uniQure N.V. 10-Q

Item 1. Financial Statements uniQure N.V. UNAUDITED CONSOLIDATED BALANCE SHEETS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2023 2022 ​ ​ (in thousands, except share and per share amounts) Current assets ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 153,851 ​ $ 228,012 Current investment securities ​ ​ 161,487 ​ ​ 124,831 Accounts receivable and contract asset ​ ​ 104,793 ​ ​ 102,376 Inventories ​ ​ 7,477 ​ ​ 6,924 Prepaid expenses ​ ​ 12,567 ​ ​ 11,817 Other current assets and receivables ​ ​ 2,468 ​ ​ 2,814 Total current assets ​ ​ 442,643 ​ ​ 476,774 Non-current assets ​ ​ ​ ​ ​ ​ Property, plant and equipment, net of accumulated depreciation of $ 46.9 million as of March 31, 2023 and $ 44.1 million as of December 31, 2022 ​ ​ 50,072 ​ ​ 50,532 Non-current investment securities ​ ​ — ​ ​ 39,984 Operating lease right-of-use assets ​ ​ 32,135 ​ ​ 32,726 Intangible assets, net, including an in-process research and development asset of $ 58.3 million as of March 31, 2023 and $ 57.3 million as of December 31, 2022 ​ ​ 59,704 ​ ​ 58,778 Goodwill ​ ​ 25,998 ​ ​ 25,581 Deferred tax assets, net ​ ​ 14,331 ​ ​ 14,528 Other non-current assets ​ ​ 6,121 ​ ​ 6,061 Total non-current assets ​ ​ 188,361 ​ ​ 228,190 Total assets ​ $ 631,004 ​ $ 704,964 Current liabilities ​ ​ ​ ​ ​ ​ Accounts payable ​ $ 8,885 ​ $ 10,984 Accrued expenses and other current liabilities ​ ​ 22,529 ​ ​ 30,571 Current portion of contingent consideration ​ ​ 27,253 ​ ​ 25,982 Current portion of operating lease liabilities ​ ​ 7,669 ​ ​ 8,382 Total current liabilities ​ ​ 66,336 ​ ​ 75,919 Non-current liabilities ​ ​ ​ ​ ​ ​ Long-term debt ​ ​ 103,253 ​ ​ 102,791 Operating lease liabilities, net of current portion ​ ​ 31,075 ​ ​ 31,719 Contingent consideration, net of current portion ​ ​ 9,641 ​ ​ 9,334 Deferred tax liability, net ​ ​ 6,970 ​ ​ 8,257 Other non-current liabilities ​ ​ 958 ​ ​ 935 Total non-current liabilities ​ ​ 151,897 ​ ​ 153,036 Total liabilities ​ ​ 218,233 ​ ​ 228,955 Commitments and contingencies ​ ​ ​ ​ ​ ​ Shareholders' equity ​ ​ ​ ​ ​ ​ Ordinary shares, € 0.05 par value: 80,000,000 shares authorized as of March 31, 2023 and December 31, 2022 and 47,546,673 and 46,968,032 ordinary shares issued and outstanding as of March 31, 2023 and December 31, 2022, respectively ​ ​ 2,869 ​ ​ 2,838 Additional paid-in-capital ​ ​ 1,121,554 ​ ​ 1,113,393 Accumulated other comprehensive loss ​ ​ ( 52,494 ) ​ ​ ( 58,291 ) Accumulated deficit ​ ​ ( 659,158 ) ​ ​ ( 581,931 ) Total shareholders' equity ​ ​ 412,771 ​ ​ 476,009 Total liabilities and shareholders' equity ​ $ 631,004 ​ $ 704,964 ​ 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 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ 2023 2022 ​ ​ (in thousands, except share and per share amounts) Contract manufacturing revenues ​ $ 4,937 ​ $ — Collaboration revenues ​ ​ 388 ​ ​ 1,792 Total revenues ​ ​ 5,325 ​ ​ 1,792 Operating expenses: ​ ​ ​ ​ ​ ​ Cost of contract manufacturing revenues ​ ​ ( 2,435 ) ​ ​ — Research and development expenses ​ ​ ( 60,809 ) ​ ​ ( 45,003 ) Selling, general and administrative expenses ​ ​ ( 17,848 ) ​ ​ ( 10,987 ) Total operating expenses ​ ​ ( 81,092 ) ​ ​ ( 55,990 ) Other income ​ ​ 1,811 ​ ​ 311 Other expense ​ ​ ( 216 ) ​ ​ ( 193 ) Loss from operations ​ ​ ( 74,172 ) ​ ​ ( 54,080 ) Interest income ​ ​ 1,669 ​ ​ 42 Interest expense ​ ​ ( 3,562 ) ​ ​ ( 2,515 ) Foreign currency (losses) / gains, net ​ ​ ( 2,369 ) ​ ​ 8,567 Other non-operating gains, net ​ ​ — ​ ​ 692 Loss before income tax benefit ​ $ ( 78,434 ) ​ $ ( 47,294 ) Income tax benefit ​ ​ 1,207 ​ ​ 616 Net loss ​ $ ( 77,227 ) ​ $ ( 46,678 ) Other comprehensive loss: ​ ​ ​ ​ ​ ​ Foreign currency