uniQure N.V. 10-Q

Item 1. Financial Statements uniQure N.V. ​ UNAUDITED CONSOLIDATED BALANCE SHEETS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ September 30, ​ December 31, ​ 2022 2021 ​ ​ (in thousands, except share and per share amounts) Current assets ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 440,313 ​ $ 556,256 Accounts receivable and contract asset ​ ​ 3,603 ​ ​ 58,768 Inventories ​ ​ 4,075 ​ ​ - Prepaid expenses ​ ​ 13,692 ​ ​ 10,540 Other current assets and receivables ​ ​ 2,894 ​ ​ 2,675 Total current assets ​ ​ 464,577 ​ ​ 628,239 Non-current assets ​ ​ ​ ​ ​ ​ Property, plant and equipment, net of accumulated depreciation of $ 40.5 million as of September 30, 2022 and $ 36.9 million as of December 31, 2021 ​ ​ 47,886 ​ ​ 43,505 Operating lease right-of-use assets ​ ​ 27,804 ​ ​ 25,573 Intangible assets, net, including an in-process research and development asset of $ 52.5 million as of September 30, 2022 and $ 60.8 million as of December 31, 2021 ​ ​ 53,837 ​ ​ 62,686 Goodwill ​ ​ 23,418 ​ ​ 27,633 Deferred tax assets, net ​ ​ 14,627 ​ ​ 15,647 Other non-current assets ​ ​ 6,085 ​ ​ 5,897 Total non-current assets ​ ​ 173,657 ​ ​ 180,941 Total assets ​ $ 638,234 ​ $ 809,180 Current liabilities ​ ​ ​ ​ ​ ​ Accounts payable ​ $ 7,566 ​ $ 2,502 Accrued expenses and other current liabilities ​ ​ 26,437 ​ ​ 28,487 Current portion of contingent consideration ​ ​ 23,537 ​ ​ — Current portion of operating lease liabilities ​ ​ 6,434 ​ ​ 5,774 Total current liabilities ​ ​ 63,974 ​ ​ 36,763 Non-current liabilities ​ ​ ​ ​ ​ ​ Long-term debt ​ ​ 102,394 ​ ​ 100,963 Operating lease liabilities, net of current portion ​ ​ 29,893 ​ ​ 28,987 Contingent consideration, net of current portion ​ ​ 9,158 ​ ​ 29,542 Deferred tax liability, net ​ ​ 8,592 ​ ​ 12,913 Other non-current liabilities ​ ​ 3,053 ​ ​ 4,236 Total non-current liabilities ​ ​ 153,090 ​ ​ 176,641 Total liabilities ​ ​ 217,064 ​ ​ 213,404 Commitments and contingencies ​ ​ ​ ​ ​ ​ Shareholders' equity ​ ​ ​ ​ ​ ​ Ordinary shares, € 0.05 par value: 80,000,000 shares authorized as of September 30, 2022 and December 31, 2021 and 46,815,109 and 46,298,635 ordinary shares issued and outstanding as of September 30, 2022 and December 31, 2021, respectively ​ ​ 2,830 ​ ​ 2,802 Additional paid-in-capital ​ ​ 1,100,078 ​ ​ 1,076,972 Accumulated other comprehensive loss ​ ​ ( 93,000 ) ​ ​ ( 28,856 ) Accumulated deficit ​ ​ ( 588,738 ) ​ ​ ( 455,142 ) Total shareholders' equity ​ ​ 421,170 ​ ​ 595,776 Total liabilities and shareholders' equity ​ $ 638,234 ​ $ 809,180 ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ 2 Table of Contents ​ uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE (LOSS) INCOME ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended September 30, ​ Nine months ended September 30, ​ 2022 2021 2022 2021 ​ ​ (in thousands, except share and per share amounts) ​ (in thousands, except share and per share amounts) License revenues ​ $ — ​ $ — ​ $ — ​ $ 462,400 Collaboration revenues ​ ​ 1,449 ​ ​ 1,989 ​ ​ 3,738 ​ ​ 3,911 Total revenues ​ ​ 1,449 ​ ​ 1,989 ​ ​ 3,738 ​ ​ 466,311 Operating expenses: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cost of contract revenues ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 23,178 ) Cost of contract manufacturing ​ ​ ( 861 ) ​ ​ — ​ ​ ( 1,693 ) ​ ​ — Research and development expenses ​ ​ ( 48,068 ) ​ ​ ( 36,432 ) ​ ​ ( 139,263 ) ​ ​ ( 101,209 ) Selling, general and administrative expenses ​ ​ ( 13,324 ) ​ ​ ( 12,023 ) ​ ​ ( 36,802 ) ​ ​ ( 42,323 ) Total operating expenses ​ ​ ( 62,253 ) ​ ​ ( 48,455 ) ​ ​ ( 177,758 ) ​ ​ ( 166,710 ) Other income ​ ​ 1,485 ​ ​ 1,680 ​ ​ 4,981 ​ ​ 9,622 Other expense ​ ​ ( 199 ) ​ ​ ( 214 ) ​ ​ ( 621 ) ​ ​ ( 673 ) (Loss) / income from operations ​ ​ ( 59,518 ) ​ ​ ( 45,000 ) ​ ​ ( 169,660 ) ​ ​ 308,550 Interest income ​ ​ 39 ​ ​ 46 ​ ​ 117 ​ ​ 123 Interest expense ​ ​ ( 3,069 ) ​ ​ ( 1,924 ) ​ ​ ( 8,279 ) ​ ​ ( 5,377 ) Foreign currency gains, net ​ ​ 14,362 ​ ​ 10,436 ​ ​ 42,328 ​ ​ 21,645 Other non-operating (losses) / gains, net ​ ​ — ​ ​ — ​ ​ 635 ​ ​ — (Loss) / income before income tax benefit / (expense) ​ $ ( 48,186 ) ​ $ ( 36,442 ) ​ $ ( 134,859 ) ​ $ 324,941 Income tax benefit / (expense) ​ ​ 329 ​ ​ ( 89 ) ​ ​ 1,263 ​ ​ ( 3,560 ) Net (loss) / income ​ $ ( 47,857 ) ​ $ ( 36,531 ) ​ $ ( 133,596 ) ​ $ 321,381 Other comprehensive loss: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Foreign currency translation adjustments ​ ​ ( 25,370 ) ​ ​ ( 13,288 ) ​ ​ ( 64,144 ) ​ ​ ( 27,790 ) Total comprehensive (loss) / gain ​ $ ( 73,227 ) ​ $ ( 49,819 ) ​ $ ( 197,740 ) ​ $ 293,591 Earnings per ordinary share - basic ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Basic net (loss) / income per ordinary share ​ $ ( 1.02 ) ​ $ ( 0.79 ) ​ $ ( 2.86 ) ​ $ 7.00 Earnings per ordinary share - diluted ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Diluted net (loss) / income per ordinary share ​ $ ( 1.02 ) ​ $ ( 0.79 ) ​ $ ( 2.86 ) ​ $ 6.87 Weighted average shares - basic ​ ​ 46,772,430 ​ ​ 46,152,404 ​ ​ 46,680,667 ​ ​ 45,888,769 Weighted average shares - diluted ​ ​ 46,772,430 ​ ​ 46,152,404 ​ ​ 46,680,667 ​ ​ 46,780,963 ​ ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ 3 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY FOR THE THREE-MONTH PERIOD ENDED SEPTEMBER 30, 2022 AND 2021 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ other ​ ​ ​ ​ Total ​ Ordinary shares ​ paid-in ​ comprehensive ​ Accumulated ​ shareholders’ ​ No. of shares Amount capital (loss) deficit equity ​ (in thousands, except share and per share amounts) Balance at June 30, 2021 46,050,250 ​ $ 2,788 ​ $ 1,062,234 ​ $ ( 4,595 ) ​ $ ( 426,819 ) ​ $ 633,608 Loss for the period — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 36,531 ) ​ ​ ( 36,531 ) Other comprehensive loss — ​ ​ — ​ ​ — ​ ​ ( 13,288 ) ​ ​ — ​ ​ ( 13,288 ) Exercise of share options 84,811 ​ ​ 5 ​ ​ 909 ​ ​ — ​ ​ — ​ ​ 914 Restricted share units distributed during the period 69,165 ​ ​ 4 ​ ​ ( 4 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense — ​ ​ — ​ ​ 5,955 ​ ​ — ​ ​ — ​ ​ 5,955 Issuance of ordinary shares relating to employee stock purchase plan 3,550 ​ ​ — ​ ​ 97 ​ ​ — ​ ​ — ​ ​ 97 Balance at September 30, 2021 46,207,776 ​ $ 2,797 ​ $ 1,069,191 ​ $ ( 17,883 ) ​ $ ( 463,350 ) ​ $ 590,755 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at June 30, 2022 46,684,583 ​ $ 2,823 ​ $ 1,092,176 ​ $ ( 67,630 ) ​ $ ( 540,881 ) ​ $ 486,488 Loss for the period — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 47,857 ) ​ ​ ( 47,857 ) Other comprehensive loss — ​ ​ — ​ ​ — ​ ​ ( 25,370 ) ​ ​ — ​ ​ ( 25,370 ) Exercise of share options 47,290 ​ ​ 2 ​ ​ 258 ​ ​ — ​ ​ — ​ ​ 260 Restricted and performance share units distributed during the period 79,821 ​ ​ 4 ​ ​ ( 4 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense — ​ ​ — ​ ​ 7,608 ​ ​ — ​ ​ — ​ ​ 7,608 Issuance of ordinary shares relating to employee stock purchase plan 3,415 ​ ​ 1 ​ ​ 40 ​ ​ — ​ ​ — ​ ​ 41 Balance at September 30, 2022 46,815,109 ​ $ 2,830 ​ $ 1,100,078 ​ $ ( 93,000 ) ​ $ ( 588,738 ) ​ $ 421,170 ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ 4 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY FOR THE NINE-MONTH PERIOD ENDED SEPTEMBER 30, 2022 AND 2021 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ other ​ ​ ​ ​ Total ​ ​ Ordinary shares ​ paid-in ​ comprehensive ​ Accumulated ​ shareholders’ ​ ​ No. of shares Amount capital income / (loss) deficit equity ​ ​ (in thousands, except share and per share amounts) Balance at December 31, 2020 ​ 44,777,799 ​ $ 2,711 ​ $ 1,016,018 ​ $ 9,907 ​ $ ( 784,731 ) ​ $ 243,905 Income for the period ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ 321,381 ​ ​ 321,381 Other comprehensive loss ​ — ​ ​ — ​ ​ — ​ ​ ( 27,790 ) ​ ​ — ​ ​ ( 27,790 ) Issuance of ordinary shares ​ 921,730 ​ ​ 55 ​ ​ 29,510 ​ ​ — ​ ​ — ​ ​ 29,565 Income tax benefit of past share issuance cost ​ — ​ ​ — ​ ​ 2,977 ​ ​ — ​ ​ — ​ ​ 2,977 Exercises of share options ​ 156,787 ​ ​ 10 ​ ​ 1,814 ​ ​ — ​ ​ — ​ ​ 1,824 Restricted and performance share units distributed during the period ​ 346,736 ​ ​ 21 ​ ​ ( 21 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense ​ — ​ ​ — ​ ​ 18,747 ​ ​ — ​ ​ — ​ ​ 18,747 Issuance of ordinary shares relating to employee stock purchase plan ​ 4,724 ​ ​ — ​ ​ 146 ​ ​ — ​ ​ — ​ ​ 146 Balance at September 30, 2021 ​ 46,207,776 ​ $ 2,797 ​ $ 1,069,191 ​ $ ( 17,883 ) ​ $ ( 463,350 ) ​ $ 590,755 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at December 31, 2021 ​ 46,298,635 ​ $ 2,802 ​ $ 1,076,972 ​ $ ( 28,856 ) ​ $ ( 455,142 ) ​ $ 595,776 Loss for the period ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 133,596 ) ​ ​ ( 133,596 ) Other comprehensive loss ​ — ​ ​ — ​ ​ — ​ ​ ( 64,144 ) ​ ​ — ​ ​ ( 64,144 ) Exercise of share options ​ 119,884 ​ ​ 6 ​ ​ 707 ​ ​ — ​ ​ — ​ ​ 713 Restricted and performance share units distributed during the period ​ 387,285 ​ ​ 21 ​ ​ ( 21 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense ​ — ​ ​ — ​ ​ 22,290 ​ ​ — ​ ​ — ​ ​ 22,290 Issuance of ordinary shares relating to employee stock purchase plan ​ 9,305 ​ ​ 1 ​ ​ 130 ​ ​ — ​ ​ — ​ ​ 131 Balance at September 30, 2022 ​ 46,815,109 ​ $ 2,830 ​ $ 1,100,078 ​ $ ( 93,000 ) ​ $ ( 588,738 ) ​ $ 421,170 ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 5 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Nine months ended September 30, ​ ​ 2022 2021 ​ ​ (in thousands) Cash flows from operating activities ​ ​ ​ ​ ​ ​ ​ Net (loss) / income ​ $ ( 133,596 ) ​ $ 321,381 ​ Adjustments to reconcile net (loss) / income to net cash (used in) / generated from operating activities: ​ ​ ​ ​ ​ ​ ​ Depreciation and amortization expense ​ ​ 6,206 ​ ​ 5,447 ​ Share-based compensation expense ​ ​ 22,290 ​ ​ 18,747 ​ Deferred tax (income) / expense ​ ​ ( 1,263 ) ​ ​ 3,560 ​ Changes in fair value of contingent consideration and derivative financial instrument, net ​ ​ 6,875 ​ ​ 416 ​ Unrealized foreign exchange gains, net ​ ​ ( 36,390 ) ​ ​ ( 23,242 ) ​ Other non-current assets, net ​ ​ ( 581 ) ​ ​ ( 2,862 ) ​ Changes in operating assets and liabilities: ​ ​ ​ ​ ​ ​ ​ Accounts receivable and contract asset, prepaid expenses, and other current assets and receivables ​ ​ 41,992 ​ ​ ( 1,993 ) ​ Inventories ​ ​ ( 4,075 ) ​ ​ - ​ Accounts payable ​ ​ 4,529 ​ ​ 2,003 ​ Accrued expenses, other liabilities, and operating leases ​ ​ 3,633 ​ ​ 11,592 ​ Net cash (used in) / generated from operating activities ​ ​ ( 90,380 ) ​ ​ 335,049 ​ Cash flows from investing activities ​ ​ ​ ​ ​ ​ ​ Purchases of property, plant, and equipment ​ ​ ( 12,622 ) ​ ​ ( 13,885 ) ​ Acquisition of Corlieve, net of cash acquired ​ ​ ( 1,900 ) ​ ​ ( 49,949 ) ​ Net cash used in investing activities ​ ​ ( 14,522 ) ​ ​ ( 63,834 ) ​ Cash flows from financing activities ​ ​ ​ ​ ​ ​ ​ Proceeds from issuance of ordinary shares related to employee