange of convertible notes | 0 | 40,624 | 0 |
Loss on extinguishment of debt | 7,276 | 0 | 0 |
Change in fair value of derivatives | 0 | 0 | 2,739 |
Non-cash changes in the fair value of contingent consideration payable | 3,144 | 3,297 | 3,300 |
Foreign currency remeasurement loss | 5,471 | 254 | 3,217 |
Non-cash deferred taxes | (155) | (1,415) | 0 |
Other | 99 | (149) | 59 |
Changes in operating assets and liabilities: | |||
Accounts receivable | (11,219) | (11,114) | (13,294) |
Inventories | (4,639) | (5,114) | (4,205) |
Prepaid expenses and other current assets | (8,766) | (3,287) | 2,488 |
Accounts payable, accrued expenses, and other current liabilities | (10,610) | 46,742 | 17,115 |
Other non-current assets and liabilities | 4,420 | (10,117) | (581) |
Deferred reimbursements | (1,250) | (5,500) | (6,250) |
Net cash used in operating activities | (233,290) | (250,416) | (299,955) |
Investing activities | |||
Sale and redemption of marketable securities | 354,826 | 499,047 | 463,502 |
Purchases of marketable securities | (365,178) | (383,882) | (578,394) |
Capital expenditures | (3,227) | (20,012) | (6,308) |
Net cash (used in) provided by investing activities | (13,579) | 95,153 | (121,200) |
Financing activities | |||
Proceeds from issuance of common stock, net of issuance costs | 0 | 188,994 | 294,584 |
Payment of long-term debt | (155,249) | 0 | 0 |
Proceeds from long-term debt, net of issuance costs | 385,929 | 0 | 146,596 |
Payment of finance leases | (76) | (420) | (334) |
Purchase of vested restricted stock units | (10,028) | (3,235) | (2,832) |
Proceeds from termination of capped call confirmations | 0 | 19,875 | 0 |
Proceeds from exercise of stock options | 42,282 | 11,476 | 9,144 |
Proceeds from exercise of warrants | 0 | 812 | 3,617 |
Net cash provided by financing activities | 262,858 | 217,502 | 450,775 |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | 3,830 | 1,729 | 1,518 |
Net increase in cash, cash equivalents, and restricted cash | 19,819 | 63,968 | 31,138 |
Cash, cash equivalents, and restricted cash at the beginning of the year | 146,343 | 82,375 | 51,237 |
Cash, cash equivalents, and restricted cash at the end of the year | 166,162 | 146,343 | 82,375 |
Supplemental disclosures of cash flow information | |||
Tenant improvements paid through lease incentive | 470 | 19,388 | 0 |
Cash paid during the period for interest | 24,683 | 16,966 | 7,500 |
Contingent consideration paid in shares | 0 | 9,316 | 0 |
Capital expenditures unpaid at the end of period | 985 | 1,865 | 106 |
Cash paid for taxes | $ 10,371 | $ 691 | $ 1,062 |
Description of Business
Description of Business | 12 Months Ended |
Dec. 31, 2020 | |
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 led by the first, oral monotherapy for Fabry disease that has achieved widespread global approval, a differentiated biologic for Pompe disease, and an industry leading rare disease gene therapy portfolio. 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 other 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, clinical-stage, potential best-in-class treatment paradigm for Pompe disease. In February 2019, the FDA granted Breakthrough Therapy designation ("BTD") to AT-GAA for the treatment of late onset Pompe disease. The Company initiated the rolling Biologic License Application ("BLA") submission to the FDA in the fourth quarter of 2020. The Company has established an industry leading gene therapy portfolio of potential therapies for people living with rare metabolic diseases, through a license with Nationwide Children's Hospital ("Nationwide Children's") and an expanded collaboration with the University of Pennsylvania ("Penn"). The Company's pipeline includes gene therapy programs in rare, neurologic lysosomal disorders ("LDs"), specifically: CLN6, CLN3, and CLN1 Batten disease, Pompe disease, Fabry disease, CDKL5 deficiency disorder ("CDD"), Mucopolysaccharidosis Type IIIB ("MPS IIIB"), as well as a next generation program in Mucopolysaccharidosis Type IIIA ("MPS IIIA"). This expanded collaboration with Penn also provides the Company with exclusive disease-specific access and option rights to develop potentially disruptive new gene therapy platform technologies and programs for most LDs and a broader portfolio of more prevalent rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy, and select other muscular dystrophies. In the first quarter of 2020, the FDA granted Fast Track designation to the CLN3 Batten disease gene therapy, AT-GTX-502, for the treatment of pediatric patients less than 18 years of age. In September 2020 and February 2021, the European Medicines Agency granted Priority Medicines designation and the FDA granted Fast Track Designation, respectively, to the CLN6 Batten disease gene therapy, AT-GTX-501, for the treatment of patients with variant late infantile neuronal ceroid lipofuscinosis 6 ("vLINCL6"). The Company's operations have not been significantly impacted from the novel coronavirus (“COVID-19”) pandemic in 2020; however, the Company observed increased lag times between patient identification and Galafold ® initiation due to the resurgence of COVID-19 in the fourth quarter. The Company has maintained operations in all geographies, secured its global supply chain for its commercial and clinical products, and maintained the operational integrity of its clinical trials, with minimal disruption. The Company believes its ability to 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. In July 2020, the Company entered into a definitive agreement for a $400 million credit facility with Hayfin Capital Management (“Senior Secured Term Loan due 2026”) with an interest rate equal to 3-month LIBOR, subject to a 1% floor, plus 6.5% per annum and requires interest-only payments until mid-2024 and matures in six years in 2026. This transaction resulted in net proceeds of $385.9 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2026. Additionally, the Company used $156.3 million of the proceeds to voluntarily settle the principal amount, accrued interest, and early settlement premiums of the Senior Secured Term Loan with BioPharma Credit PLC that was due in 2023 ("Senior Secured Term Loan due 2023"). The Company had an accumulated deficit of $2.0 billion as of December 31, 2020 and anticipates incurring losses through the fiscal year ending December 31, 2021 and beyond. The Company has historically funded its operations through stock offerings, debt issuances, Galafold ® revenues, collaborations, and other financing arrangements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Basis of Presentation The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. 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 December 31, 2020 and through the issuance of this report. 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 comprehensive income (loss) in the 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 December 31, 2020 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 December 31, 2020, the Company recorded an allowance for doubtful accounts of $0.1 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. 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 ("EAP"). Orders for Galafold ® are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2020, one customer accounted for 25% of net product sales and 12% of accounts receivable from product sales. In 2019, one customer accounted for 22% of net product sales and 11% of accounts receivable from product sales. 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: For the Year (in thousands) 2020 2019 2018 U.S. $ 80,046 $ 52,478 $ 7,223 Ex-U.S. 180,840 129,759 84,022 Total net product sales $ 260,886 $ 182,237 $ 91,245 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. Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the FASB guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participants assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability. Contingent Liabilities On an ongoing basis, the Company may be involved in various claims and legal proceedings. On a quarterly basis, the Company reviews the status of each significant matter and assesses its potential financial exposure. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount can be reasonably estimated, the Company will accrue a liability for the estimated loss. Because of uncertainties related to claims and litigation, accruals will be based on the Company's best estimates based on available information. On a periodic basis, as additional information becomes available, or based on specific events such as the outcome of litigation or settlement of claims, the Company may reassess the potential liability related to these matters and may revise these estimates, which could result in material adverse adjustments to the Company's operating results. Research and Development Costs Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development. Interest Income and Interest Expense Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases. Income Taxes The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized. Other Comprehensive Income (Loss) Components of other comprehensive income (loss) include unrealized gains and losses on available-for-sale securities and gain (loss) on foreign currency transactions and are included in the Statements of Comprehensive Loss. Leases The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets. ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. The Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories. The lease term for all of the Company's leases include the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories. The information presented for the periods prior to January 1, 2019 has not been restated and is reported under the accounting standard in effect for those periods. For additional information, see " -Note 13. Leases". Nonqualified Cash Deferral Plan The Company's Cash Deferral Plan (the "Deferral Plan"), provides certain key employees and members of the Board of Directors as selected by the Compensation Committee of the Board of Directors of the Company (the "Compensation Committee"), with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code (the "Code"). All of the investments held in the Deferral Plan are classified as investments held-to-maturity and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. The corresponding liability for the Deferral Plan is included in other non-current liability in the Consolidated Balance Sheets. Equity-based Compensation At December 31, 2020, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 9. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award. Loss per Common Share The Company calculates net loss per share as a measurement of the Company's performance while giving effect to all dilutive potential common shares that were outstanding during the reporting period. The Company had a net loss for all periods presented; accordingly, the inclusion of common stock options, unvested RSUs, and warrants would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted earnings per share are the same. See "— Note 17. Basic and Diluted Net Loss per Common Share" for further discussion on net loss per share. Segment Information The Company currently operates in one business segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker who comprehensively manages the entire business. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Company does not accumulate discrete financial information with respect to separate service lines, and thus there is one reporting unit. Business Combinations The Company assigns fair value to the tangible and intangible assets acquired and liabilities assumed based upon their estimated fair values on the acquisition date from acquired businesses. The purchase price allocation process requires management to make significant estimates and assumptions, especially at the acquisition date with respect to intangible assets and in-process research and development ("IPR&D"). In connection with the purchase price allocations for acquisitions, the Company estimates the fair value of contingent payments utilizing a probability-based income approach inclusive of an estimated discount rate. Contingent Consideration Payable Contingent consideration payments in asset acquisitions are recognized when the contingency is resolved and the consideration is paid or becomes payable. This does not apply in circumstances when the contingent consideration meets the definition of a derivative, in which case the amount becomes part of the basis in the asset acquired. Upon recognition of the contingent consideration payment, the amount is included in the cost of the acquired asset or group of assets. The Company determines the fair value of contingent acquisition consideration payable on the acquisition date using a probability-based income approach utilizing an appropriate discount rate. The short term and long term portions of the contingent consideration payable is shown on the Company's Consolidated Balance Sheets. The fair value of the contingent consideration payable will be determined each period end and the resulting change will be recorded on the Consolidated Statements of Operations. Intangible Assets and Goodwill The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Purchased IPR&D is accounted for as an indefinite lived intangible asset until the underlying project is completed, at which point the intangible asset will be accounted for as a definite lived intangible asset, or abandoned, at which point the intangible asset will be written off or partially impaired. Goodwill and indefinite lived intangible assets are assessed annually for impairment and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. If it is determined that the full carrying amount of an asset is not recoverable, an impairment loss is recorded in the amount by which the carrying amount of the asset exceeds its fair value. No indicators of impairment were noted during the years ended December 31, 2020 and December 31, 2019. Recent Accounting Developments - Guidance Adopted in 2020 ASU 2018-15 - In August 2018, the Financial Accounting Standards Board ("FASB") issued Accounting Standards Update ("ASU") 2018-15, Intangibles-Goodwill and Other-Internal-Use Software (Subtopic 350-40): ("ASU 2018-15"), relating to a customer’s accounting for implementation, set-up, and other upfront costs incurred in a cloud computing arrangement that is hosted by a vendor. Under the new guidance, a customer will apply the same criteria for capitalizing implementation costs as it would for an arrangement that has a software license. The new guidance does not affect the accounting for the service element of a hosting arrangement that is a service contract. The new guidance also prescribes the balance sheet, income statement and cash flow classification of the capitalized software costs and related amortization expense and requires additional quantitative and qualitative disclosures. ASU 2018-15 is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019 for public companies. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. ASU 2018-13 - In August 2018, the FASB issued ASU 2018-03, Fair Value Measurement (Topic 820): Disclosure Framework-Changes to the Disclosure Requirements for Fair Value Measurement ("ASU 2018-13"). The amendments modify the disclosure requirements in Topic 820. ASU 2018-13 is effective for all entities for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. The amendments on (i) changes in unrealized gains and losses, (ii) the range and weighted average of significant unobservable inputs used to develop Level 3 fair value measurements, and (iii) the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. ASU 2017-04 - In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment ("ASU 2017-04"). ASU 2017-04 simplifies the recognition and measurement of a goodwill impairment loss by eliminating Step 2 of the quantitative goodwill impairment test. The guidance requires a one-step impairment test in which an entity compares the fair value of a reporting unit with its carrying amount and recognizes an impairment charge for the amount by which the carrying amount exceeds the reporting unit's fair value, if any. ASU 2017-04 is effective for fiscal years beginning after December 15, 2019 and should be applied on a prospective basis. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. ASU 2016-13 - In June 2016, the FASB issued ASU 2016-13, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments ("ASU 2016-13"). ASU 2016-13 requires financial assets measured at amortized cost basis to be presented at the net amount expected to be collected and amends guidance on the impairment of financial instruments. ASU 2016-13 is effective for public companies who are SEC filers for fiscal years beginning after December 15, 2019, including interim periods within those years. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. |