translation adjustments ​ ​ 5,797 ​ ​ ( 10,450 ) Total comprehensive loss ​ $ ( 71,430 ) ​ $ ( 57,128 ) ​ ​ ​ ​ ​ ​ ​ Basic and diluted net loss per ordinary share ​ ​ ( 1.63 ) ​ ​ ( 1.00 ) Weighted average shares used in computing basic and diluted net loss per ordinary share ​ ​ 47,436,335 ​ ​ 46,599,114 ​ 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 MONTHS ENDED MARCH 31, 2023 AND 2022 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ other ​ ​ ​ ​ Total ​ ​ Ordinary shares ​ paid-in ​ comprehensive ​ Accumulated ​ shareholders’ ​ ​ No. of shares Amount capital (loss) / income deficit equity ​ ​ (in thousands, except share and per share amounts) Balance at December 31, 2021 ​ 46,298,635 ​ $ 2,802 ​ $ 1,076,972 ​ $ ( 28,856 ) ​ $ ( 455,142 ) ​ $ 595,776 Loss for the period ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 46,678 ) ​ ​ ( 46,678 ) Other comprehensive loss ​ — ​ ​ — ​ ​ — ​ ​ ( 10,450 ) ​ ​ — ​ ​ ( 10,450 ) Exercises of share options ​ 68,124 ​ ​ 4 ​ ​ 420 ​ ​ — ​ ​ — ​ ​ 424 Restricted and performance share units distributed during the period ​ 271,131 ​ ​ 15 ​ ​ ( 15 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense ​ — ​ ​ — ​ ​ 6,868 ​ ​ — ​ ​ — ​ ​ 6,868 Issuance of ordinary shares relating to employee stock purchase plan ​ 3,558 ​ ​ — ​ ​ 61 ​ ​ — ​ ​ — ​ ​ 61 Balance at March 31, 2022 ​ 46,641,448 ​ $ 2,821 ​ $ 1,084,306 ​ $ ( 39,306 ) ​ $ ( 501,820 ) ​ $ 546,001 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Balance at December 31, 2022 ​ 46,968,032 ​ $ 2,838 ​ $ 1,113,393 ​ $ ( 58,291 ) ​ $ ( 581,931 ) ​ $ 476,009 Loss for the period ​ — ​ ​ — ​ ​ — ​ ​ — ​ ​ ( 77,227 ) ​ ​ ( 77,227 ) Other comprehensive gain ​ — ​ ​ — ​ ​ — ​ ​ 5,797 ​ ​ — ​ ​ 5,797 Exercises of share options ​ 10,055 ​ ​ 1 ​ ​ 86 ​ ​ — ​ ​ — ​ ​ 87 Restricted and performance share units distributed during the period ​ 566,091 ​ ​ 30 ​ ​ ( 30 ) ​ ​ — ​ ​ — ​ ​ — Share-based compensation expense ​ — ​ ​ — ​ ​ 8,061 ​ ​ — ​ ​ — ​ ​ 8,061 Issuance of ordinary shares relating to employee stock purchase plan ​ 2,495 ​ ​ 0 ​ ​ 44 ​ ​ ​ ​ ​ ​ ​ ​ 44 Balance at March 31, 2023 ​ 47,546,673 ​ $ 2,869 ​ $ 1,121,554 ​ $ ( 52,494 ) ​ $ ( 659,158 ) ​ $ 412,771 ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ 4 Table of Contents uniQure N.V. ​ UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ 2023 2022 ​ ​ (in thousands) Cash flows from operating activities ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ ( 77,227 ) ​ $ ( 46,678 ) ​ Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ ​ ​ ​ Depreciation and amortization expense ​ ​ 2,527 ​ ​ 2,122 ​ Share-based compensation expense ​ ​ 8,061 ​ ​ 6,868 ​ Deferred tax income ​ ​ ( 1,207 ) ​ ​ ( 616 ) ​ Changes in fair value of contingent consideration and derivative financial instrument, net ​ ​ 975 ​ ​ 1,512 ​ Unrealized foreign exchange losses / (gains), net ​ ​ 1,193 ​ ​ ( 7,356 ) ​ Other items, net ​ ​ ( 469 ) ​ ​ - ​ Changes in operating assets and liabilities: ​ ​ ​ ​ ​ ​ ​ Accounts receivable and contract asset, prepaid expenses, and other current assets and receivables ​ ​ ( 936 ) ​ ​ 17,209 ​ Inventories ​ ​ ( 553 ) ​ ​ - ​ Accounts payable ​ ​ ( 1,661 ) ​ ​ 7,306 ​ Accrued expenses, other liabilities, and operating leases ​ ​ ( 9,005 ) ​ ​ ( 6,078 ) ​ Net cash used in operating activities ​ ​ ( 78,302 ) ​ ​ ( 25,711 ) ​ Cash flows from investing activities ​ ​ ​ ​ ​ ​ ​ Purchases of property, plant, and equipment ​ ​ ( 2,342 ) ​ ​ ( 4,058 ) ​ Proceeds on maturity of investment securities ​ ​ 5,330 ​ ​ - ​ Acquisition of Corlieve, net of cash acquired ​ ​ - ​ ​ ( 822 ) ​ Net cash generated from / (used in) investing activities ​ ​ 2,988 ​ ​ ( 4,880 ) ​ Cash flows from