stock option and purchase plans ​ ​ 844 ​ ​ 1,971 ​ Proceeds from loan increment, net of debt issuance costs ​ ​ - ​ ​ 34,603 ​ Proceeds from issuance of ordinary shares ​ ​ - ​ ​ 30,899 ​ Share issuance costs from issuance of ordinary shares ​ ​ - ​ ​ ( 1,334 ) ​ Repayment of debt acquired through acquisition of Corlieve ​ ​ - ​ ​ ( 1,175 ) ​ Net cash generated from financing activities ​ ​ 844 ​ ​ 64,964 ​ Currency effect on cash, cash equivalents and restricted cash ​ ​ ( 11,876 ) ​ ​ ( 2,589 ) ​ Net (decrease) / increase in cash, cash equivalents and restricted cash ​ ​ ( 115,934 ) ​ ​ 333,590 ​ Cash, cash equivalents and restricted cash at beginning of period ​ ​ 559,353 ​ ​ 247,680 ​ Cash, cash equivalents and restricted cash at the end of period ​ $ 443,419 ​ $ 581,270 ​ Cash and cash equivalents ​ $ 440,313 ​ $ 578,464 ​ Restricted cash related to leasehold and other deposits ​ ​ 3,106 ​ ​ 2,806 ​ Total cash, cash equivalents and restricted cash ​ $ 443,419 ​ $ 581,270 ​ Supplemental cash flow disclosures: ​ ​ ​ ​ ​ ​ ​ Cash paid for interest ​ $ ( 6,410 ) ​ $ ( 4,503 ) ​ Non-cash (decrease) / increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment ​ $ 1,766 ​ $ 415 ​ ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ 6 Table of Contents 1 General business information ​ uniQure (the “Company”) was incorporated on January 9, 2012 as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid ) under the laws of the Netherlands. The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company’s business was founded in 1998 and was initially operated through its predecessor company, Amsterdam Molecular Therapeutics (AMT) Holding N.V (“AMT”). In 2012, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT. Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability ( naamloze vennootschap ) and changed its legal name from uniQure B.V. to uniQure N.V. ​ The Company is registered in the trade register of the Chamber of Commerce ( Kamer van Koophandel ) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000. ​ The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”. ​ ​ 2 Summary of significant accounting policies ​ 2.1 Basis of preparation ​ The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the SEC regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). ​ The unaudited consolidated financial statements are presented in U.S. dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in parenthesis, converted at the foreign exchange rate as of the transaction date. ​ 2.2 Unaudited interim financial information ​ The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented. ​ Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the three and nine months ended September 30, 2022, are not necessarily indicative of the results to be expected for the full year ending December 31, 2022, or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report . ​ 2.3 Use of estimates ​ The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. ​ ​ 7 Table of Contents 2.4 Accounting policies ​ The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2021, and the notes thereto, which are included in the Company’s Annual Report . There have been no material changes in the Company’s significant accounting policies during the nine months ended September 30, 2022, except as noted below. ​ Inventories ​ The Company started producing commercial materials in April 2022 to supply CSL Behring LLC (“CSL Behring”) with etranacogene dezaparvovec (the “Product”) in accordance with the June 2020 Development and Commercial Supply Agreement between the Company and CSL Behring. From this date onwards, the Company presents the costs associated with the aforementioned activities as cost of contract manufacturing. The Company capitalizes inventory to be sold to CSL Behring to the extent it expects to generate probable future benefits from such sales. Refer to Note 3, “CSL Behring collaboration” for further detail. ​ Per ASC 330, Inventory, inventory is stated at the lower of cost or estimated net realizable value, on a first-in, first-out basis. The Company capitalizes raw materials to the extent these can be used in the manufacturing of the Product. The Company uses standard costs, approximating average costs to determine its cost basis for work in progress and finished goods. The Company’s assessment of recoverability value requires the use of estimates regarding the net realizable value of its inventory balances, including an assessment of excess or obsolete inventory. As applicable, write-downs resulting from adjustments to net realizable value will be recorded to cost of contract manufacturing. ​ 2.5 Recent accounting pronouncements ​ There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 2022, as compared to the recent accounting pronouncements described in Note 2.3.23 of the Company’s Annual Report , which could be expected to materially impact the Company’s unaudited consolidated financial statements. ​ ​ 8 Table of Contents 3 CSL Behring collaboration ​ On June 24, 2020 (the “Signing Date”), uniQure biopharma B.V., a wholly owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring, pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, the Company’s investigational gene therapy for patients with hemophilia B. ​ The transaction became fully effective on May 6, 2021, one day after the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (the “HSR Act”) expired on May 5, 2021 (“Closing”). The