Acquisitions
Acquisitions | 12 Months Ended |
Dec. 31, 2020 | |
Business Combinations [Abstract] | |
Acquisitions | Acquisitions Acquisition of Celenex In September 2018, the Company expanded its pipeline by acquiring the rights and related intellectual property of ten gene therapy programs through its acquisition of Celenex, Inc., ("Celenex"). Celenex is a private, clinical stage gene therapy company whose lead programs are ten gene therapy programs including CLN6 and CLN3, which are in clinical stage, and several programs in pre-clinical stage. Pursuant to the terms of the agreement, the Company agreed to pay up to an additional $10 million in connection with the achievement of certain development milestones, $220 million in connection with the achievement of certain regulatory approval milestones across multiple programs and up to $75 million in tiered sales milestone payments. Celenex has an exclusive license agreement with Nationwide Children’s Hospital ("Nationwide Children’s"). Under this license agreement, Nationwide Children’s is eligible to receive development and sales-based milestones of up to $7.8 million for each product. The Company evaluated the Celenex transaction and concluded that the transaction did not meet the definition of a business and was an asset acquisition. Given the fact that the license has no alternative future use, the $100 million upfront payment was expensed to research and development expense in the Consolidated Statements of Operations for the year ended December 31, 2018 . |
Goodwill and Intangible Assets
Goodwill and Intangible Assets | 12 Months Ended |
Dec. 31, 2020 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Goodwill and Intangible Assets | Goodwill and Intangible Assets As of December 31, 2020, in connection with the acquisitions, the Company had goodwill of $197.8 million and IPR&D of $23.0 million, which are reported at fair value on the Company's Consolidated Balance Sheets. Intangible assets related to IPR&D assets are considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts. During the period the assets are considered indefinite-lived, they will not be amortized but will be tested for impairment on an annual basis and between annual tests if the Company becomes aware of any events occurring or changes in circumstances that would indicate a reduction in the fair value of the IPR&D assets below their respective carrying amounts. Goodwill and intangible assets are assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. If it is determined that the full carrying amount of an asset is not recoverable, an impairment loss is recorded in the amount by which the carrying amount of the asset exceeds its fair value. The following table represents the changes in IPR&D for the years ended December 31, 2020 and 2019, respectively: (in millions) Balance at December 31, 2018 $ 23.0 Change in IPR&D — Balance at December 31, 2019 $ 23.0 Change in IPR&D — Balance at December 31, 2020 $ 23.0 The following table represents the changes in Goodwill for the years ended December 31, 2020 and 2019, respectively: (in millions) Balance at December 31, 2018 $ 197.8 Change in goodwill — Balance at December 31, 2019 $ 197.8 Change in goodwill — Balance at December 31, 2020 $ 197.8 |
Cash, Cash Equivalents, Marketa
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash | 12 Months Ended |
Dec. 31, 2020 | |
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 December 31, 2020, the Company held $163.2 million in cash and cash equivalents and $320.0 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within accumulated other comprehensive loss in the 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 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 December 31, 2020 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 163,240 $ — $ — $ 163,240 Corporate debt securities 39,525 4 (16) 39,513 Commercial paper 217,087 14 (6) 217,095 Asset-backed securities 9,420 18 — 9,438 U.S. government agency bonds 53,583 3 (4) 53,582 Money market 350 — — 350 Certificates of deposit 51 — — 51 $ 483,256 $ 39 $ (26) $ 483,269 Included in cash and cash equivalents $ 163,240 $ — $ — $ 163,240 Included in marketable securities 320,016 39 (26) 320,029 Total cash, cash equivalents, and marketable securities $ 483,256 $ 39 $ (26) $ 483,269 As of December 31, 2019 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 142,837 $ — $ — $ 142,837 Corporate debt securities 145,875 121 (5) 145,991 Commercial paper 73,659 53 (2) 73,710 Asset-backed securities 77,731 79 — 77,810 U.S. government agency bonds 11,999 2 (10) 11,991 Money market 350 — — 350 Certificates of deposit 51 — — 51 $ 452,502 $ 255 $ (17) $ 452,740 Included in cash and cash equivalents $ 142,837 $ — $ — $ 142,837 Included in marketable securities (1) 309,665 255 (17) 309,903 Total cash, cash equivalents, and marketable securities $ 452,502 $ 255 $ (17) $ 452,740 ______________________________ (1) As of December 31, 2019, $9.5 million of marketable securities had maturity dates greater than 12 months and were available to convert into cash, if needed. For the year ended December 31, 2020 there were nominal realized gains. For the fiscal year ended December 31, 2019, 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 December 31, 2020 and 2019 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 gain (loss). The fair value of these marketable securities in unrealized loss positions was $124.9 million and $42.6 million as of December 31, 2020 and December 31, 2019, 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. (in thousands) December 31, 2020 December 31, 2019 December 31, 2018 Cash and cash equivalents $ 163,240 $ 142,837 $ 79,749 Restricted cash 2,922 3,506 2,626 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 166,162 $ 146,343 $ 82,375 |
Inventories
Inventories | 12 Months Ended |
Dec. 31, 2020 | |
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) December 31, 2020 December 31, 2019 Raw materials $ 5,547 $ 6,544 Work-in-process 7,693 3,660 Finished goods 6,316 3,837 Total inventories $ 19,556 $ 14,041 The Company recorded a reserve for inventory of $0.1 million and $0.2 million as of December 31, 2020 and December 31, 2019, respectively. |
Property and Equipment
Property and Equipment | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment | Property and Equipment Property and equipment consist of the following: December 31, (in thousands) 2020 2019 Property and equipment consist of the following: Computer equipment $ 5,546 $ 6,185 Computer software 1,207 1,607 Research equipment 15,387 14,568 Furniture and fixtures 2,880 5,157 Leasehold improvements 24,146 30,294 Vehicles 124 124 Land 3,190 3,190 Construction in progress 5,870 4,184 Gross property and equipment 58,350 65,309 Less accumulated depreciation (14,487) (17,604) Net property and equipment $ 43,863 $ 47,705 Depreciation expense was $8.4 million and $4.5 million for the years ended December 31, 2020 and December 31, 2019, respectively. |
Accrued Expenses and Other Curr
Accrued Expenses and Other Current Liabilities | 12 Months Ended |
Dec. 31, 2020 | |
Payables and Accruals [Abstract] | |
Accrued Expenses and Other Current Liabilities | Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consist of the following: December 31, (in thousands) 2020 2019 Accrued professional fees $ 3,700 $ 7,832 Accrued contract manufacturing & contract research costs 19,617 17,800 Accrued compensation and benefits 23,025 27,156 Accrued program fees 16,599 17,016 Accrued royalties 6,550 5,196 Accrued interest — 3,679 Accrued milestones — 4,000 Accrued sales rebates and discounts 12,079 5,900 Accrued taxes 11,177 7,300 Deferred reimbursements 1,500 1,250 Other 2,594 2,772 $ 96,841 $ 99,901 |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2020 | |
Equity [Abstract] | |
Stockholders' Equity | Stockholders' Equity Common Stock and Warrants As of December 31, 2020, the Company was authorized to issue 500 million shares of common stock. Dividends on common stock will be paid when, and if, declared by the board of directors. Each holder of common stock is entitled to vote on all matters that are appropriate for stockholder voting and is entitled to one vote for each share held. In February 2021, 1,050,000 warrants were exercised at $7.06 per share of common stock resulting in gross cash proceeds of $7.4 million. During the second quarter of 2019, the Company completed an underwritten equity offering and issued 18.7 million shares of its common stock at $10.75 per share, inclusive of the fully exercised option to purchase additional shares from the initial offering. This transaction resulted in net proceeds of $189.0 million, after deducting underwriting discounts and commissions and offering expenses. In March 2019, 101,787 warrants were exercised at $7.98 per share of common stock resulting in gross cash proceeds of $0.8 million. As discussed in "— Note 11. Assets and Liabilities Measured at Fair Value", during the fourth quarter of 2018, the Company reached a clinical milestone, which was the dosing of the first patient in a Phase 3 study, related to the contingent consideration from the acquisition of Callidus Biopharma, Inc. ("Callidus"). The milestone for this event was $9.0 million, which was paid in Company common stock in the first quarter of 2019 and resulted in a $9.3 million impact on stockholder's equity. As discussed in ''— Note 12. Debt'', during the first and second quarters of 2019, the Company entered into separate, privately negotiated Exchange Agreements with a limited number of holders of the Convertible Notes. Under the terms of the Exchange Agreements, the holders of the Convertible Notes agreed to exchange an aggregate principal amount of $247.2 million of Convertible Notes held by them in exchange for an aggregate of approximately 44.0 million shares of Company's common stock, par value $0.01 per share. In February 2018, the Company completed an underwritten offering of 20.2 million shares of its common stock at $15.50 per share, inclusive of the fully exercised option to purchase additional shares from the initial offering. This transaction resulted in net proceeds of $294.6 million, after deducting underwriting discounts and commissions and offering expenses. In April 2018, 453,214 warrants were exercised at $7.98 per share of common stock resulting in gross cash proceeds of $3.6 million. In June 2018, the Company’s stockholders approved an amendment to the Company’s Restated Certificate of Incorporation to increase the number of shares of common stock, par value $0.01 per share, that the Company is authorized to issue from 250 million shares to 500 million shares. Nonqualified Cash Plan The Company's Deferral Plan, (the "Deferral Plan") provides certain key employees and members of the Board of Directors as selected by the Compensation Committee, with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code of 1986 as amended. The Company had a deferred compensation investment balance of $4.1 million and $4.4 million as of December 31, 2020 and December 31, 2019, respectively, with corresponding approximate amounts of liability. Deferral Plan investment assets are classified as trading securities and are recorded at fair value with changes in the investments' fair value recognized in AOCI in the period they occur. Deferred compensation liability amounts under the Deferral Plan are included in other long-term liabilities. Equity Incentive Plan The Company's Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. Under the provisions of the Plan, no option will have a term in excess of 10 years. 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. Options granted pursuant to the Plan generally vest 25% on the first year anniversary date of grant plus an additional 1/48th for each month thereafter and may be exercised in whole or in part for 100% of the shares vested at any time after the date of grant. As of December 31, 2020, the Company has reserved up to 15,018,368 shares for issuance under the Plan. |
Share based Compensation
Share based Compensation | 12 Months Ended |
Dec. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | |
Share based Compensation | Share based Compensation The Company’s Equity Incentive 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. On December 21, 2018, the Board of Directors of the Company approved an amendment (the "Amendment") to the Plan. The Amendment provides for certain benefits to qualifying Plan Participants who separate from service with the Company due to death, disability or "Retirement" (as such term is defined under the Plan) ("Qualified Participants"). Options granted under the Plan ("Options") to a Qualified Participant shall continue to vest until the 2nd anniversary of the Qualified Participant’s separation and all vested Options held by such Qualified Participant shall remain exercisable until the earlier of the 4th anniversary of the Qualified Participant’s separation or the original expiration date of the Option. Options that are not exercised during this exercise period shall be forfeited. Time-based restricted stock units and restricted stock granted to a Qualified Participant under the Plan that was scheduled to vest within the two two Stock Option Grants The Company uses the fair value method of measuring stock-based compensation, using the fair value of each equity award granted. The Company chose the ‘‘straight-line’’ attribution method for allocating compensation costs and recognized the fair value of each stock option on a straight-line basis over the vesting period of the related awards. The Company uses the Black-Scholes option pricing model when estimating the grant date fair value for stock-based awards. Use of a valuation model requires management to make certain assumptions with respect to selected model inputs. Expected volatility was based on our historical volatility since our initial public offering in May 2007. The average expected life is determined using our actual historical data. The risk-free interest rate is based on U.S. Treasury, zero-coupon issues with a remaining term equal to the expected life assumed at the date of grant. Forfeitures are estimated based on voluntary termination behavior, as well as a historical analysis of actual option forfeitures. 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: Years Ended December 31, 2020 2019 2018 Expected stock price volatility 75.1 % 74.1 % 78.6 % Risk free interest rate 1.6 % 2.4 % 2.4 % Expected life of options (years) 5.67 5.68 5.62 Expected annual dividend per share $ 0.00 $ 0.00 $ 0.00 The weighted average grant-date fair value per share of options granted during 2020, 2019, and 2018 were $6.40, $6.67, and $10.19, respectively. A summary of the Company's stock options for the year ended December 31, 2020 were as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Options outstanding, December 31, 2017 15,181 $ 7.48 Granted 2,348 $ 14.96 Exercised (1,398) $ 6.54 Forfeited (313) $ 9.55 Expired (8) $ 10.76 Options outstanding, December 31, 2018 15,810 $ 8.63 Granted 4,091 $ 10.29 Exercised (1,967) $ 5.83 Forfeited (879) $ 11.07 Expired (331) $ 13.38 Options outstanding, December 31, 2019 16,724 $ 9.15 Granted 4,362 $ 9.98 Exercised (5,243) $ 8.11 Forfeited (1,376) $ 10.42 Expired (435) $ 13.33 Options outstanding, December 31, 2020 14,032 $ 9.54 6.5 $ 190.1 Vested and unvested expected to vest, December 31, 2020 13,267 $ 9.49 6.4 $ 180.5 Exercisable at December 31, 2020 8,130 $ 8.86 5.1 $ 115.7 The aggregate intrinsic value of options exercised during the years ended December 31, 2020, 2019 and 2018 was $40.9 million, $11.8 million, and $11.9 million respectively. Cash proceeds from stock options exercised during the years ended December 31, 2020, 2019, and 2018 were $42.3 million, $11.5 million, and $9.1 million, respectively. As of December 31, 2020, the total unrecognized compensation cost related to non-vested stock options granted was $29.4 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 year ended December 31, 2020 is as follows: Number of Weighted Average Grant Date Fair Value Weighted Average Remaining Years Aggregate Intrinsic Value (in thousands) (in millions) Non-vested units as of December 31, 2017 2,575 $ 5.85 Granted 1,812 $ 16.11 Vested (530) $ 6.01 Forfeited (145) $ 9.65 Non-vested units as of December 31, 2018 3,712 $ 10.59 Granted 3,526 $ 10.92 Vested (921) $ 8.49 Forfeited (525) $ 10.52 Non-vested units as of December 31, 2019 5,792 $ 11.18 Granted 4,692 $ 11.29 Vested (2,164) $ 10.70 Forfeited (1,240) $ 11.14 Non-vested units as of December 31, 2020 7,080 $ 11.35 2.3 $ 158.0 For the year ended December 31, 2020, 2,163,732 RSUs have vested and all non-vested units are expected to vest over their normal term. As of December 31, 2020, there was $59.4 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years. Compensation Expense Related to Equity Awards The following table summarizes information related to compensation expense recognized in the Consolidated Statements of Operations related to the equity awards: Years Ended December 31, (in thousands) 2020 2019 2018 Equity compensation expense recognized in: Research and development expense $ 20,817 $ 17,575 $ 11,740 Selling, general, and administrative expense 28,334 26,855 17,520 Total equity compensation expense $ 49,151 $ 44,430 $ 29,260 |
Assets and Liabilities Measured
Assets and Liabilities Measured at Fair Value | 12 Months Ended |
Dec. 31, 2020 | |
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 December 31, 2020 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 217,095 $ 217,095 Asset-backed securities 9,438 9,438 Corporate debt securities 39,513 39,513 U.S. government agency bonds 53,582 53,582 Money market funds 4,427 4,427 $ 324,055 $ 324,055 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 25,825 $ 25,825 Deferred compensation plan liability 4,078 — 4,078 $ 4,078 $ 25,825 $ 29,903 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, 2019 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 73,710 $ 73,710 Asset-backed securities 77,810 77,810 Corporate debt securities 145,991 145,991 U.S. government agency bonds 11,991 11,991 Money market funds 4,768 4,768 $ 314,270 $ 314,270 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 22,681 $ 22,681 Deferred compensation plan liability 4,419 — 4,419 $ 4,419 $ 22,681 $ 27,100 The Company’s Convertible Notes fall into the Level 2 category within the fair value level hierarchy. The fair value was determined using broker quotes in a non-active market for valuation. The fair value of the Convertible Notes at December 31, 2020 was $10.9 million. 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 December 31, 2020 or December 31, 2019. 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. No changes in valuation techniques or inputs occurred during the year ended December 31, 2020. No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the year ended December 31, 2020. Contingent Consideration Payable The contingent consideration payable resulted from the acquisition of Callidus. 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 ATB-200 Pompe program: Contingent Consideration Liability Fair Value as of December 31, 2020 Valuation Technique Unobservable Input Range (in thousands) Discount rate 7.5% Clinical and regulatory milestones $ 25,101 Probability weighted discounted cash flow Probability of achievement of milestones 75%-78% Projected year of payments 2021-2022 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. During the fourth quarter of 2018, the Company reached a clinical milestone, which was the dosing of the first patient in a Phase 3 study, related to the contingent consideration from the acquisition of Callidus. The milestone for this event was $9.0 million, which was paid in Company common stock in the first quarter of 2019 and resulted in a $9.3 million impact on stockholder's equity. The following table shows the change in the balance of contingent consideration payable for the year ended December 31, 2020 and December 31, 2019, respectively: Years ended December 31, (in thousands) 2020 2019 Balance, beginning of the period $ 22,681 $ 19,700 Changes in fair value during the period, included in the Consolidated Statements of Operations 3,144 3,297 Adjustment for contingent consideration paid in stock — (316) Balance, end of the period $ 25,825 $ 22,681 |
Debt
Debt | 12 Months Ended |
Dec. 31, 2020 | |
Debt Disclosure [Abstract] | |
Debt | Debt The Company's debt consists of the following: (in thousands) December 31, 2020 December 31, 2019 Senior Secured Term Loan due 2026: Principal $ 400,000 $ — Less: debt discount (1) (7,434) — Less: deferred financing (1) (5,592) — Net carrying value of the Senior Secured Term Loan $ 386,974 $ — Senior Secured Term Loan due 2023 (2) : Principal $ — $ 150,000 Less: debt discount (1) — (2,315) Less: deferred financing (1) — (311) Net carrying value of the Senior Secured Term Loan $ — $ 147,374 Convertible Notes (3) : Principal $ 2,825 $ 2,825 Less: debt discount (1) (518) (659) Less: deferred financing (1) (27) (35) Net carrying value of the Convertible Notes $ 2,280 $ 2,131 Net carrying value of Long-term debt $ 389,254 $ 149,505 ______________________________ (1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Convertible Notes and Senior Secured Term Loans using the effective interest rate method. (2) The principle, accrued interest and early settlement premiums associated with the Senior Secured Term Loan due 2023 were fully paid and settled in July 2020. (3) The Convertible Notes are currently convertible as the last reported sale price of the Company's common stock was equal to or more than 130% of the conversion price for at least 20 trading days of the 30 consecutive trading days ending on the last day of the quarter. Senior Secured Term Loan due 2026 In July 2020, the Company entered into a definitive agreement for a $400 million credit facility with Hayfin Capital Management (“Senior Secured Term Loan due 2026”) with an interest rate equal to 3-month LIBOR, subject to a 1% floor, plus 6.5% per annum and requires interest-only payments until mid-2024. The Senior Secured Term Loan due 2026 will be repaid in nine quarterly payments of $44.4 million, starting on July 2024 with the final balance due on the maturity date in July 2026. This transaction resulted in net proceeds of $385.9 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2026. The Company used the proceeds to voluntarily settle the Senior Secured Term Loan with BioPharma Credit PLC that was due in 2023 ("Senior Secured Term Loan due 2023"). The Senior Secured Term Loan due 2026 is subject to mandatory prepayment provisions that require prepayment upon a change of control, the incurrence of certain additional indebtedness, asset sale, or an event of loss, subject to certain conditions set forth in the Senior Secured Term Loan due 2026. The Company may prepay the Senior Secured Term Loan due 2026 in whole, at its option at any time. Any prepayment of the Senior Secured Term Loan due 2026 is subject to certain make-whole premiums and prepayment premiums, the latter of which decrease until the fourth anniversary of the transaction date at which point no prepayment penalty shall exist. The obligations under the Senior Secured Term Loan due 2026 are secured by a first lien security interest in certain assets of the Company. The Senior Secured Term Loan due 2026 contains certain customary representations and warranties, affirmative and negative covenants and events of default applicable to the Company. The Senior Secured Term Loan due 2026 also contains a minimum liquidity covenant of $75 million, and an incremental minimum consolidated revenue covenant, measured as of the previous four consecutive fiscal quarters. The minimum consolidated revenue covenant ranges from $140 million, beginning March 31, 2021, and peaks at $225 million by June 30, 2023, continuing at that level until the Senior Secured Term Loan due 2026 is repaid. If an event of default occurs and is continuing, Hayfin Capital Management may declare all amounts outstanding under the Senior Secured Term Loan due 2026 to be immediately due and payable. Senior Secured Term Loan due 2023 In September 2018, the Company entered into a loan agreement with BioPharma Credit PLC (“Senior Secured Term Loan due 2023") as the lender. The loan agreement provided for a $150 million senior secured term loan with an interest rate equal to the 3-month LIBOR plus 7.5% per annum and would mature in 5 years from the maturity date. The Senior Secured Term Loan due 2023 would be repaid in four quarterly payments equal to 12.5% thereof starting on the forty-eight month anniversary of the date of the first credit extension with the balance due on the Maturity Date. Interest was payable quarterly in arrears. The Senior Secured Term Loan due 2023 contained certain customary representations and warranties, affirmative and negative covenants, and events of default applicable to the Company and certain of its subsidiaries but did not include any financial covenants relating to the achievement or maintenance of revenue or cash flow. If an event of default occurred and was continuing, the lender could declare all amounts outstanding under the Senior Secured Term Loan due 2023 to be immediately due and payable. The Company received net proceeds of $146.6 million in September 2018, after deducting fees and expenses. In July 2020, the Company used $156.3 million of the proceeds from the Senior Secured Term Loan due 2026 to voluntarily settle the principal amount, $1.1 million accrued interest, and $5.2 million in early settlement premiums. As a result of this early extinguishment, the Company recognized a loss on extinguishment of debt of $7.3 million in the Consolidated Statements of Operations. Convertible Notes In December 2016, the Company issued at par value $250 million aggregate principal amount of Convertible Notes, which included the exercise in full of the $25 million over-allotment option granted to the initial purchasers of the Convertible Notes in a private offering to qualified institutional buyers pursuant to Rule 144A under the Securities Act (the "Note Offering"). Interest is payable semiannually on June 15 and December 15 of each year, beginning on June 15, 2017. The Convertible Notes will mature on December 15, 2023, unless earlier repurchased, redeemed, or converted in accordance with their terms. The Convertible Notes are convertible at the option of the holders of the Convertible Notes (the "Holders"), under certain circumstances and during certain periods, into cash, shares of the Company’s common stock or a combination thereof. The net proceeds from the Note Offering were $243.0 million, after deducting fees and expenses. In addition, the Company used approximately $13.5 million of the net proceeds from the issuance of the Convertible Notes to pay the cost of the capped call transactions ("Capped Call Confirmations") that the Company entered into in connection with the issuance of the Convertible Notes. In accounting for the issuance of the Convertible Notes, the Company separated the Convertible Notes into liability and equity components based on their relative values. The Convertible Notes were initially convertible into approximately 40.8 million shares of the Company’s common stock under certain circumstances prior to maturity at a conversion rate of 163.3987 shares per $1,000 principal amount of Convertible Notes, which represents a conversion price of approximately $6.12 per share of common stock, subject to adjustment under certain conditions. Holders may convert their Convertible Notes at their option at specified times prior to the maturity date of December 15, 2023, only if: • during any fiscal quarter commencing after March 31, 2017, if the last reported sale price of the Company’s common stock for at least 20 trading days in the period of 30 consecutive trading days ending on the last trading day of the immediately preceding fiscal quarter is equal to or more than 130% of the conversion price of the Convertible Notes on the last day of such preceding fiscal quarter; • a holder submits its Convertible Notes for conversion during the five • the Company issues to all or substantially all of the holders of common stock rights options or warrants entitling then them for a period of not more than 60 calendar days after the date of such issuance to subscribe for or purchase shares of the common stock, at a price per share less than the average of the Last Reported Sale Prices of the common stock for the 10 consecutive Trading Day period ending on, and including, the Trading Day immediately preceding the date of announcement of such issuance or distributes to all or substantially all holders of the common stock the Company’s assets, debt securities or rights to purchase the Company’s securities which distribution has a per share value of exceeding 10% of the Last Reported Sale Price of the common stock on the Trading Day immediately preceding the date of announcement of such distribution; • the Company enters into specified corporate transactions; or • the Company has had a call for redemption, the holder can convert up until the second trading day immediately preceding the redemption date. The Convertible Notes will be convertible, at the option of the note holders, regardless of whether any of the foregoing conditions have been satisfied, on or after September 15, 2023 at any time prior to the close of business on the second scheduled trading day immediately preceding the stated maturity date of December 15, 2023. During the first and second quarters of 2019, the Company entered into separate, privately negotiated Exchange Agreements with a limited number of holders of the Convertible Notes. Under the terms of the Exchange Agreements, the limited number of Holders agreed to exchange an aggregate principal amount of $247.2 million of Convertible Notes held by them in exchange for an aggregate of approximately 44.0 million shares of Company common stock, par value $0.01 per share. In addition, pursuant to the Exchange Agreements, the Company made aggregate cash payments of $1.3 million to the limited number of Holders to satisfy accrued and unpaid interest to the closing date of the transactions, along with cash in lieu of fractional shares. These transactions resulted in $215.0 million in additional paid-in-capital and common stock of $0.4 million on the Consolidated Balance Sheets as of December 31, 2019. Additionally, the Company recognized a loss on the exchange of debt of $40.6 million on the Consolidated Statements of Operations during the year ended December 31, 2019. During the second half of the year ended December 31, 2019, there were no additional debt conversion transactions . During the first and second quarter of 2019, the Company terminated the Capped Call Confirmations related to the exchange of the Convertible Notes for proceeds of $19.9 million. Interest Expense The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2020 and 2019 respectively: (in thousands) December 31, 2020 December 31, 2019 Contractual interest expense $ 20,933 $ 16,483 Amortization of debt discount $ 1,267 $ 2,381 Amortization of deferred financing $ 527 $ 165 Effective interest rate of the liability component, Convertible debt 10.6 % 10.6 % Effective interest rate of the liability component, Senior Secured Term Loan due 2026 8.4 % — % Effective interest rate of the liability component, Senior Secured Term Loan due 2023 — % 10.2 % |