financing activities ​ ​ ​ ​ ​ ​ ​ Proceeds from issuance of ordinary shares related to employee stock option and purchase plans ​ ​ 131 ​ ​ 485 ​ Net cash generated from financing activities ​ ​ 131 ​ ​ 485 ​ Currency effect on cash, cash equivalents and restricted cash ​ ​ 1,034 ​ ​ ( 1,168 ) ​ Net decrease in cash, cash equivalents and restricted cash ​ ​ ( 74,149 ) ​ ​ ( 31,274 ) ​ Cash, cash equivalents and restricted cash at beginning of period ​ ​ 231,173 ​ ​ 559,353 ​ Cash, cash equivalents and restricted cash at the end of period ​ $ 157,024 ​ $ 528,079 ​ Cash and cash equivalents ​ $ 153,851 ​ $ 524,886 ​ Restricted cash related to leasehold and other deposits ​ ​ 3,173 ​ ​ 3,193 ​ Total cash, cash equivalents and restricted cash ​ $ 157,024 ​ $ 528,079 ​ Supplemental cash flow disclosures: ​ ​ ​ ​ ​ ​ ​ Cash paid for interest ​ $ ( 3,057 ) ​ $ ( 1,906 ) ​ Non-cash (decrease) / increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment ​ $ ( 753 ) ​ $ ( 611 ) ​ ​ The accompanying notes are an integral part of these unaudited consolidated financial statements. ​ ​ 5 Table of Contents 1 General business information ​ uniQure N.V. (the “Company”) was incorporated on January 9, 2012, initially 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 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 on the Nasdaq Global Select Market, 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 25a, 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 United States Securities and Exchange Commission (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 months ended March 31, 2023, are not necessarily indicative of the results to be expected for the full year ending December 31, 2023, 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 on Form 10-K filed by the Company with the SEC on February 27, 2023 (the “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. ​ 6 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, 2022, and the notes thereto, which are included in the Annual Report . There have been no material changes in the Company’s significant accounting policies during the three months ended March 31, 2023. ​ 2.5 Recent accounting pronouncements ​ There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2023, as compared to the recent accounting pronouncements described in Note 2.3.25 of the Annual Report , which could be expected to materially impact the Company’s unaudited consolidated financial statements. ​ 3 CSL Behring collaboration ​ On June 24, 2020 (the “Signing Date”), uniQure biopharma B.V., a wholly owned subsidiary of the Company entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”), pursuant to which CSL Behring received exclusive global rights to HEMGENIX® (the “Product”). ​ The transaction became fully effective on May 6, 2021 (“Closing”). Accounts Receivable and Contract Asset ​ As of December 31, 2022, the Company recorded accounts receivable of $ 2.2 million from CSL Behring related to collaboration services as well as a contract asset of $ 100.0 million associated to a milestone due from CSL Behring following the first sale of HEMGENIX® in the U.S. , which was deemed to be probable. ​ As of March 31, 2023, the Company had accounts receivable of $ 4.8 million from CSL Behring related to collaboration services and contract manufacturing revenue and as well as the contract asset of $ 100.0 million. ​ 4 Investment securities The following tables summarize the Company’s investments in sovereign debt as of March 31, 2023 and December 31, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ At March 31, 2023 ​ ​ Amortized cost, as adjusted ​ Gross unrealized holding gains ​ Gross unrealized holding losses ​ Estimated fair