Company, as of the effective date, identified two material performance obligations related to the CSL Behring Agreement: ​ (i) Sale of the exclusive global rights to the Product (“License Sale”); and (ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of the Product and to provide any such information generated to CSL Behring (“Manufacturing Development”). License Sale ​ The Company continued to develop the Product between the Signing Date and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” that are included as part of the “License Sale”). The Additional Covenants are not considered distinct from the performance obligation to sell the license to CSL Behring as CSL Behring could not benefit from the Additional Covenants on their own, or have these activities be performed with readily available resources. ​ The Company determined that the fixed upfront payment of $ 450.0 million and the $ 12.4 million that the Company received in relation to the Additional Covenants should be allocated to the License Sale. The Company determined that the License Sale was completed on May 6, 2021, when it transferred the license and CSL Behring assumed full responsibility for the development and commercialization of the Product. At Closing, the Company evaluated the amounts of potential payments and the likelihood that the payments will be received. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments are constrained as of Closing. The Company determined that it would recognize revenue related to these payments only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter. Similarly, the Company records expenses related to its existing license and other agreements as well as its financial advisor for a mid-single digit percentage of any such revenue recognized associated to meeting a milestone. The Company includes payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of the Product once these have been earned. The Company recognized nil and $ 462.4 million of license revenue in the three and nine months ended September 30, 2021 related to the License Sale. During the three and nine months ended September 30, 2022, the Company did no t recognize any license revenue. ​ Collaboration Services ​ The Company recognized $ 0.7 million and $ 2.1 million of collaboration revenue in the three and nine months ended September 30, 2022, compared to $ 0.5 and $ 0.9 million in the three and nine months ended September 30, 2021. The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring as well as additional development services that CSL Behring requested. These collaboration services are reimbursed at the pre-agreed full-time-employee rate (“FTE-rate”). ​ Contract Manufacturing ​ The Company commenced capitalizing inventory in April 2022 for the Product to be sold to CSL Behring. ​ 9 Table of Contents On September 6, 2022, CSL Behring notified the Company of its intent to transfer manufacturing technology related to the Product to a third-party contract manufacturer designated by CSL Behring. CSL Behring also requested that the Company continue to serve as a manufacturer of the Product after the Company completes the technology transfer to a third party. The Company and CSL Behring are in the process of negotiating the terms of the transfer of manufacturing responsibility pursuant to the CSL Behring Agreement. ​ Accounts Receivable and Contract Asset ​ As of December 31, 2021, the Company recorded accounts receivable of $ 2.9 million from CSL Behring related to collaboration services as well as a contract asset of $ 55.0 million associated with milestone payments due upon CSL Behring’s global regulatory submissions for etranacogene dezaparvovec, which were deemed to be probable. In March and April 2022, CSL Behring submitted marketing applications in the United States and European Union, and as of March and April 2022, the Company had collected the $ 55.0 million owed. As of September 30, 2022, the Company had accounts receivable of $ 3.0 million from CSL Behring. ​ ​ ​ ​ 4 Inventories ​ The Company commenced capitalizing inventory in April 2022 when the Company started producing commercial materials to supply the Product to CSL Behring. The following table summarizes the inventory balances for the nine months ended September 30, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ September 30, ​ December 31, ​ 2022 2021 ​ ​ (in thousands) Raw materials ​ $ 1,447 ​ $ — Work in progress ​ ​ 2,178 ​ ​ — Finished goods ​ ​ 450 ​ ​ — Inventories ​ $ 4,075 ​ $ — ​ ​ ​ ​ ​ 10 Table of Contents 5 Fair value measurement ​ The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. U.S. GAAP requires disclosure of methodologies used in determining the reported fair values and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below: ​ Level 1 – Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date. ​ Level 2 – Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly. ​ Level 3 – Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable. ​ To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. ​ The carrying amount of cash and cash equivalents, accounts receivable, prepaid expenses, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Consolidated balance sheets approximate their fair values due to their short-term maturities. ​ The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of September 30, 2022, and December 31, 2021: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Quoted prices in active markets (Level 1) Significant other observable inputs (Level 2) Significant unobservable inputs (Level 3) Total Classification in Consolidated balance sheets ​ ​ ​ (in thousands) ​ ​ At December 31, 2021 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 556,256 ​ $ — ​ $ — ​ $ 556,256 ​ Cash and cash equivalents Restricted cash ​ ​ 3,097 ​ ​ — ​ ​ — ​ ​ 3,097 ​ Other non-current assets Total assets ​ $ 559,353 ​ $ — ​ $ — ​ $ 559,353 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 29,542 ​ ​ 29,542 ​ Contingent consideration Derivative financial instrument ​ ​ — ​ ​ — ​ ​ 2,805 ​ ​ 2,805 ​ Other non-current liabilities Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 846 ​ ​ 846 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 33,193 ​ $ 33,193 ​ ​ At September 30, 2022 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 440,313 ​ $ — ​ $ — ​ $ 440,313 ​ Cash and cash equivalents Restricted cash ​ ​ 3,106 ​ ​ — ​ ​ — ​ ​ 3,106 ​ Other non-current assets Total assets ​ $ 443,419 ​ $ — ​ $ — ​ $ 443,419 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 32,695 ​ ​ 32,695 ​ Current portion of contingent consideration; contingent consideration, net of current portion Derivative financial instrument ​ ​ — ​ ​ — ​ ​ 2,171 ​ ​ 2,171 ​ Other non-current liabilities Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 312 ​ ​ 312 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 35,178 ​ $ 35,178 ​ ​ ​ ​ ​ ​ ​ ​ ​ 11 Table of Contents Contingent consideration ​ The Company is required to pay up to EUR 178.8 million ($ 175.2 million) at September 30, 2022 to the former shareholders of Corlieve upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve. The fair market value of the contingent consideration was determined using unobservable initial inputs with respect to (i) the probability of achieving the relevant milestones, or POS, (ii) the estimated timing of achieving such milestones, and (iii) the interest rate used to discount the payments. The Company determined the fair market value of the contingent consideration by calculating the probability-adjusted payments based on each milestone’s probability of achievement. The probability-adjusted payments were then discounted to present value using a discount rate representing the Company’s credit risk. The discount rate was determined using the effective interest rate of the Company’s existing debt facility adjusted for difference in maturity dates based on market data on effective yields for US bonds with a CCC credit rating. ​ The fair value of the contingent consideration as of September 30, 2022 was $ 32.7 million (December 31, 2021: $ 29.5 million) using discount rates of approximately 11.9 % to 13.3 % (December 31, 2021: 10.9 % to 11.9 %) as well as a 66.0 % (December 31, 2021: 55.0 %) likelihood of Corlieve’s target candidate for treatment of temporal lobe epilepsy (“AMT-260”) advancing into clinical development by no later than late 2023. If as of September 30, 2022 the Company had assumed a 100 % likelihood of AMT-260 advancing into clinical development, then the fair value of the contingent consideration would have increased to $ 45.4 million. If as of September 30, 2022 the Company assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would be released to income. Changes in fair value of the contingent consideration are recognized within research and development expenses in the consolidated statements of operations. ​ The following table presents the changes in fair value of contingent consideration between December 31, 2021 and September 30, 2022: ​ ​ ​ ​ ​ ​ Amount of ​ contingent ​ consideration ​ ​ 2022 ​ (in thousands) Balance at December 31, 2021 $ 29,542 Change in fair value (presented within research and development expenses) ​ 7,510 Currency translation effects ​ ( 4,357 ) Balance at September 30, 2022 $ 32,695 ​ As of September 30, 2022, the Company classified $ 23.5 million of the total contingent consideration of $ 32.7 million as current liabilities. The balance sheet classification between current and non-current liabilities is based upon the Company’s best estimate of the timing of settlement of the remaining relevant milestones. ​ Derivative financial instrument ​ The Company issued a derivative financial instrument related to its collaboration with BMS. ​ On December 1, 2020, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four collaboration targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $ 70.0 million, provided that (x) if $ 70.0 million is greater than five percent ( 5.0 %) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $ 70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”). The Company has not consummated any change of control transaction as of September 30, 2022 that would obligate it to make a CoC-payment. ​ ​ ​ ​ ​ 12 Table of Contents The Company determined that the CoC-payment should be recorded as a derivative financial liability as of December 1, 2020 and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss. The fair market value of the derivative financial liability is materially impacted by probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment. This probability represents an unobservable input. The Company determines the fair market value of the derivative financial liability by using a present value model based on expected cash flow. The expected cash flows are materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction within the biotechnology