Leases
Leases | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Leases | Leases The Company currently has operating leases for office and research laboratory space, equipment, and vehicles under agreements expiring at various dates through 2044, which include renewal options on leases which the Company is reasonably certain to exercise. For the years ended December 31, 2020, and 2019, the operating lease expense was $11.1 million and $10.8 million, respectively. For the years ended December 31, 2020, and 2019, the Company paid $9.6 million and $4.9 million, respectively, for amounts included in the measurement of operating lease liabilities and recorded $1.7 million and $21.8 million, respectively, of right-of-use assets and $0.5 million and $19.4 million, respectively of tenant improvements paid through lease incentives in exchange for new operating lease liabilities. Commitments under finance leases are not significant for the years ended December 31, 2020, and 2019. Supplemental balance sheet information related to operating leases were as follows: (in thousands, except year and discount rate amounts) December 31, 2020 December 31, 2019 Operating lease ROU asset $ 23,296 $ 33,315 Current portion of the operating lease liabilities $ 6,872 $ 7,189 Non-current portion of the operating lease liabilities 45,604 53,531 Total operating lease liability $ 52,476 $ 60,720 Weighted-average remaining lease terms (years) 19.8 18.2 Weighted-average discount rate 13.0 % 13.1 % At December 31, 2020, the future minimum operating lease payments were as follows: (in thousands) Operating Lease 2021 $ 8,549 2022 8,494 2023 8,691 2024 8,404 2025 8,371 Thereafter 153,241 Total lease payments 195,750 Less lease incentives (28,939) Less imputed interest (114,335) Total operating lease liability $ 52,476 At December 31, 2019, the future minimum operating lease payments were as follows: (in thousands) Operating Lease 2020 $ 9,738 2021 10,787 2022 10,459 2023 10,809 2024 10,582 Thereafter 163,266 Total lease payments $ 215,641 Less lease incentives (28,939) Less imputed interest (125,982) Total operating lease liability $ 60,720 |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes For financial reporting purposes, loss before income taxes includes the following components: Years Ended December 31, (in thousands) 2020 2019 2018 United States $ (365,332) $ (393,955) $ (309,183) Foreign 91,078 38,045 (39,906) Total $ (274,254) $ (355,910) $ (349,089) Following were the components of income tax expense (benefit) for the years ended December 31, 2020, 2019, and 2018: (in thousands) 2020 2019 2018 Current Federal $ — $ — $ — State — — 6 Foreign 4,163 2,877 (100) Deferred Federal (1,410) (984) — State (155) (1,415) — Foreign — — — Total $ 2,598 $ 478 $ (94) A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2020, 2019, and 2018 are as follows: Years Ended 2020 2019 2018 Statutory rate (21) % (21) % (21) % State taxes, net of federal benefit — — (4) Nondeductible IPR&D — — 6 Contingent consideration — — 1 Tax credits (7) (16) (10) Impact of foreign operations 4 9 2 Nondeductible executive compensation 2 1 — Nondeductible debt conversion — (1) — Other — (1) — Valuation allowance 23 29 26 Net 1 % — % — % On December 22, 2017, the U.S. government enacted the Tax Cuts and Jobs Act ("Tax Act"). The Tax Act significantly revises U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Tax Act also introduced an additional U.S. tax on certain non-U.S. subsidiaries’ earnings which are considered to be Global Intangible Low Taxed Income (referred to as "GILTI"). After consideration of the relevant guidance and completing the accounting for the tax effects of the Tax Act, the Company has elected to treat GILTI as a period cost. The Company recorded an income tax expense of $4.2 million in 2020 for taxes in foreign jurisdictions and a $1.6 million tax benefit in 2020 for taxes in U.S. federal and state jurisdictions. The Company did not recognize interest or penalties related to income tax during the period ended December 31, 2020 and did not accrue for interest or penalties as of December 31, 2020. The Company does not have an accrual for uncertain tax positions as of December 31, 2020. Tax returns for years 2015 through 2019 are open to examination by tax authorities. The Company is also subject to examination in any period for which it has net operating losses. Deferred income taxes reflect the net effect of temporary difference between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The significant components of the deferred tax assets and liabilities are as follows: Years Ended (in thousands) 2020 2019 Deferred tax assets Intellectual property $ 68,209 $ 46,521 Amortization/depreciation 489 2,329 Research tax credit 182,651 141,669 Net operating loss carry forwards 305,876 286,850 Deferred reimbursements 1,912 2,230 Non-cash stock issue 17,553 19,058 Interest carry forward limitation 5,655 2,224 Lease liability 9,235 6,983 Other 10,855 13,921 Gross deferred tax assets 602,435 521,785 Deferred tax liabilities Business acquisition (4,937) (5,051) Royalty payable (68,209) (46,521) Convertible notes (94) (653) Advanced R&D payments (1,776) (2,611) Right of use asset (3,420) (5,643) Total net deferred tax assets 523,999 461,306 Less: valuation allowance (528,895) (466,357) Net deferred tax liability $ (4,896) $ (5,051) The Company records a valuation allowance for temporary differences for which it is more likely than not that the Company will not receive future tax benefits. At December 31, 2020 and 2019, the Company recorded valuation allowances of $528.9 million and $466.4 million, respectively, representing an increase in the valuation allowance of $62.5 million in 2020, due to the uncertainty regarding the realization of such deferred tax assets, to offset the benefits of net operating losses generated during those years. The deferred tax liability related to business acquisitions pertains to the basis difference in IPR&D acquired by the Company. The Company's policy is to record a deferred tax liability related to acquired IPR&D that may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. As of December 31, 2020, the Company had federal and state net operating loss carry forwards ("NOLs") of approximately $1,156 million and $889.4 million, respectively. The federal carry forward for losses generated prior to 2018 will expire in 2029 through 2037. Federal net operating losses incurred in 2018 and onward have an indefinite expiration under the 2017 Tax Act. Most of the state carry forwards generated prior to 2009 have expired through 2016. The remaining state carry forwards including those generated in 2009 through 2020 will expire in 2029 through 2040. Utilization of NOLs may be subject to a substantial limitation pursuant to Section 382 of the Internal Revenue Code of 1986, as amended ( the "Code") as well as similar state statutes in the event of an ownership change. Such ownership changes have occurred in the past and could occur again in the future. Under Section 382 of the Internal Revenue Code of 1986, as amended, or Section 382, if a corporation undergoes an "ownership change," generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation's ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development tax credits) to offset its post-change income may be limited. The Company may experience ownership changes in the future as a result of shifts in the stock ownership some of which are outside the Company's control. The Company completed a detailed study of the NOLs and determined that there was not an ownership change in excess of 50%. Ownership changes in future periods may place additional limits on the Company's ability to utilize net operating loss and tax credit carry forwards. In addition, at the state level, there may be periods during which the use of NOLs is suspended or otherwise limited, which could accelerate or permanently decrease the amount of state attributes and increase state taxes owed. The Company also has research and experimentation and orphan drug credit carryforwards of approximately $30.4 million and $141.8 million, respectively, which will expire in the years 2021 through 2040. Deferred tax assets for these carryforwards are subject to a full valuation allowance. |
Collaborative Agreements
Collaborative Agreements | 12 Months Ended |
Dec. 31, 2020 | |
Collaborative Agreements | |
Collaborative Agreements | Collaborative Agreements University of Pennsylvania In October 2018, as amended, the Company entered into a collaboration agreement with Penn to pursue research and development of novel gene therapies. The Company's gene therapy portfolio pipeline expanded to include Pompe disease, Fabry disease, CDD, CLN1, MPS IIIB, as well as a next generation program in MPS IIIA. This expanded collaboration with Penn also provides the Company with exclusive disease-specific access and option rights to develop potentially disruptive new gene therapy platform technologies and programs for most LDs and a broader portfolio of rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy, and select other muscular dystrophies. Under the expanded collaboration agreement with Penn, Penn is eligible to receive certain milestone, royalty and discovery research payments with respect to licensed products for each indication. Milestone payments are payable following the achievement of certain development and commercial milestone events in each indication, up to an aggregate of $88.0 million per indication. Royalty payments are based on net sales of licensed products on a licensed product-by-licensed product and country- by-country basis. The Company will provide $10.0 million each year during the five GlaxoSmithKline In July 2012, as amended in November 2013, the Company entered into an agreement with GlaxoSmithKline ("GSK"), pursuant to which Amicus obtained global rights to develop and commercialize Galafold ® as a monotherapy and in combination with ERT for Fabry disease (“Collaboration Agreement”). Under the terms of the Collaboration Agreement, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S. The contingent milestone payments due to GSK are recorded within the short term and long term deferred reimbursements accounts on the Consolidated Balance Sheets. Sales based tiered royalties due to GSK are recorded within the cost of goods sold within the Consolidated Statements of Operations. |
Basic and Diluted Net Loss per
Basic and Diluted Net Loss per Common Share | 12 Months Ended |
Dec. 31, 2020 | |
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: Years Ended December 31, (in thousands, except per share amounts) 2020 2019 2018 Numerator: Net loss attributable to common stockholders $ (276,852) $ (356,388) $ (348,995) Denominator: Weighted average common shares outstanding — basic and diluted 258,867,380 240,421,001 185,790,021 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. 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: Years ended December 31, (in thousands) 2020 2019 2018 Options to purchase common stock 14,032 16,724 15,810 Convertible notes 462 462 40,850 Outstanding warrants, convertible to common stock 2,555 2,555 2,657 Unvested restricted stock units 7,080 5,792 3,712 Total number of potentially issuable shares 24,129 25,533 63,029 |
Selected Quarterly Financial Da
Selected Quarterly Financial Data (Unaudited) | 12 Months Ended |
Dec. 31, 2020 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected Quarterly Financial Data (Unaudited) | Selected Quarterly Financial Data (Unaudited) Quarters Ended (in thousands except per share data) March 31 June 30 September 30 December 31 2020 Net product sales $ 60,525 $ 62,353 $ 67,437 $ 70,571 Net loss $ (88,948) $ (52,492) $ (64,011) $ (71,401) Basic and diluted net loss per common share (1) $ (0.35) $ (0.20) $ (0.25) $ (0.27) 2019 Net product sales $ 34,046 $ 44,130 $ 48,768 $ 55,293 Net loss $ (120,299) $ (84,551) $ (61,809) $ (89,729) Basic and diluted net loss per common share (1) $ (0.56) $ (0.36) $ (0.24) $ (0.35) _______________________________ (1) Per common share amounts for the quarters and full years have been calculated separately. Accordingly, quarterly amounts do not add to the annual amounts because of differences on the weighted-average common shares outstanding during each period principally due to the effect of the Company issuing shares of its common stock during the year. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. |
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 December 31, 2020 and through the issuance of this report. 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 comprehensive income (loss) in the 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. |
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 ("EAP"). Orders for Galafold ® are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2020, one customer accounted for 25% of net product sales and 12% of accounts receivable from product sales. In 2019, one customer accounted for 22% of net product sales and 11% of accounts receivable from product sales. 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. |
Fair Value Measurements | Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the FASB guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participants assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability. |