value ​ ​ (in thousands) Current investments: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Government debt securities (held-to-maturity) ​ $ 161,487 ​ $ — ​ $ ( 277 ) ​ $ 161,210 Total ​ $ 161,487 ​ $ — ​ $ ( 277 ) ​ $ 161,210 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ At December 31, 2022 ​ ​ Amortized cost, as adjusted ​ Gross unrealized holding gains ​ Gross unrealized holding losses ​ Estimated fair value ​ ​ (in thousands) Current investments: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Government debt securities (held-to-maturity) ​ $ 124,831 ​ $ — ​ $ ( 283 ) ​ $ 124,548 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Non-current investments: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Government debt securities (held-to-maturity) ​ ​ 39,984 ​ ​ — ​ ​ ( 43 ) ​ ​ 39,941 Total ​ $ 164,815 ​ $ — ​ $ ( 326 ) ​ $ 164,489 ​ Inputs to the fair value of the investments are considered Level 2 inputs. ​ 7 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. ASC 820, Fair Value Measurements and Disclosures 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 from licensing and collaboration partners, 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 March 31, 2023, and December 31, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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, 2022 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 228,012 ​ $ — ​ $ — ​ $ 228,012 ​ Cash and cash equivalents Restricted cash ​ ​ 3,161 ​ ​ — ​ ​ — ​ ​ 3,161 ​ Other non-current assets Total assets ​ $ 231,173 ​ $ — ​ $ — ​ $ 231,173 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 35,316 ​ ​ 35,316 ​ Contingent consideration Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 297 ​ ​ 297 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 35,613 ​ $ 35,613 ​ ​ At March 31, 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 153,851 ​ $ — ​ $ — ​ $ 153,851 ​ Cash and cash equivalents Restricted cash ​ ​ 3,173 ​ ​ — ​ ​ — ​ ​ 3,173 ​ Other non-current assets Total assets ​ $ 157,024 ​ $ — ​ $ — ​ $ 157,024 ​ ​ Liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Contingent consideration ​ ​ — ​ ​ — ​ ​ 36,894 ​ ​ 36,894 ​ Contingent consideration Consideration for post-acquisition services ​ ​ — ​ ​ — ​ ​ 328 ​ ​ 328 ​ Other non-current liabilities Total liabilities ​ $ — ​ $ — ​ $ 37,222 ​ $ 37,222 ​ ​ ​ Contingent consideration ​ The Company is required to pay up to EUR 178.8 million ($ 194.5 million at March 31, 2023) at March 31, 2023 to the former shareholders of Corlieve Therapeutics SAS (“Corlieve”) upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve. ​ The fair value of the contingent consideration as of March 31, 2023 was $ 36.9 million (December 31, 2022: $ 35.3 million) using discount rates of approximately 14.3 % to 15.0 % (December 31, 2022: 14.0 % to 14.4 %) as well as a 66.0 % (December 31, 2022: 66.0 %) 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 March 31, 2023 the 8 Table of Contents 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 $ 51.0 million. If as of March 31, 2023 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, 2022 and March 31, 2023: ​ ​ ​ ​ ​ ​ Amount of ​ contingent ​ consideration ​ ​ 2022 ​ (in thousands) Balance at December 31, 2022 $ 35,316 Change in fair value (presented within research and development expenses) ​ 975 Currency translation effects ​ 603 Balance at March 31, 2023 $ 36,894 ​ As of March 31, 2023, the Company classified $ 27.3 million of the total contingent consideration of $ 36.9 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. ​ Investment Securities ​ Refer to Note 4 “ Investment securities ” for the fair value of the investment