industry. The Company estimated this unobservable input using the best information available as of September 30, 2022 and December 31, 2021. The Company obtained reasonably available market information that it believed market participants would use in determining the likelihood of the occurrence of a change-of control transaction within the biotechnology industry. Selecting and evaluating market information involves considerable judgement and uncertainty. Based on all such information and its judgment, the Company estimated that the fair market value of the derivative financial liability (presented within “Other non-current liabilities”) as of September 30, 2022 was $ 2.2 million and as of December 31, 2021 was $ 2.8 million. The change in the fair market value of the derivative financial liability was nil for the three months ended September 30, 2022 and was $ 0.6 million for the nine months ended September 30, 2022 related to a decrease in the fair market value ( nil in the same periods in 2021). The decrease in fair market value for the nine months ended September 30, 2022 was recorded as a gain within Other non-operating (losses) / gains . ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 6 Accrued expenses and other current liabilities ​ Accrued expenses and other current liabilities include the following items: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ September 30, ​ December 31, ​ 2022 2021 ​ ​ (in thousands) Accruals for goods received from and services provided by vendors-not yet billed ​ $ 13,497 ​ $ 13,012 Personnel related accruals and liabilities ​ ​ 12,940 ​ ​ 12,603 Accrued contract fulfillment costs and costs to obtain a contract ​ ​ — ​ ​ 2,872 Total ​ $ 26,437 ​ $ 28,487 ​ ​ ​ ​ 13 Table of Contents 7 Long-term debt ​ On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc. (formerly known as Hercules Technology Growth Capital, Inc.) (“Hercules”). The facility was amended and restated in 2014, 2016, 2018 (“2018 Amended Facility”), January 2021 (“2021 Amended Facility”) and in December 2021 (“2021 Restated Facility”). On January 29, 2021, the Company drew down $ 35.0 million as part of the amendment of the facility at that time. ​ Pursuant to the 2021 Restated Facility, Tranche A and Tranche B of the 2021 Amended Facility with a total outstanding balance of $ 70.0 million were consolidated into one tranche with a total commitment of $ 100.0 million. The Company drew down an additional $ 30.0 million at the time of the December 2021 amendment, resulting in total principal outstanding as of September 30, 2022 of $ 100.0 million. The 2021 Restated Facility extended the loan’s maturity date from June 1, 2023 until December 1, 2025. The interest-only period was extended from January 1, 2023 to December 1, 2024, or December 1, 2025 if, prior to June 30, 2024, either (a) the Biologics License Application (“BLA”) for AMT-061 is approved by the U.S. Food and Drug Administration (“FDA”) or (b) AMT-130 is advanced into a pivotal trial. The interest rate is adjustable and is the greater of (i) 7.95 % and (ii) 7.95 % plus the prime rate less 3.25 % per annum. Under the 2021 Restated Facility, the Company owes a back-end fee of 4.85 % of the outstanding debt. The Company is required to repay the facility in equal monthly installments of principal and interest between the end of the interest-only period and the maturity date. The Company continues to owe a $ 2.5 million back-end fee related to the 2021 Amended Facility which is due on June 1, 2023. The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2021 Restated Facility was $ 103.3 million as of September 30, 2022, compared to $ 101.6 million as of December 31, 2021, and is recorded net of discount and debt issuance costs. The foreign currency loss on the facility in the three and nine months ended September 30, 2022 was $ 6.6 million and $ 15.0 million, respectively compared to a foreign currency loss of $ 1.8 million and $ 4.0 million during the same periods in 2021 for the 2018 Amended Facility and the 2021 Amended Facility. Interest expense associated with the 2021 Restated Facility during the three and nine months ended September 30, 2022 was $ 3.0 million and $ 8.1 million, respectively compared to $ 1.9 million and $ 5.2 million, respectively, during the same periods in 2021 for the 2018 Amended Facility and the 2021 Amended Facility. As a covenant in the 2021 Restated Facility the Company has periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the United States equivalent to the lesser of (i) 65 % of the outstanding balance of principal due or (ii) 100 % of worldwide cash and cash equivalents. This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company. The Company, beginning on April 1, 2023, is also required to keep a minimum of unrestricted cash of at least 50 % of the loan amount outstanding. If, prior to June 30, 2024, either (a) the BLA for AMT-061 is approved by the FDA or (b) AMT-130 is advanced into a pivotal trial, the minimum cash covenant will be lowered to at least 30 % of the loan amount outstanding and its effectiveness will be deferred to April 1, 2024. In combination with other covenants, the 2021 Restated Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers, and directors, and to make dividend payments and other distributions to its shareholders. The Company secured the facilities by directly or indirectly pledging its total assets of $ 638.2 million, less $ 75.3 million