Contingent Liabilities | Contingent Liabilities On an ongoing basis, the Company may be involved in various claims and legal proceedings. On a quarterly basis, the Company reviews the status of each significant matter and assesses its potential financial exposure. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount can be reasonably estimated, the Company will accrue a liability for the estimated loss. Because of uncertainties related to claims and litigation, accruals will be based on the Company's best estimates based on available information. On a periodic basis, as additional information becomes available, or based on specific events such as the outcome of litigation or settlement of claims, the Company may reassess the potential liability related to these matters and may revise these estimates, which could result in material adverse adjustments to the Company's operating results. |
Research and Development Costs | Research and Development Costs Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development. |
Interest Income and Interest Expense | Interest Income and Interest Expense Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases. |
Income Taxes | Income Taxes The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized. |
Other Comprehensive Income (Loss) | Other Comprehensive Income (Loss) Components of other comprehensive income (loss) include unrealized gains and losses on available-for-sale securities and gain (loss) on foreign currency transactions and are included in the Statements of Comprehensive Loss. |
Leases | Leases The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets. ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. The Company uses its incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories. The lease term for all of the Company's leases include the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories. The information presented for the periods prior to January 1, 2019 has not been restated and is reported under the accounting standard in effect for those periods. For additional information, see " -Note 13. Leases". |
Nonqualified Cash Deferral Plan | Nonqualified Cash Deferral Plan The Company's Cash Deferral Plan (the "Deferral Plan"), provides certain key employees and members of the Board of Directors as selected by the Compensation Committee of the Board of Directors of the Company (the "Compensation Committee"), with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code (the "Code"). All of the investments held in the Deferral Plan are classified as investments held-to-maturity and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. The corresponding liability for the Deferral Plan is included in other non-current liability in the Consolidated Balance Sheets. |
Equity-based Compensation | Equity-based Compensation At December 31, 2020, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 9. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award. |
Loss per Common Share | Loss per Common Share The Company calculates net loss per share as a measurement of the Company's performance while giving effect to all dilutive potential common shares that were outstanding during the reporting period. The Company had a net loss for all periods presented; accordingly, the inclusion of common stock options, unvested RSUs, and warrants would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted earnings per share are the same. See "— Note 17. Basic and Diluted Net Loss per Common Share" for further discussion on net loss per share. |
Segment Information | Segment Information The Company currently operates in one business segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker who comprehensively manages the entire business. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Company does not accumulate discrete financial information with respect to separate service lines, and thus there is one reporting unit. |
Business Combinations | Business Combinations The Company assigns fair value to the tangible and intangible assets acquired and liabilities assumed based upon their estimated fair values on the acquisition date from acquired businesses. The purchase price allocation process requires management to make significant estimates and assumptions, especially at the acquisition date with respect to intangible assets and in-process research and development ("IPR&D"). In connection with the purchase price allocations for acquisitions, the Company estimates the fair value of contingent payments utilizing a probability-based income approach inclusive of an estimated discount rate. |
Contingent Consideration Payable | Contingent Consideration Payable Contingent consideration payments in asset acquisitions are recognized when the contingency is resolved and the consideration is paid or becomes payable. This does not apply in circumstances when the contingent consideration meets the definition of a derivative, in which case the amount becomes part of the basis in the asset acquired. Upon recognition of the contingent consideration payment, the amount is included in the cost of the acquired asset or group of assets. The Company determines the fair value of contingent acquisition consideration payable on the acquisition date using a probability-based income approach utilizing an appropriate discount rate. The short term and long term portions of the contingent consideration payable is shown on the Company's Consolidated Balance Sheets. The fair value of the contingent consideration payable will be determined each period end and the resulting change will be recorded on the Consolidated Statements of Operations. |
Intangible Assets and Goodwill | Intangible Assets and GoodwillThe Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Purchased IPR&D is accounted for as an indefinite lived intangible asset until the underlying project is completed, at which point the intangible asset will be accounted for as a definite lived intangible asset, or abandoned, at which point the intangible asset will be written off or partially impaired. Goodwill and indefinite lived intangible assets are assessed annually for impairment and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. If it is determined that the full carrying amount of an asset is not recoverable, an impairment loss is recorded in the amount by which the carrying amount of the asset exceeds its fair value. |
Recent Accounting Developments | Recent Accounting Developments - Guidance Adopted in 2020 ASU 2018-15 - In August 2018, the Financial Accounting Standards Board ("FASB") issued Accounting Standards Update ("ASU") 2018-15, Intangibles-Goodwill and Other-Internal-Use Software (Subtopic 350-40): ("ASU 2018-15"), relating to a customer’s accounting for implementation, set-up, and other upfront costs incurred in a cloud computing arrangement that is hosted by a vendor. Under the new guidance, a customer will apply the same criteria for capitalizing implementation costs as it would for an arrangement that has a software license. The new guidance does not affect the accounting for the service element of a hosting arrangement that is a service contract. The new guidance also prescribes the balance sheet, income statement and cash flow classification of the capitalized software costs and related amortization expense and requires additional quantitative and qualitative disclosures. ASU 2018-15 is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019 for public companies. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. ASU 2018-13 - In August 2018, the FASB issued ASU 2018-03, Fair Value Measurement (Topic 820): Disclosure Framework-Changes to the Disclosure Requirements for Fair Value Measurement ("ASU 2018-13"). The amendments modify the disclosure requirements in Topic 820. ASU 2018-13 is effective for all entities for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. The amendments on (i) changes in unrealized gains and losses, (ii) the range and weighted average of significant unobservable inputs used to develop Level 3 fair value measurements, and (iii) the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. ASU 2017-04 - In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment ("ASU 2017-04"). ASU 2017-04 simplifies the recognition and measurement of a goodwill impairment loss by eliminating Step 2 of the quantitative goodwill impairment test. The guidance requires a one-step impairment test in which an entity compares the fair value of a reporting unit with its carrying amount and recognizes an impairment charge for the amount by which the carrying amount exceeds the reporting unit's fair value, if any. ASU 2017-04 is effective for fiscal years beginning after December 15, 2019 and should be applied on a prospective basis. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. ASU 2016-13 - In June 2016, the FASB issued ASU 2016-13, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments ("ASU 2016-13"). ASU 2016-13 requires financial assets measured at amortized cost basis to be presented at the net amount expected to be collected and amends guidance on the impairment of financial instruments. ASU 2016-13 is effective for public companies who are SEC filers for fiscal years beginning after December 15, 2019, including interim periods within those years. The Company adopted this guidance on January 1, 2020. The adoption did not have a material impact on the Company's Consolidated Financial Statements or related disclosures. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Revenue Disaggregated by Geographical Area | The following table summarizes the Company's net product sales from Galafold ® disaggregated by geographic area: For the Year (in thousands) 2020 2019 2018 U.S. $ 80,046 $ 52,478 $ 7,223 Ex-U.S. 180,840 129,759 84,022 Total net product sales $ 260,886 $ 182,237 $ 91,245 |
Goodwill and Intangible Assets
Goodwill and Intangible Assets (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Schedule of Changes in IPR&D | The following table represents the changes in IPR&D for the years ended December 31, 2020 and 2019, respectively: (in millions) Balance at December 31, 2018 $ 23.0 Change in IPR&D — Balance at December 31, 2019 $ 23.0 Change in IPR&D — Balance at December 31, 2020 $ 23.0 |
Schedule of Changes in Goodwill | The following table represents the changes in Goodwill for the years ended December 31, 2020 and 2019, respectively: (in millions) Balance at December 31, 2018 $ 197.8 Change in goodwill — Balance at December 31, 2019 $ 197.8 Change in goodwill — Balance at December 31, 2020 $ 197.8 |
Cash, Cash Equivalents, Marke_2
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Cash, Cash Equivalents, and Short-term Investments [Abstract] | |
Schedule of Cash and Available for Sale Securities | Cash, cash equivalents, and marketable securities are classified as current unless mentioned otherwise below and consisted of the following: As of December 31, 2020 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 163,240 $ — $ — $ 163,240 Corporate debt securities 39,525 4 (16) 39,513 Commercial paper 217,087 14 (6) 217,095 Asset-backed securities 9,420 18 — 9,438 U.S. government agency bonds 53,583 3 (4) 53,582 Money market 350 — — 350 Certificates of deposit 51 — — 51 $ 483,256 $ 39 $ (26) $ 483,269 Included in cash and cash equivalents $ 163,240 $ — $ — $ 163,240 Included in marketable securities 320,016 39 (26) 320,029 Total cash, cash equivalents, and marketable securities $ 483,256 $ 39 $ (26) $ 483,269 As of December 31, 2019 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 142,837 $ — $ — $ 142,837 Corporate debt securities 145,875 121 (5) 145,991 Commercial paper 73,659 53 (2) 73,710 Asset-backed securities 77,731 79 — 77,810 U.S. government agency bonds 11,999 2 (10) 11,991 Money market 350 — — 350 Certificates of deposit 51 — — 51 $ 452,502 $ 255 $ (17) $ 452,740 Included in cash and cash equivalents $ 142,837 $ — $ — $ 142,837 Included in marketable securities (1) 309,665 255 (17) 309,903 Total cash, cash equivalents, and marketable securities $ 452,502 $ 255 $ (17) $ 452,740 ______________________________ |
Schedule of Reconciliation of Cash and 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. (in thousands) December 31, 2020 December 31, 2019 December 31, 2018 Cash and cash equivalents $ 163,240 $ 142,837 $ 79,749 Restricted cash 2,922 3,506 2,626 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 166,162 $ 146,343 $ 82,375 |
Inventories (Tables)
Inventories (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventories for the Period | The following table summarizes the components of inventories: (in thousands) December 31, 2020 December 31, 2019 Raw materials $ 5,547 $ 6,544 Work-in-process 7,693 3,660 Finished goods 6,316 3,837 Total inventories $ 19,556 $ 14,041 |
Property and Equipment (Tables)
Property and Equipment (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment | Property and equipment consist of the following: December 31, (in thousands) 2020 2019 Property and equipment consist of the following: Computer equipment $ 5,546 $ 6,185 Computer software 1,207 1,607 Research equipment 15,387 14,568 Furniture and fixtures 2,880 5,157 Leasehold improvements 24,146 30,294 Vehicles 124 124 Land 3,190 3,190 Construction in progress 5,870 4,184 Gross property and equipment 58,350 65,309 Less accumulated depreciation (14,487) (17,604) Net property and equipment $ 43,863 $ 47,705 |
Accrued Expenses and Other Cu_2
Accrued Expenses and Other Current Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Payables and Accruals [Abstract] | |
Schedule of Accounts Payable and Accrued Expenses | Accrued expenses and other current liabilities consist of the following: December 31, (in thousands) 2020 2019 Accrued professional fees $ 3,700 $ 7,832 Accrued contract manufacturing & contract research costs 19,617 17,800 Accrued compensation and benefits 23,025 27,156 Accrued program fees 16,599 17,016 Accrued royalties 6,550 5,196 Accrued interest — 3,679 Accrued milestones — 4,000 Accrued sales rebates and discounts 12,079 5,900 Accrued taxes 11,177 7,300 Deferred reimbursements 1,500 1,250 Other 2,594 2,772 $ 96,841 $ 99,901 |
Share based Compensation (Table
Share based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
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: Years Ended December 31, 2020 2019 2018 Expected stock price volatility 75.1 % 74.1 % 78.6 % Risk free interest rate 1.6 % 2.4 % 2.4 % Expected life of options (years) 5.67 5.68 5.62 Expected annual dividend per share $ 0.00 $ 0.00 $ 0.00 |
Schedule of Stock Options Activity | A summary of the Company's stock options for the year ended December 31, 2020 were as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Options outstanding, December 31, 2017 15,181 $ 7.48 Granted 2,348 $ 14.96 Exercised (1,398) $ 6.54 Forfeited (313) $ 9.55 Expired (8) $ 10.76 Options outstanding, December 31, 2018 15,810 $ 8.63 Granted 4,091 $ 10.29 Exercised (1,967) $ 5.83 Forfeited (879) $ 11.07 Expired (331) $ 13.38 Options outstanding, December 31, 2019 16,724 $ 9.15 Granted 4,362 $ 9.98 Exercised (5,243) $ 8.11 Forfeited (1,376) $ 10.42 Expired (435) $ 13.33 Options outstanding, December 31, 2020 14,032 $ 9.54 6.5 $ 190.1 Vested and unvested expected to vest, December 31, 2020 13,267 $ 9.49 6.4 $ 180.5 Exercisable at December 31, 2020 8,130 $ 8.86 5.1 $ 115.7 |