securities as of March 31, 2023 and December 31, 2022. ​ 6 Accrued expenses and other current liabilities ​ Accrued expenses and other current liabilities include the following items: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ March 31, ​ December 31, ​ 2023 2022 ​ ​ (in thousands) Accruals for goods received from and services provided by vendors-not yet billed ​ $ 10,929 ​ $ 11,120 Personnel related accruals and liabilities ​ ​ 9,350 ​ ​ 17,201 Accrued contract fulfillment costs and costs to obtain a contract ​ ​ 2,250 ​ ​ 2,250 Total ​ $ 22,529 ​ $ 30,571 ​ ​ 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”). ​ The Company drew down an additional $ 30.0 million at the time of the 2021 Restated Facility, resulting in total principal outstanding $ 100.0 million. The 2021 Restated Facility matures on December 1, 2025. ​ The Company is required to repay the entire principal balance on the maturity date. 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 $ 2.5 million on June 1, 2023 and a back-end fee of $ 4.9 million on the maturity date. The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2021 Restated Facility was $ 104.3 million as of March 31, 2023, compared to $ 103.8 million as of December 31, 2022, and is recorded net of discount and debt issuance costs. The foreign currency gain on the facility in the three ended March 31, 2023 was $ 0.7 million compared to a foreign currency loss of $ 2.1 million during the same period in 2022. 9 Table of Contents Interest expense associated with the 2021 Restated Facility during the three months ended March 31, 2023 was $ 3.6 million, compared to $ 2.4 million during the same period in 2022. Under the 2021 Restated Facility the Company must remain current in its 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. Beginning on April 1, 2024, the Company is required to keep a minimum of unrestricted cash equal to at least 30 % of the loan amount outstanding. 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 $ 631.0 million, less $ 26.0 million of cash and cash equivalents and other current assets held by uniQure N.V. and $ 85.7 million of other current assets and investment held by Corlieve. Under the 2021 Restated Facility, the occurrence of a material adverse effect, as defined therein, would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of March 31, 2023, the Company was in material compliance with all covenants and provisions. ​ 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”). The number of shares authorized for issuance under the 2014 Plan is 12,601,471 . On April 28, 2023, the Company filed a definitive proxy statement with the SEC, seeking shareholder approval to, among other things, amend and restate the 2014 Plan to increase the shares available thereunder. ​ 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 March 31, ​ ​ 2023 ​ 2022 ​ ​ (in thousands) Cost of manufacturing services revenue ​ $ 24 ​ $ — Research and development ​ ​ 4,305 ​ ​ 4,054 Selling, general and administrative ​ ​ 3,732 ​ ​ 2,814 Total ​ $ 8,061 ​ $ 6,868 ​ 10 Table of Contents Share-based compensation expense recognized by award type of the 2014 Plans as well as the ESPP was as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2023 ​ 2022 ​ ​ (in thousands) Award type/ESPP ​ ​ ​ ​ ​ ​ Share options ​ $ 3,274 ​ $ 3,255 Restricted share units ​ ​ 4,630 ​ ​ 3,270 Performance share units ​ ​ 150 ​ ​ 338 Employee share purchase plan ​ ​ 7 ​ ​ 5 Total ​ $ 8,061 ​ $ 6,868 ​ As of March 31, 2023, 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 ​ $ 32,390 ​ 2.89 Restricted share units ​ ​ 43,140 ​ 2.35 Performance share units ​ ​ 12 ​ 0.12 Total ​ $ 75,542 ​ 2.58 ​ 