of cash and cash equivalents and other current assets held by uniQure N.V. and $ 80.2 million of other current assets and investment held by Corlieve Therapeutics SAS. The 2021 Restated Facility contains provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of September 30, 2022, the Company was in material compliance with all covenants and provisions. ​ ​ ​ ​ ​ ​ ​ 14 Table of Contents 8 Share-based compensation ​ The Company’s share-based compensation plans include the 2014 Amended and Restated Share Option Plan (the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”). At the annual general meeting of shareholders in June 2021, the Company’s shareholders approved an increase in the number of ordinary shares authorized for issuance under the 2014 Plan from 8,601,471 to 12,601,471 . ​ In June 2018, the Company’s shareholders adopted and approved an employee share purchase plan (“ESPP”) allowing the Company to issue up to 150,000 ordinary shares. The ESPP is intended to qualify under Section 423 of the Internal Revenue Code of 1986. Under the ESPP, employees are eligible to purchase ordinary shares through payroll deductions, subject to any plan limitations. The purchase price of the ordinary shares on each purchase date is equal to 85 % of the lower of the closing market price on the offering date and the closing market price on the purchase date of each three-month offering period. ​ 2014 Plans and ESPP ​ Share-based compensation expense recognized by classification included in the Consolidated statements of operations and comprehensive loss in relation to the 2014 Plans and the ESPP for the periods indicated below was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended September 30, ​ Nine months ended September 30, ​ 2022 2021 ​ 2022 ​ 2021 ​ ​ (in thousands) ​ (in thousands) Research and development ​ $ 3,730 ​ $ 3,249 ​ $ 12,090 ​ $ 9,222 Selling, general and administrative ​ ​ 3,758 ​ ​ 2,707 ​ ​ 9,932 ​ ​ 9,525 Cost of contract manufacturing ​ ​ 120 ​ ​ — ​ ​ 268 ​ ​ — Total ​ $ 7,608 ​ $ 5,956 ​ $ 22,290 ​ $ 18,747 ​ Share-based compensation expense recognized by award type of the 2014 Plans as well as the ESPP was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended September 30, ​ Nine months ended September 30, ​ 2022 2021 ​ 2022 ​ 2021 ​ ​ (in thousands) ​ (in thousands) Award type/ESPP ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Share options ​ $ 3,448 ​ $ 2,965 ​ $ 10,141 ​ $ 9,155 Restricted share units ​ ​ 3,957 ​ ​ 2,582 ​ ​ 11,354 ​ ​ 8,364 Performance share units ​ ​ 197 ​ ​ 403 ​ ​ 777 ​ ​ 1,206 ESPP ​ ​ 6 ​ ​ 6 ​ ​ 18 ​ ​ 22 Total ​ $ 7,608 ​ $ 5,956 ​ $ 22,290 ​ $ 18,747 ​ As of September 30, 2022, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were: ​ ​ ​ ​ ​ ​ ​ ​ Unrecognized Weighted average ​ share-based remaining ​ ​ compensation ​ period for ​ ​ expense ​ recognition ​ ​ (in thousands) ​ (in years) Award type ​ ​ ​ ​ ​ Share options ​ $ 27,720 ​ 2.64 Restricted share units ​ ​ 28,629 ​ 2.04 Total ​ $ 56,349 ​ 2.34 ​ The Company satisfies the exercise of share options and vesting of Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) through newly issued ordinary shares. ​ 15 Table of Contents Share options Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years . The first 25 % vests after one year from the initial grant date and the remainder vests in equal quarterly installments over years two, three and four. Certain grants to non-executive directors vest in full after one year . Any options that vest must be exercised by the tenth anniversary of the initial grant date. The following tables summarize option activity under the Company’s 2014 Plans for the nine months ended September 30, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ Options ​ ​ Number of ​ Weighted average ​ ordinary shares exercise price ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2021 ​ 3,308,325 ​ $ 31.02 Granted ​ 1,399,466 ​ $ 15.75 Forfeited ​ ( 183,870 ) ​ $ 38.74 Expired ​ ( 133,381 ) ​ $ 35.79 Exercised ​ ( 115,884 ) ​ $ 6.03 Outstanding at September 30, 2022 ​ 4,274,656 ​ $ 26.22 Thereof, fully vested and exercisable on September 30, 2022 ​ 2,068,810 ​ $ 28.33 Thereof, outstanding and expected to vest after September 30, 2022 ​ 2,205,846 ​ $ 24.24 ​ ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of options issued during the period (in $ millions) ​ ​ ​ $ 12.5 Proceeds from option sales during the period (in $ millions) ​ ​ ​ $ 0.7 ​ The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended September 30, Nine months ended September 30, Assumptions 2022 2021 2022 2021 Expected volatility ​ 70 % ​ 75 % ​ 70 % ​ 75 % Expected terms ​ 10 years ​ 10 years ​ 10 years ​ 10 years Risk free interest rate ​ 4.16 % ​ 1.47 % ​ 2.12 % - 4.16 % ​ 1.21 - 1.85 % Expected dividend yield ​ 0 % ​ 0 % ​ 0 % ​ 0 % ​ Restricted share units (“RSUs”) ​ The following table summarizes the RSUs activity for the nine months ended September 30, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ RSU ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2021 ​ 710,617 ​ $ 38.89 Granted ​ 1,562,133 ​ $ 15.91 Vested ​ ( 283,349 ) ​ $ 39.47 Forfeited ​ ( 176,110 ) ​ $ 23.76 Non-vested at September 30, 2022 ​ 1,813,291 ​ $ 20.47 ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of RSUs granted during the period (in $ millions) ​ ​ ​ $ 24.8 ​ RSUs vest over one to three years . RSUs granted to non-executive directors vest one year from the date