Schedule of Non-Vested RSU Activity under the Plan | A summary of non-vested RSU activity under the Plan for the year ended December 31, 2020 is as follows: Number of Weighted Average Grant Date Fair Value Weighted Average Remaining Years Aggregate Intrinsic Value (in thousands) (in millions) Non-vested units as of December 31, 2017 2,575 $ 5.85 Granted 1,812 $ 16.11 Vested (530) $ 6.01 Forfeited (145) $ 9.65 Non-vested units as of December 31, 2018 3,712 $ 10.59 Granted 3,526 $ 10.92 Vested (921) $ 8.49 Forfeited (525) $ 10.52 Non-vested units as of December 31, 2019 5,792 $ 11.18 Granted 4,692 $ 11.29 Vested (2,164) $ 10.70 Forfeited (1,240) $ 11.14 Non-vested units as of December 31, 2020 7,080 $ 11.35 2.3 $ 158.0 |
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: Years Ended December 31, (in thousands) 2020 2019 2018 Equity compensation expense recognized in: Research and development expense $ 20,817 $ 17,575 $ 11,740 Selling, general, and administrative expense 28,334 26,855 17,520 Total equity compensation expense $ 49,151 $ 44,430 $ 29,260 |
Assets and Liabilities Measur_2
Assets and Liabilities Measured at Fair Value (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
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 December 31, 2020 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 217,095 $ 217,095 Asset-backed securities 9,438 9,438 Corporate debt securities 39,513 39,513 U.S. government agency bonds 53,582 53,582 Money market funds 4,427 4,427 $ 324,055 $ 324,055 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 25,825 $ 25,825 Deferred compensation plan liability 4,078 — 4,078 $ 4,078 $ 25,825 $ 29,903 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, 2019 are identified in the following tables: (in thousands) Level 2 Total Assets: Commercial paper $ 73,710 $ 73,710 Asset-backed securities 77,810 77,810 Corporate debt securities 145,991 145,991 U.S. government agency bonds 11,991 11,991 Money market funds 4,768 4,768 $ 314,270 $ 314,270 (in thousands) Level 2 Level 3 Total Liabilities: Contingent consideration payable $ — $ 22,681 $ 22,681 Deferred compensation plan liability 4,419 — 4,419 $ 4,419 $ 22,681 $ 27,100 |
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 ATB-200 Pompe program: Contingent Consideration Liability Fair Value as of December 31, 2020 Valuation Technique Unobservable Input Range (in thousands) Discount rate 7.5% Clinical and regulatory milestones $ 25,101 Probability weighted discounted cash flow Probability of achievement of milestones 75%-78% Projected year of payments 2021-2022 |
Schedule of Changes in Contingent Consideration Payable | The following table shows the change in the balance of contingent consideration payable for the year ended December 31, 2020 and December 31, 2019, respectively: Years ended December 31, (in thousands) 2020 2019 Balance, beginning of the period $ 22,681 $ 19,700 Changes in fair value during the period, included in the Consolidated Statements of Operations 3,144 3,297 Adjustment for contingent consideration paid in stock — (316) Balance, end of the period $ 25,825 $ 22,681 |
Debt (Tables)
Debt (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Debt Disclosure [Abstract] | |
Schedule of Liability Components of the Convertible Notes | The Company's debt consists of the following: (in thousands) December 31, 2020 December 31, 2019 Senior Secured Term Loan due 2026: Principal $ 400,000 $ — Less: debt discount (1) (7,434) — Less: deferred financing (1) (5,592) — Net carrying value of the Senior Secured Term Loan $ 386,974 $ — Senior Secured Term Loan due 2023 (2) : Principal $ — $ 150,000 Less: debt discount (1) — (2,315) Less: deferred financing (1) — (311) Net carrying value of the Senior Secured Term Loan $ — $ 147,374 Convertible Notes (3) : Principal $ 2,825 $ 2,825 Less: debt discount (1) (518) (659) Less: deferred financing (1) (27) (35) Net carrying value of the Convertible Notes $ 2,280 $ 2,131 Net carrying value of Long-term debt $ 389,254 $ 149,505 ______________________________ (1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Convertible Notes and Senior Secured Term Loans using the effective interest rate method. (2) The principle, accrued interest and early settlement premiums associated with the Senior Secured Term Loan due 2023 were fully paid and settled in July 2020. (3) The Convertible Notes are currently convertible as the last reported sale price of the Company's common stock was equal to or more than 130% of the conversion price for at least 20 trading days of the 30 consecutive trading days ending on the last day of the quarter. |
Components of Total Interest Expense Recognized Related to the Convertible Notes | The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2020 and 2019 respectively: (in thousands) December 31, 2020 December 31, 2019 Contractual interest expense $ 20,933 $ 16,483 Amortization of debt discount $ 1,267 $ 2,381 Amortization of deferred financing $ 527 $ 165 Effective interest rate of the liability component, Convertible debt 10.6 % 10.6 % Effective interest rate of the liability component, Senior Secured Term Loan due 2026 8.4 % — % Effective interest rate of the liability component, Senior Secured Term Loan due 2023 — % 10.2 % |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Lease Cost | Supplemental balance sheet information related to operating leases were as follows: (in thousands, except year and discount rate amounts) December 31, 2020 December 31, 2019 Operating lease ROU asset $ 23,296 $ 33,315 Current portion of the operating lease liabilities $ 6,872 $ 7,189 Non-current portion of the operating lease liabilities 45,604 53,531 Total operating lease liability $ 52,476 $ 60,720 Weighted-average remaining lease terms (years) 19.8 18.2 Weighted-average discount rate 13.0 % 13.1 % |
Future Minimum Operating Lease Payments | At December 31, 2020, the future minimum operating lease payments were as follows: (in thousands) Operating Lease 2021 $ 8,549 2022 8,494 2023 8,691 2024 8,404 2025 8,371 Thereafter 153,241 Total lease payments 195,750 Less lease incentives (28,939) Less imputed interest (114,335) Total operating lease liability $ 52,476 At December 31, 2019, the future minimum operating lease payments were as follows: (in thousands) Operating Lease 2020 $ 9,738 2021 10,787 2022 10,459 2023 10,809 2024 10,582 Thereafter 163,266 Total lease payments $ 215,641 Less lease incentives (28,939) Less imputed interest (125,982) Total operating lease liability $ 60,720 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Schedule of loss before Income Taxes | For financial reporting purposes, loss before income taxes includes the following components: Years Ended December 31, (in thousands) 2020 2019 2018 United States $ (365,332) $ (393,955) $ (309,183) Foreign 91,078 38,045 (39,906) Total $ (274,254) $ (355,910) $ (349,089) |
Schedule of Income Tax Expense (Benefit) | Following were the components of income tax expense (benefit) for the years ended December 31, 2020, 2019, and 2018: (in thousands) 2020 2019 2018 Current Federal $ — $ — $ — State — — 6 Foreign 4,163 2,877 (100) Deferred Federal (1,410) (984) — State (155) (1,415) — Foreign — — — Total $ 2,598 $ 478 $ (94) |
Schedule of Reconciliation of the Statutory Tax Rates and the Effective Tax Rates | A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2020, 2019, and 2018 are as follows: Years Ended 2020 2019 2018 Statutory rate (21) % (21) % (21) % State taxes, net of federal benefit — — (4) Nondeductible IPR&D — — 6 Contingent consideration — — 1 Tax credits (7) (16) (10) Impact of foreign operations 4 9 2 Nondeductible executive compensation 2 1 — Nondeductible debt conversion — (1) — Other — (1) — Valuation allowance 23 29 26 Net 1 % — % — % |
Schedule of Significant Components of the Deferred Tax Assets and Liabilities | The significant components of the deferred tax assets and liabilities are as follows: Years Ended (in thousands) 2020 2019 Deferred tax assets Intellectual property $ 68,209 $ 46,521 Amortization/depreciation 489 2,329 Research tax credit 182,651 141,669 Net operating loss carry forwards 305,876 286,850 Deferred reimbursements 1,912 2,230 Non-cash stock issue 17,553 19,058 Interest carry forward limitation 5,655 2,224 Lease liability 9,235 6,983 Other 10,855 13,921 Gross deferred tax assets 602,435 521,785 Deferred tax liabilities Business acquisition (4,937) (5,051) Royalty payable (68,209) (46,521) Convertible notes (94) (653) Advanced R&D payments (1,776) (2,611) Right of use asset (3,420) (5,643) Total net deferred tax assets 523,999 461,306 Less: valuation allowance (528,895) (466,357) Net deferred tax liability $ (4,896) $ (5,051) |
Basic and Diluted Net Loss pe_2
Basic and Diluted Net Loss per Common Share (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
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: Years Ended December 31, (in thousands, except per share amounts) 2020 2019 2018 Numerator: Net loss attributable to common stockholders $ (276,852) $ (356,388) $ (348,995) Denominator: Weighted average common shares outstanding — basic and diluted 258,867,380 240,421,001 185,790,021 |
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: Years ended December 31, (in thousands) 2020 2019 2018 Options to purchase common stock 14,032 16,724 15,810 Convertible notes 462 462 40,850 Outstanding warrants, convertible to common stock 2,555 2,555 2,657 Unvested restricted stock units 7,080 5,792 3,712 Total number of potentially issuable shares 24,129 25,533 63,029 |
Selected Quarterly Financial _2
Selected Quarterly Financial Data (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of Selected Quarterly Financial Data | Quarters Ended (in thousands except per share data) March 31 June 30 September 30 December 31 2020 Net product sales $ 60,525 $ 62,353 $ 67,437 $ 70,571 Net loss $ (88,948) $ (52,492) $ (64,011) $ (71,401) Basic and diluted net loss per common share (1) $ (0.35) $ (0.20) $ (0.25) $ (0.27) 2019 Net product sales $ 34,046 $ 44,130 $ 48,768 $ 55,293 Net loss $ (120,299) $ (84,551) $ (61,809) $ (89,729) Basic and diluted net loss per common share (1) $ (0.56) $ (0.36) $ (0.24) $ (0.35) _______________________________ (1) Per common share amounts for the quarters and full years have been calculated separately. Accordingly, quarterly amounts do not add to the annual amounts because of differences on the weighted-average common shares outstanding during each period principally due to the effect of the Company issuing shares of its common stock during the year. |
Description of Business (Detail
Description of Business (Details) - USD ($) | 1 Months Ended | ||
Jul. 31, 2020 | Dec. 31, 2020 | Dec. 31, 2019 | |
Debt Instrument [Line Items] | |||
Accumulated deficit | $ 2,045,462,000 | $ 1,768,610,000 | |
Senior Secured Term Loan Due2023 | Senior Loans | |||
Debt Instrument [Line Items] | |||
Early repayment of senior debt | $ 156,300,000 | ||
Secured Debt | Senior Secured Term Loan due 2026 | |||
Debt Instrument [Line Items] | |||
Maximum borrowing capacity | 400,000,000 | ||
Proceeds from secured lines of credit | 385,900,000 | ||
Early repayment of senior debt | $ 156,300,000 | ||
Secured Debt | Senior Secured Term Loan due 2026 | LIBOR | |||
Debt Instrument [Line Items] | |||
Variable rate | 1.00% | ||
Basis spread on variable rate | 6.50% | ||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | |||
Debt Instrument [Line Items] | |||
Maximum borrowing capacity | $ 400,000,000 | ||
Debt instrument term | 6 years | ||
Proceeds from secured lines of credit | $ 385,900,000 | ||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | Minimum | LIBOR | |||
Debt Instrument [Line Items] | |||
Variable rate | 1.00% | ||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | Maximum | LIBOR | |||
Debt Instrument [Line Items] | |||
Basis spread on variable rate | 6.50% |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Concentration of Credit Risk (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Concentration Risk [Line Items] | ||
Allowance for doubtful accounts receivable | $ 0.1 | |
Net product sales | Customer Concentration Risk | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 25.00% | 22.00% |
Accounts receivable | Customer Concentration Risk | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 12.00% | 11.00% |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Property and Equipment (Details) | 12 Months Ended |
Dec. 31, 2020 | |
Minimum | |
Property and Equipment | |
Estimated useful lives | 3 years |
Maximum | |
Property and Equipment | |
Estimated useful lives | 5 years |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Revenue by Geographical Area (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Disaggregation of Revenue [Line Items] | |||||||||||
Net product sales | $ 70,571 | $ 67,437 | $ 62,353 | $ 60,525 | $ 55,293 | $ 48,768 | $ 44,130 | $ 34,046 | $ 260,886 | $ 182,237 | $ 91,245 |
U.S. | |||||||||||
Disaggregation of Revenue [Line Items] | |||||||||||
Net product sales | 80,046 | 52,478 | 7,223 | ||||||||
Ex-U.S. | |||||||||||
Disaggregation of Revenue [Line Items] | |||||||||||
Net product sales | $ 180,840 | $ 129,759 | $ 84,022 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Equity Incentive Plan and Stock-Based Compensation (Details) | Dec. 31, 2020plan |
Equity-based Compensation | |
Number of equity-based employee compensation plans | 1 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Segment Information and Recent Accounting Policies (Details) | 12 Months Ended |
Dec. 31, 2020segmentunit | |
Accounting Policies [Abstract] | |
Number of business segments | segment | 1 |
Number of reporting units | unit | 1 |
Acquisitions (Details)
Acquisitions (Details) $ in Millions | 1 Months Ended | 12 Months Ended |
Sep. 30, 2018USD ($)program | Dec. 31, 2018USD ($) | |
Acquisitions | ||
Number of gene therapy programs for neurologic LSDs developed | program | 10 | |
Celenex | ||
Acquisitions | ||
Contingent consideration based on the achievement of certain milestones | $ 10 | |
Contingent consideration based on the achievement of regulatory milestones | 220 | |
Maximum payment under contingent consideration | 75 | |
Research and development expense | $ 100 | |
Celenex | Nationwide Children's Hospital | ||
Acquisitions | ||
Contingent consideration based on the achievement of certain milestones | $ 7.8 |
Goodwill and Intangible Asset_2
Goodwill and Intangible Assets - Narrative (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Goodwill and Intangible Assets Disclosure [Abstract] | |||
Goodwill | $ 197,797 | $ 197,797 | $ 197,800 |
In-process research & development | $ 23,000 | $ 23,000 |
Goodwill and Intangible Asset_3
Goodwill and Intangible Assets (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Changes in IPR&D | ||
Beginning balance | $ 23,000 | $ 23,000 |
Change in IPR&D | 0 | 0 |
Ending balance | 23,000 | 23,000 |
Changes in goodwill | ||
Beginning balance | 197,797 | 197,800 |
Change in goodwill | 0 | 0 |
Ending balance | $ 197,797 | $ 197,797 |
Cash, Cash Equivalents, Marke_3
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Cash, Cash Equivalents, and Short-term Investments [Abstract] | |||
Cash and cash equivalents | $ 163,240,000 | $ 142,837,000 | $ 79,749,000 |
Debt securities, available-for-sale | 320,029,000 | 309,903,000 | |
Debt securities, realized gain (loss) | 0 | 0 | |
Fair value of available-for-sale debt securities in unrealized loss positions | $ 124,900,000 | $ 42,600,000 |
Cash, Cash Equivalents, Marke_4
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash and Available for Sale Securities (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Debt Securities, Available-for-sale [Line Items] | |||
Cash and cash equivalents | $ 163,240 | $ 142,837 | $ 79,749 |
Cost, debt securities | 320,016 | 309,665 | |
Cost, cash balances and debt securities | 483,256 | 452,502 | |
Gross Unrealized Gain | 39 | 255 | |
Gross Unrealized Loss | (26) | (17) | |
Fair value, cash balances | 163,240 | 142,837 | |
Debt securities, available-for-sale | 320,029 | 309,903 | |
Fair value, cash balances and debt securities | 483,269 | 452,740 | |
Marketable securities with maturity date greater than 12 months | 9,500 | ||
Corporate debt securities | |||
Debt Securities, Available-for-sale [Line Items] | |||
Cost, debt securities | 39,525 | 145,875 | |
Gross Unrealized Gain | 4 | 121 | |
Gross Unrealized Loss | (16) | (5) | |
Debt securities, available-for-sale | 39,513 | 145,991 | |
Commercial paper | |||
Debt Securities, Available-for-sale [Line Items] | |||
Cost, debt securities | 217,087 | 73,659 | |
Gross Unrealized Gain | 14 | 53 | |
Gross Unrealized Loss | (6) | (2) | |
Debt securities, available-for-sale | 217,095 | 73,710 | |
Asset-backed securities | |||
Debt Securities, Available-for-sale [Line Items] | |||
Cost, debt securities | 9,420 | 77,731 | |
Gross Unrealized Gain | 18 | 79 | |
Gross Unrealized Loss | 0 | 0 | |
Debt securities, available-for-sale | 9,438 | 77,810 | |
U.S. government agency bonds | |||
Debt Securities, Available-for-sale [Line Items] | |||
Cost, debt securities | 53,583 | 11,999 | |
Gross Unrealized Gain | 3 | 2 | |
Gross Unrealized Loss | (4) | (10) | |
Debt securities, available-for-sale | 53,582 | 11,991 | |
Money market | |||
Debt Securities, Available-for-sale [Line Items] | |||
Cost, debt securities | 350 | 350 | |
Gross Unrealized Gain | 0 | 0 | |
Gross Unrealized Loss | 0 | 0 | |
Debt securities, available-for-sale | 350 | 350 | |
Certificates of deposit | |||
Debt Securities, Available-for-sale [Line Items] | |||
Cost, debt securities | 51 | 51 | |
Gross Unrealized Gain | 0 | 0 | |
Gross Unrealized Loss | 0 | 0 | |
Debt securities, available-for-sale | $ 51 | $ 51 |
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 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Cash, Cash Equivalents, and Short-term Investments [Abstract] | ||||
Cash and cash equivalents | $ 163,240 | $ 142,837 | $ 79,749 | |
Restricted cash | 2,922 | 3,506 | 2,626 | |
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows | $ 166,162 | $ 146,343 | $ 82,375 | $ 51,237 |
Inventories (Details)
Inventories (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 5,547 | $ 6,544 |
Work-in-process | 7,693 | 3,660 |
Finished goods | 6,316 | 3,837 |
Total inventories | 19,556 | 14,041 |
Reserve for inventory | $ 100 | $ 200 |
Property and Equipment (Details
Property and Equipment (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Property and Equipment | ||
Property and equipment, gross | $ 58,350 | $ 65,309 |
Less accumulated depreciation | (14,487) | (17,604) |
Net property and equipment | 43,863 | 47,705 |
Depreciation and amortization | 8,400 | 4,500 |
Computer equipment | ||
Property and Equipment | ||
Property and equipment, gross | 5,546 | 6,185 |
Computer software | ||
Property and Equipment | ||
Property and equipment, gross | 1,207 | 1,607 |
Research equipment | ||
Property and Equipment | ||
Property and equipment, gross | 15,387 | 14,568 |
Furniture and fixtures | ||
Property and Equipment | ||
Property and equipment, gross | 2,880 | 5,157 |
Leasehold improvements | ||
Property and Equipment | ||
Property and equipment, gross | 24,146 | 30,294 |
Vehicles | ||
Property and Equipment | ||
Property and equipment, gross | 124 | 124 |
Land | ||
Property and Equipment | ||
Property and equipment, gross | 3,190 | 3,190 |
Construction in progress | ||
Property and Equipment | ||
Property and equipment, gross | $ 5,870 | $ 4,184 |
Accrued Expenses and Other Cu_3
Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Accounts payable and accrued expenses | ||
Accrued professional fees | $ 3,700 | $ 7,832 |
Accrued contract manufacturing & contract research costs | 19,617 | 17,800 |
Accrued compensation and benefits | 23,025 | 27,156 |
Accrued program fees | 16,599 | 17,016 |
Accrued royalties | 6,550 | 5,196 |
Accrued interest | 0 | 3,679 |
Accrued milestones | 0 | 4,000 |
Accrued sales rebates and discounts | 12,079 | 5,900 |
Accrued taxes | 11,177 | 7,300 |
Deferred reimbursements | 1,500 | 1,250 |
Other | 2,594 | 2,772 |
Accrued expenses and other current liabilities | $ 96,841 | $ 99,901 |
Stockholders' Equity - Common S
Stockholders' Equity - Common Stock and Warrants (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||
Feb. 28, 2021USD ($)$ / sharesshares | Mar. 31, 2019USD ($)$ / sharesshares | Apr. 30, 2018USD ($)$ / sharesshares | Feb. 28, 2018USD ($)$ / sharesshares | Jun. 30, 2019USD ($)$ / sharesshares | Mar. 31, 2019USD ($)$ / shares | Dec. 31, 2020USD ($)vote$ / sharesshares | Dec. 31, 2019USD ($)$ / sharesshares | Dec. 31, 2018USD ($)shares | Jun. 30, 2018$ / sharesshares | May 31, 2018shares | |
Convertible Debt | |||||||||||
Common stock, shares authorized (in shares) | shares | 500,000,000 | 500,000,000 | 500,000,000 | 250,000,000 | |||||||
Voting right for each share held, number | vote | 1 | ||||||||||
Stock issued from equity financing (in shares) | shares | 18,700,000 | ||||||||||
Gross cash proceeds from warrant exercises | $ 0 | $ 812 | $ 3,617 | ||||||||
Net proceeds from the issuance of common stock after deducting underwriting discounts and commissions | $ 189,000 | $ 0 | $ 188,994 | $ 294,584 | |||||||
Contingent consideration, milestone payment | $ 9,000 | ||||||||||
Milestone payment, stock issued as consideration | 9,000 | ||||||||||
Exchange agreement, aggregate principal amount | $ 247,200 | ||||||||||
Exchange agreement, authorized (in shares) | shares | 44,000,000 | ||||||||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | $ 0.01 | $ 0.01 | |||||||
Common Stock | |||||||||||
Convertible Debt | |||||||||||
Stock issued from equity financing (in shares) | shares | 18,720,930 | 20,239,839 | |||||||||
Milestone payment, stock issued as consideration | $ 9,300 | ||||||||||
Underwritten Offering | |||||||||||
Convertible Debt | |||||||||||
Stock issued (in dollars per share) | $ / shares | $ 10.75 | ||||||||||
Common Stock | |||||||||||
Convertible Debt | |||||||||||
Stock issued from equity financing (in shares) | shares | 20,200,000 | ||||||||||
Net proceeds from the issuance of common stock after deducting underwriting discounts and commissions | $ 294,600 | ||||||||||
Price per share of common stock issued (in dollars per share) | $ / shares | $ 15.50 | ||||||||||
Warrants | |||||||||||
Convertible Debt | |||||||||||
Stock issued from equity financing (in shares) | shares | 101,787 | 453,214 | |||||||||
Gross cash proceeds from warrant exercises | $ 800 | $ 3,600 | |||||||||
Warrants | Common Stock | |||||||||||
Convertible Debt | |||||||||||
Exercise price of warrants (in dollars per share) | $ / shares | $ 7.98 | $ 7.98 | $ 7.98 | ||||||||
Warrants | Subsequent Event | |||||||||||
Convertible Debt | |||||||||||
Stock issued from equity financing (in shares) | shares | 1,050,000 | ||||||||||
Gross cash proceeds from warrant exercises | $ 7,400 | ||||||||||
Warrants | Subsequent Event | Common Stock | |||||||||||
Convertible Debt | |||||||||||
Exercise price of warrants (in dollars per share) | $ / shares | $ 7.06 |
Stockholders' Equity - Nonquali
Stockholders' Equity - Nonqualified Cash Plan and Equity Incentive Plans (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Stockholders' Equity | ||
Deferred compensation investment | $ 4.1 | $ 4.4 |
2007 Plan and 2007 Director Plan | ||
Stockholders' Equity | ||
Expiration period | 10 years | |
Vesting percentage | 100.00% | |
2007 Plan and 2007 Director Plan | Maximum | Options to purchase common stock | ||
Stockholders' Equity | ||
Number of shares reserved for issuance (in shares) | 15,018,368 | |
2007 Plan and 2007 Director Plan | First Anniversary | ||
Stockholders' Equity | ||
Monthly vesting percentage | 2.08% | |
Vesting percentage | 25.00% |
Share based Compensation - Narr
Share based Compensation - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands | Dec. 21, 2018 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Proceeds from exercise of stock options | $ 42,282 | $ 11,476 | $ 9,144 | |
Unvested restricted stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Period following separation for accelerated vesting | 2 years | |||
Unvested restricted stock units | Stock Option Plan Amended Restated 2007 Equity Incentive Plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Unrecognized compensation costs, period for recognition (in years) | 2 years | |||
RSUs vested and non-vested units expected to vest (in shares) | 2,163,732 | 921,000 | 530,000 | |
Unrecognized compensation cost | $ 59,400 | |||
Options to purchase common stock | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Weighted average grant-date fair value, options (in dollars per share) | $ 6.40 | $ 6.67 | $ 10.19 | |
Aggregate intrinsic value of options exercised | $ 40,900 | $ 11,800 | $ 11,900 | |
Proceeds from exercise of stock options | 42,300 | $ 11,500 | $ 9,100 | |
Total unrecognized compensation costs | $ 29,400 | |||
Unrecognized compensation costs, period for recognition (in years) | 3 years |
Share based Compensation - Weig
Share based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected stock price volatility | 75.10% | 74.10% | 78.60% |
Risk free interest rate | 1.60% | 2.40% | 2.40% |
Expected life of options (years) | 5 years 8 months 1 day | 5 years 8 months 4 days | 5 years 7 months 13 days |
Expected annual dividend per share | $ 0 | $ 0 | $ 0 |
Share based Compensation - Stoc
Share based Compensation - Stock Option Activity (Details) - Options to purchase common stock - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Number of Shares | |||
Balance at the beginning of the period (in shares) | 16,724,000 | 15,810,000 | 15,181,000 |
Options, Granted (in shares) | 4,362,000 | 4,091,000 | 2,348,000 |
Options, Exercised (in shares) | (5,243,000) | (1,967,000) | (1,398,000) |
Options, Forfeited (in shares) | (1,376,000) | (879,000) | (313,000) |
Options, Expired (in shares) | (435,000) | (331,000) | (8,000) |
Balance at the end of the period (in shares) | 14,032,000 | 16,724,000 | 15,810,000 |
Vested and unvested expected to vest as of the end of the period (in shares) | 13,267,000 | ||
Exercisable at the end of the period (in shares) | 8,130,000 | ||
Weighted Average Exercise Price | |||
Balance at the beginning of the period (in dollars per share) | $ 9.15 | $ 8.63 | $ 7.48 |
Options, Granted (in dollars per share) | 9.98 | 10.29 | 14.96 |
Options, Exercised (in dollars per share) | 8.11 | 5.83 | 6.54 |
Options, Forfeited (in dollars per share) | 10.42 | 11.07 | 9.55 |
Options, Expired (in dollars per share) | 13.33 | 13.38 | 10.76 |
Balance at the end of the period (in dollars per share) | 9.54 | $ 9.15 | $ 8.63 |
Vested and unvested expected to vest as of the end of the period (in dollars per share) | 9.49 | ||
Exercisable at the end of the period (in dollars per share) | $ 8.86 | ||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | |||
Balance at the end of the period | 6 years 6 months | ||
Vested and unvested expected to vest at the end of the period | 6 years 4 months 24 days | ||
Exercisable at the end of the period | 5 years 1 month 6 days | ||
Aggregate intrinsic value of options outstanding | $ 190.1 | ||
Aggregate intrinsic value of options vested and unvested expected to vest | 180.5 | ||
Aggregate intrinsic value of options exercisable | $ 115.7 |
Share based Compensation - RSUs
Share based Compensation - RSUs and PBRSUs Summary (Details) - Stock Option Plan Amended Restated 2007 Equity Incentive Plan - Unvested restricted stock units - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Number of Shares | |||
Non-vested units as of the beginning of the period (in shares) | 5,792,000 | 3,712,000 | 2,575,000 |
Granted (in shares) | 4,692,000 | 3,526,000 | 1,812,000 |
Vested (in shares) | (2,163,732) | (921,000) | (530,000) |
Forfeited (in shares) | (1,240,000) | (525,000) | (145,000) |
Non-vested units as of the end of the period (in shares) | 7,080,000 | 5,792,000 | 3,712,000 |
Weighted Average Grant Date Fair Value | |||
Non-vested units as of the beginning of the period (in dollars per share) | $ 11.18 | $ 10.59 | $ 5.85 |
Granted (in dollars per share) | 11.29 | 10.92 | 16.11 |
Vested (in dollars per share) | 10.70 | 8.49 | 6.01 |
Forfeited (in dollars per share) | 11.14 | 10.52 | 9.65 |
Non-vested units as of the end of the period (in dollars per share) | $ 11.35 | $ 11.18 | $ 10.59 |
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Additional Disclosures [Abstract] | |||
Non-vested units, weighted average remaining years | 2 years 3 months 18 days | ||
Non-vested units, aggregate intrinsic value | $ 158 |
Share based Compensation - Expe
Share based Compensation - Expense Summary (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total equity compensation expense | $ 49,151 | $ 44,430 | $ 29,260 |
Research and development expense | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total equity compensation expense | 20,817 | 17,575 | 11,740 |
Selling, general, and administrative expense | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total equity compensation expense | $ 28,334 | $ 26,855 | $ 17,520 |
Assets and Liabilities Measur_3
Assets and Liabilities Measured at Fair Value (Details) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2019USD ($) | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | |
Assets: | |||
Fair value of assets | $ 324,055 | $ 314,270 | |
Liabilities: | |||
Contingent consideration payable | 25,825 | 22,681 | |
Deferred compensation plan liability | 4,078 | 4,419 | |
Fair value of liabilities | 29,903 | 27,100 | |
Milestone payment, stock issued as consideration | $ 9,000 | ||
Common Stock | |||
Liabilities: | |||
Milestone payment, stock issued as consideration | $ 9,300 | ||
ATB-200 Pompe Program | Clinical and Regulatory Approval Milestones | Callidus | |||
Liabilities: | |||
Contingent consideration payable | $ 25,101 | ||
ATB-200 Pompe Program | Clinical and Regulatory Approval Milestones | Callidus | Probability of Milestone Achievement | Minimum | |||
Liabilities: | |||
Contingent consideration, measurement input | 0.75 | ||
ATB-200 Pompe Program | Clinical and Regulatory Approval Milestones | Callidus | Probability of Milestone Achievement | Maximum | |||
Liabilities: | |||
Contingent consideration, measurement input | 0.78 | ||
Convertible Notes | 2016 Convertible Notes | |||
Liabilities: | |||
Fair value of the debt | $ 10,900 | ||
Commercial paper | |||
Assets: | |||
Fair value of assets | 217,095 | 73,710 | |
Asset-backed securities | |||
Assets: | |||
Fair value of assets | 9,438 | 77,810 | |
Corporate debt securities | |||
Assets: | |||
Fair value of assets | 39,513 | 145,991 | |
U.S. government agency bonds | |||
Assets: | |||
Fair value of assets | 53,582 | 11,991 | |
Money market | |||
Assets: | |||
Fair value of assets | 4,427 | 4,768 | |
Level 2 | |||
Assets: | |||
Fair value of assets | 324,055 | 314,270 | |
Liabilities: | |||
Contingent consideration payable | 0 | 0 | |
Deferred compensation plan liability | 4,078 | 4,419 | |
Fair value of liabilities | 4,078 | 4,419 | |
Level 2 | Commercial paper | |||
Assets: | |||
Fair value of assets | 217,095 | 73,710 | |
Level 2 | Asset-backed securities | |||
Assets: | |||
Fair value of assets | 9,438 | 77,810 | |
Level 2 | Corporate debt securities | |||
Assets: | |||
Fair value of assets | 39,513 | 145,991 | |