of grant. ​ 16 Table of Contents Performance share units (“PSUs”) ​ The following table summarizes the PSUs activity for the nine months ended September 30, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ PSU ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2021 ​ 632,930 ​ $ 33.54 Granted ​ 34,700 ​ $ 15.11 Vested ​ ( 103,970 ) ​ $ 52.14 Forfeited ​ ( 48,800 ) ​ $ 29.35 Non-vested at September 30, 2022 ​ 514,860 ​ $ 28.94 ​ ​ ​ ​ ​ ​ ​ PSUs granted in 2019 vested on the third anniversary of the grant, subject to the grantee’s continued employment. ​ The Company granted shares to certain employees in September and December 2021 and at various dates during the nine months ended September 30, 2022 that will be earned upon achievement of defined milestones. Earned shares will vest upon the later of a minimum service period of one year or three years , or the achievement of defined milestones, subject to the grantee’s continued employment. In addition, portions of the December 2021 shares granted to executives and other members of senior management are subject to achieving a minimum total shareholder return relative to the Nasdaq biotechnology index. The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable. ​ ESPP ​ During the nine months ended September 30, 2022, 9,305 ordinary shares were issued under the ESPP compared to 4,724 during the same period in 2021. As of September 30, 2022, a total of 117,997 ordinary shares remain available for issuance under the ESPP plan compared to a total of 127,302 as of September 30, 2021. ​ ​ ​ ​ 9 Income taxes ​ The Company recorded $ 0.3 and $ 1.3 million deferred tax benefit in relation to its operations in the United States and France during the three and nine months ended September 30, 2022, respectively. The Company recorded $ 0.1 million and $ 3.6 million in deferred income tax expense in the prior year in the United States and the Netherlands for the three and nine months ended September 30, 2021, respectively, of which nil and $ 3.0 million in the three and nine months periods related to the Netherlands, respectively. ​ The effective income tax rate of - 0.7 % and - 0.9 % during the three and nine months ended September 30, 2022 is substantially lower than the enacted rate of 25.8 % in the Netherlands as the Company records a valuation allowance against its net deferred tax assets in the Netherlands. The effective income tax rate during the three and nine months ended September 30, 2021 was 0.2 % and - 1.1 %, respectively, as the Company had recorded a valuation allowance against all its net deferred tax assets in the Netherlands. ​ ​ ​ 17 Table of Contents 10 Basic and diluted earnings per share ​ Basic net (loss) / income per ordinary share is computed by dividing net (loss) / income for the period by the weighted average number of ordinary shares outstanding during the period. Diluted earnings per ordinary share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. For the three and nine months ended September 30, 2021, dilutive net income per ordinary share is computed using the treasury method. As the Company has incurred a loss in the three and nine months ended September 30, 2022, all potentially dilutive ordinary shares for these periods would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share for the three and nine months ended September 30, 2022. ​ The potentially dilutive ordinary shares are summarized below: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended September 30, ​ Nine months ended September 30, ​ 2022 2021 ​ 2022 ​ 2021 ​ ​ (in thousands, except share amounts) ​ (in thousands, except share amounts) Numerator: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net (loss) / income attributable to ordinary shares ​ $ ( 47,857 ) ​ $ ( 36,531 ) ​ $ ( 133,596 ) ​ $ 321,381 ​ ​ ​ ( 47,857 ) ​ ​ ( 36,531 ) ​ ​ ( 133,596 ) ​ ​ 321,381 Denominator: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-average number of ordinary shares outstanding - basic ​ ​ 46,772,430 ​ ​ 46,152,404 ​ ​ 46,680,667 ​ ​ 45,888,769 Stock options under 2014 Plans and previous plan ​ ​ — ​ ​ — ​ ​ — ​ ​ 784,942 Non-vested RSUs and PSUs ​ ​ — ​ ​ — ​ ​ — ​ ​ 105,870 Employee share purchase plan ​ ​ — ​ ​ — ​ ​ — ​ ​ 1,382 Weighted-average number of ordinary shares outstanding - diluted ​ ​ 46,772,430 ​ ​ 46,152,404 ​ ​ 46,680,667 ​ ​ 46,780,963 ​ The following table presents ordinary share equivalents that were excluded from the calculation of diluted net (loss) / income per ordinary share for the three and nine months ended September 30, 2022 and 2021 as the effect of their inclusion would have been anti-dilutive: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended September 30, ​ Nine months ended September 30, ​ 2022 2021 ​ 2022 ​ 2021 Anti-dilutive ordinary share equivalents ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Stock options under 2014 Plans and previous plan ​ ​ 4,284,656 ​ ​ 3,357,412 ​ ​ 4,284,656 ​ ​ 2,572,470 Non-vested RSUs and PSUs ​ ​ 2,328,151 ​ ​ 841,480 ​ ​ 2,328,151 ​ ​ 735,610 Employee share purchase plan ​ ​ 721 ​ ​ 1,219 ​ ​ 721 ​ ​ 1,219 Total anti-dilutive ordinary share equivalents ​ ​ 6,613,528 ​ ​ 4,200,111 ​ ​ 6,613,528 ​ ​ 3,309,299 ​ ​ The anti-dilutive ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that exceeded the price of the Company’s ordinary shares as of the three and nine months ended September 30, 2022. ​ ​ 11 Subsequent events ​ None. ​ ​ 18 Table of Contents