Level 2 | U.S. government agency bonds | |||
Assets: | |||
Fair value of assets | 53,582 | 11,991 | |
Level 2 | Money market | |||
Assets: | |||
Fair value of assets | 4,427 | 4,768 | |
Level 3 | |||
Liabilities: | |||
Contingent consideration payable | 25,825 | 22,681 | |
Deferred compensation plan liability | 0 | 0 | |
Fair value of liabilities | $ 25,825 | $ 22,681 | |
Level 3 | ATB-200 Pompe Program | Clinical and Regulatory Approval Milestones | Probability Weighted Discounted Cash Flow | Callidus | Discount Rate | |||
Liabilities: | |||
Contingent consideration, measurement input | 0.075 |
Assets and Liabilities Measur_4
Assets and Liabilities Measured at Fair Value - Level 3 Roll Forward (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | |||
Balance, beginning of the period | $ 22,681 | $ 19,700 | |
Changes in fair value during the period, included in the Consolidated Statements of Operations | 3,144 | 3,297 | $ 3,300 |
Adjustment for contingent consideration paid in stock | 0 | (316) | |
Balance, end of the period | $ 25,825 | $ 22,681 | $ 19,700 |
Debt - Summary of Long Term Deb
Debt - Summary of Long Term Debt (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Dec. 31, 2016day | Dec. 31, 2020USD ($)day | Dec. 31, 2019USD ($) | |
Debt Instruments | |||
Long-term Debt, Total | $ 389,254 | $ 149,505 | |
Convertible Notes | Senior Secured Term Loan due 2026 | |||
Debt Instruments | |||
Principal | 400,000 | 0 | |
Less: debt discount | (7,434) | 0 | |
Less: deferred financing | (5,592) | 0 | |
Long-term Debt, Total | 386,974 | 0 | |
Convertible Notes | Senior Secured Term Loan Due2023 | |||
Debt Instruments | |||
Principal | 0 | 150,000 | |
Less: debt discount | 0 | (2,315) | |
Less: deferred financing | 0 | (311) | |
Long-term Debt, Total | 0 | 147,374 | |
Convertible Notes | Convertible Senior Notes 2016, Due 2023 | |||
Debt Instruments | |||
Principal | 2,825 | 2,825 | |
Less: debt discount | (518) | (659) | |
Less: deferred financing | (27) | (35) | |
Long-term Debt, Total | $ 2,280 | $ 2,131 | |
Threshold percentage | 130.00% | 130.00% | |
Threshold trading days | 20 days | ||
Threshold consecutive trading days | day | 30 | 30 |
Debt - Narrative (Details)
Debt - Narrative (Details) | 1 Months Ended | 6 Months Ended | 12 Months Ended | |||||
Jul. 31, 2020USD ($)day | Sep. 30, 2018USD ($) | Dec. 31, 2016USD ($)day$ / sharesshares | Jun. 30, 2019USD ($)$ / sharesshares | Dec. 31, 2020USD ($)day$ / shares | Dec. 31, 2019USD ($)$ / shares | Dec. 31, 2018USD ($) | Jun. 30, 2018$ / shares | |
Convertible Debt | ||||||||
Loss on extinguishment of debt | $ 7,276,000 | $ 0 | $ 0 | |||||
Proceeds from long-term debt, net of issuance costs | $ 385,929,000 | $ 0 | 146,596,000 | |||||
Exchange agreement, aggregate principal amount | $ 247,200,000 | |||||||
Exchange agreement, authorized (in shares) | shares | 44,000,000 | |||||||
Common stock, par value (in dollars per share) | $ / shares | $ 0.01 | $ 0.01 | $ 0.01 | $ 0.01 | ||||
Accrued and unpaid interest on exchange agreement | $ 1,300,000 | |||||||
Gain (loss) on extinguishment of debt, additional paid-in capital | $ 215,000,000 | |||||||
Gain (loss) on extinguishment of debt, fair value of common shares issued | 400,000 | |||||||
Loss on exchange of convertible notes | $ 0 | 40,624,000 | 0 | |||||
Proceeds from capped call confirmations, exchange of convertible debt | $ 19,900,000 | $ 0 | $ (19,875,000) | $ 0 | ||||
2016 Convertible Notes | Convertible Notes | ||||||||
Convertible Debt | ||||||||
Aggregate principal amount | $ 250,000,000 | |||||||
Proceeds from long-term debt, net of issuance costs | 243,000,000 | |||||||
Proceeds for capped call confirmations | $ 13,500,000 | |||||||
Shares issued per increment of convertible debt (in shares) | shares | 40,800,000 | |||||||
Debt conversion ratio (in shares) | 0.1633987 | |||||||
Conversion price (in dollars per share) | $ / shares | $ 6.12 | |||||||
Threshold trading days | 20 days | |||||||
Threshold consecutive trading days | day | 30 | 30 | ||||||
Threshold percentage | 130.00% | 130.00% | ||||||
Period following 5 consecutive trading day period, trading price less than 98% of product of sales price | 5 years | |||||||
Period following issuance of stock rights options or warrants with price per share less than last reported sales price | 60 days | |||||||
Period for measurement of sales price | 10 days | |||||||
Excess of value per share for triggering event, percent | 10.00% | |||||||
2016 Convertible Notes | Convertible Notes | Private Placement Purchase Agreement | Over-allotment Option | ||||||||
Convertible Debt | ||||||||
Aggregate principal amount | $ 25,000,000 | |||||||
2016 Convertible Notes | Convertible Notes | Minimum | ||||||||
Convertible Debt | ||||||||
Threshold trading days | 20 days | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | ||||||||
Convertible Debt | ||||||||
Maximum borrowing capacity | $ 400,000,000 | |||||||
Number of payments | day | 9 | |||||||
Periodic payment | $ 44,400,000 | |||||||
Proceeds from secured lines of credit | 385,900,000 | |||||||
Early repayment of senior debt | 156,300,000 | |||||||
Interest expense, debt | 1,100,000 | |||||||
Early settlement premiums | 5,200,000 | |||||||
Loss on extinguishment of debt | $ 7,300,000 | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | LIBOR | ||||||||
Convertible Debt | ||||||||
Variable rate | 1.00% | |||||||
Basis spread on variable rate | 6.50% | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | ||||||||
Convertible Debt | ||||||||
Maximum borrowing capacity | $ 400,000,000 | |||||||
Proceeds from secured lines of credit | 385,900,000 | |||||||
Minimum liquidity covenant | $ 75,000,000 | |||||||
Debt instrument term | 6 years | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | Minimum | ||||||||
Convertible Debt | ||||||||
Consolidated revenue covenant, amount | $ 140,000,000 | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | Maximum | ||||||||
Convertible Debt | ||||||||
Consolidated revenue covenant, amount | $ 225,000,000 | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | LIBOR | Minimum | ||||||||
Convertible Debt | ||||||||
Variable rate | 1.00% | |||||||
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | LIBOR | Maximum | ||||||||
Convertible Debt | ||||||||
Basis spread on variable rate | 6.50% | |||||||
Line of Credit | ||||||||
Convertible Debt | ||||||||
Maximum borrowing capacity | $ 150,000,000 | |||||||
Debt instrument term | 5 years | |||||||
Periodic payment, percentage | 12.50% | |||||||
Proceeds from issuance of debt | $ 146,600,000 | |||||||
Line of Credit | LIBOR | ||||||||
Convertible Debt | ||||||||
Basis spread on variable rate | 7.50% |
Debt - Interest Expense (Detail
Debt - Interest Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Convertible Notes | 2016 Convertible Notes | ||
Interest expense | ||
Contractual interest expense | $ 20,933 | $ 16,483 |
Amortization of debt discount | 1,267 | 2,381 |
Amortization of deferred financing | $ 527 | $ 165 |
Effective interest rate of the liability component | 10.60% | 10.60% |
Senior Notes | ||
Interest expense | ||
Effective interest rate of the liability component | 8.40% | 10.20% |
Leases (Details)
Leases (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Leases [Abstract] | |||
Operating lease cost | $ 11,100 | $ 10,800 | |
Cash paid for amounts included in operating lease liabilities | 9,600 | 4,900 | |
Right-of-use assets obtained in exchange for new operating lease liabilities | 1,700 | 21,800 | |
Tenant improvements paid through lease incentive | 470 | 19,388 | $ 0 |
Operating lease ROU asset | 23,296 | 33,315 | |
Current portion of the operating lease liabilities | 6,872 | 7,189 | |
Non-current portion of the operating lease liabilities | 45,604 | 53,531 | |
Operating lease liability | $ 52,476 | $ 60,720 | |
Weighted-average remaining lease terms (years) | 19 years 9 months 18 days | 18 years 2 months 12 days | |
Weighted-average discount rate | 13.00% | 13.10% | |
Operating Lease | |||
2021 | $ 8,549 | $ 9,738 | |
2022 | 8,494 | 10,787 | |
2023 | 8,691 | 10,459 | |
2024 | 8,404 | 10,809 | |
2025 | 8,371 | 10,582 | |
Thereafter | 153,241 | 163,266 | |
Total lease payments | 195,750 | 215,641 | |
Lessee, Operating Lease, Liability, Tenant Incentives | (28,939) | (28,939) | |
Lessee, Operating Lease, Liability, Undiscounted Excess Amount | (114,335) | (125,982) | |
Total operating lease liability | $ 52,476 | $ 60,720 | |
Rent expense, including fees for utilities and common area maintenances | $ 5,700 |
Income Taxes - Statutory Rate (
Income Taxes - Statutory Rate (Details) - USD ($) | Dec. 22, 2017 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Income (loss) before income taxes: | ||||
United States | $ (365,332,000) | $ (393,955,000) | $ (309,183,000) | |
Foreign | 91,078,000 | 38,045,000 | (39,906,000) | |
Loss before income tax | (274,254,000) | (355,910,000) | (349,089,000) | |
Current | ||||
Federal | 0 | 0 | 0 | |
State | 0 | 0 | 6,000 | |
Foreign | 4,163,000 | 2,877,000 | (100,000) | |
Deferred | ||||
Federal | (1,410,000) | (984,000) | 0 | |
State | (155,000) | (1,415,000) | 0 | |
Foreign | 0 | 0 | 0 | |
Income tax expense (benefit) | $ 2,598,000 | $ 478,000 | $ (94,000) | |
Reconciliation of the statutory tax rates and the effective tax rates | ||||
Statutory rate | (21.00%) | (21.00%) | (21.00%) | (21.00%) |
State taxes, net of federal benefit | 0.00% | 0.00% | (4.00%) | |
Nondeductible IPR&D | 0.00% | 0.00% | 6.00% | |
Contingent consideration | 0.00% | 0.00% | 1.00% | |
Tax credits | (7.00%) | (16.00%) | (10.00%) | |
Impact of foreign operations | 4.00% | 9.00% | 2.00% | |
Nondeductible executive compensation | 2.00% | 1.00% | 0.00% | |
Nondeductible debt conversion | 0.00% | (1.00%) | 0.00% | |
Other | 0.00% | (1.00%) | 0.00% | |
Valuation allowance | 23.00% | 29.00% | 26.00% | |
Net | 1.00% | 0.00% | 0.00% | |
Deferred federal, state and local, tax expense (benefit) | $ (1,600,000) | |||
Accrual for interest and penalties | 0 | |||
Accrual for uncertain tax positions | $ 0 |
Income Taxes - Deferred and Val
Income Taxes - Deferred and Valuation (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Deferred tax assets | ||
Intellectual property | $ 68,209 | $ 46,521 |
Amortization/depreciation | 489 | 2,329 |
Research tax credit | 182,651 | 141,669 |
Net operating loss carry forwards | 305,876 | 286,850 |
Deferred reimbursements | 1,912 | 2,230 |
Non-cash stock issue | 17,553 | 19,058 |
Interest carry forward limitation | 5,655 | 2,224 |
Lease liability | 9,235 | 6,983 |
Other | 10,855 | 13,921 |
Gross deferred tax assets | 602,435 | 521,785 |
Deferred tax liabilities | ||
Business acquisition | (4,937) | (5,051) |
Royalty payable | (68,209) | (46,521) |
Convertible notes | (94) | (653) |
Advanced R&D payments | (1,776) | (2,611) |
Right of use asset | (3,420) | (5,643) |
Total net deferred tax assets | 523,999 | 461,306 |
Less: valuation allowance | (528,895) | (466,357) |
Net deferred tax liability | (4,896) | $ (5,051) |
Increase in the valuation allowance | $ 62,500 |
Income Taxes - Loss Carryforwar
Income Taxes - Loss Carryforwards (Details) $ in Millions | Dec. 31, 2020USD ($) |
Research and experimentation tax credit carryforward | |
Operating Loss Carryforwards [Line Items] | |
Tax credit carryforward amount | $ 30.4 |
Orphan drug tax credit carryforwards | |
Operating Loss Carryforwards [Line Items] | |
Tax credit carryforward amount | 141.8 |
Federal | |
Operating Loss Carryforwards [Line Items] | |
Amount of net operating loss carry forwards | 1,156 |
State | |
Operating Loss Carryforwards [Line Items] | |
Amount of net operating loss carry forwards | $ 889.4 |
Collaborative Agreements (Detai
Collaborative Agreements (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||||
Oct. 31, 2018USD ($) | Nov. 30, 2013USD ($)market | Dec. 31, 2020USD ($) | Sep. 30, 2020USD ($) | Jun. 30, 2020USD ($) | Mar. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | |
Collaborative Agreements | |||||||||||||
Revenue recognized | $ 70,571 | $ 67,437 | $ 62,353 | $ 60,525 | $ 55,293 | $ 48,768 | $ 44,130 | $ 34,046 | $ 260,886 | $ 182,237 | $ 91,245 | ||
Deferred reimbursements | 7,406 | 8,906 | 7,406 | 8,906 | |||||||||
Accrued expenses and other current liabilities | 96,841 | $ 99,901 | 96,841 | $ 99,901 | |||||||||
Collaborative Arrangements | |||||||||||||
Collaborative Agreements | |||||||||||||
Conditional aggregate payments per indication | $ 88,000 | ||||||||||||
Discovery program fee | $ 10,000 | ||||||||||||
Term of agreement | 5 years | ||||||||||||
Payments for collaborative agreement | $ 7,000 | ||||||||||||
Collaborative Arrangements | GSK | |||||||||||||
Collaborative Agreements | |||||||||||||
Payments for collaborative agreement | 1,300 | ||||||||||||
Revised Agreement | GSK | |||||||||||||
Collaborative Agreements | |||||||||||||
Potential milestone payments upon achievement of post-approval and sales-based milestones | $ 40,000 | ||||||||||||
Number of major markets outside the U.S. from whom parties to contractual arrangement is eligible to receive post-approval and sales-based milestones | market | 8 | ||||||||||||
Revenue recognized | 21,700 | ||||||||||||
Deferred reimbursements | 8,900 | 8,900 | |||||||||||
Accrued expenses and other current liabilities | $ 6,600 | $ 6,600 |
Basic and Diluted Net Loss pe_3
Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Numerator: | |||||||||||
Net loss attributable to common stockholders | $ (71,401) | $ (64,011) | $ (52,492) | $ (88,948) | $ (89,729) | $ (61,809) | $ (84,551) | $ (120,299) | $ (276,852) | $ (356,388) | $ (348,995) |
Denominator: | |||||||||||
Weighted-average common shares outstanding - basic and diluted (in shares) | 258,867,380 | 240,421,001 | 185,790,021 | ||||||||
Antidilutive securities | |||||||||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 24,129,000 | 25,533,000 | 63,029,000 | ||||||||
Options to purchase common stock | |||||||||||
Antidilutive securities | |||||||||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 14,032,000 | 16,724,000 | 15,810,000 | ||||||||
Convertible notes | |||||||||||
Antidilutive securities | |||||||||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 462,000 | 462,000 | 40,850,000 | ||||||||
Outstanding warrants, convertible to common stock | |||||||||||
Antidilutive securities | |||||||||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 2,555,000 | 2,555,000 | 2,657,000 | ||||||||
Unvested restricted stock units | |||||||||||
Antidilutive securities | |||||||||||
Antidilutive securities excluded from computation of diluted earnings per share (in shares) | 7,080,000 | 5,792,000 | 3,712,000 |
Selected Quarterly Financial _3
Selected Quarterly Financial Data (Unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Selected Quarterly Financial Data | |||||||||||
Net product sales | $ 70,571 | $ 67,437 | $ 62,353 | $ 60,525 | $ 55,293 | $ 48,768 | $ 44,130 | $ 34,046 | $ 260,886 | $ 182,237 | $ 91,245 |
Net loss | $ (71,401) | $ (64,011) | $ (52,492) | $ (88,948) | $ (89,729) | $ (61,809) | $ (84,551) | $ (120,299) | $ (276,852) | $ (356,388) | $ (348,995) |
Basic and diluted net loss per common share (in dollars per share) | $ (0.27) | $ (0.25) | $ (0.20) | $ (0.35) | $ (0.35) | $ (0.24) | $ (0.36) | $ (0.56) | $ (1.07) | $ (1.48) | $ (1.88) |
Uncategorized Items - fold-2020
Label | Element | Value |
Accounting Standards Update [Extensible List] | us-gaap_AccountingStandardsUpdateExtensibleList | us-gaap:AccountingStandardsUpdate201802Member |