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. ​ 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 share options that vest must be exercised by the tenth anniversary of the initial grant date. The following tables summarize share option activity under the 2014 Plans for the three months ended March 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ Options ​ ​ Number of ​ Weighted average ​ ordinary shares exercise price ​ ​ ​ ​ ​ ​ Outstanding at December 31, 2022 ​ 4,237,917 ​ $ 26.13 Granted ​ 1,049,350 ​ $ 20.06 Forfeited ​ ( 74,916 ) ​ $ 24.38 Expired ​ ( 51,606 ) ​ $ 54.74 Exercised ​ ( 10,055 ) ​ $ 8.64 Outstanding at March 31, 2023 ​ 5,150,690 ​ $ 24.66 Thereof, fully vested and exercisable on March 31, 2023 ​ 2,438,321 ​ $ 27.41 Thereof, outstanding and expected to vest after March 31, 2023 ​ 2,712,369 ​ $ 22.20 ​ ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of options issued during the period (in $ millions) ​ ​ ​ $ 12.3 Proceeds from option sales during the period (in $ millions) ​ ​ ​ $ 0.1 ​ 11 Table of Contents The fair value of each share 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 March 31, Assumptions 2023 2022 Expected volatility ​ 70 % ​ 70 % Expected terms ​ 10 years ​ 10 years Risk free interest rate ​ 4.10 % ​ 2.12 % Expected dividend yield ​ 0 % ​ 0 % ​ RSUs ​ The following table summarizes the RSU activity for the three months ended March 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ RSUs ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2022 ​ 1,818,774 ​ $ 20.46 Granted ​ 1,202,150 ​ $ 20.06 Vested ​ ( 520,046 ) ​ $ 22.86 Forfeited ​ ( 65,982 ) ​ $ 19.50 Non-vested at March 31, 2023 ​ 2,434,896 ​ $ 19.78 ​ ​ ​ ​ ​ ​ Total weighted average grant date fair value of RSUs granted during the period (in $ millions) ​ ​ ​ $ 24.1 ​ RSUs vest over one to three years . RSUs granted to non-executive directors vest one year from the date of grant. ​ PSUs ​ The following table summarizes the PSU activity for the three months ended March 31, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ PSUs ​ ​ Weighted average ​ ​ Number of ​ grant-date fair ​ ​ ordinary shares ​ value Non-vested at December 31, 2022 ​ 400,690 ​ $ 28.82 Vested ​ ( 46,045 ) ​ $ 28.60 Forfeited ​ ( 17,395 ) ​ $ 26.12 Non-vested at March 31, 2023 ​ 337,250 ​ $ 28.99 ​ ​ ​ ​ ​ ​ ​ The Company granted ordinary shares to certain employees in September and December 2021 and at various dates during the year ended December 31, 2022 that will be earned upon the achievement of defined milestones. Such ordinary 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 ordinary shares granted in December 2021 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. Achievement of one of the total five defined milestones was met as of December 31, 2022 and another one of the total five defined milestones was met as of March 31, 2023. ​ 12 Table of Contents ESPP ​ During the three months ended March 31, 2023, 2,495 ordinary shares were issued under the ESPP compared to 3,558 during the same period in 2022. As of March 31, 2023, a total of 113,565 ordinary shares remains available for issuance under the ESPP plan compared to a total of 123,744 as of March 31, 2022. ​ 9 Income taxes ​ The Company recorded $ 1.2 million deferred tax benefit in relation to its operations in the United States and France during the three months ended March 31, 2023. The Company recorded $ 0.6 million deferred tax benefit in relation to its operations in the United States, France and Switzerland for the three months ended March 31, 2022. ​ The effective income tax rate of ( 1.5 %) during the three months ended March 31, 2023 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 months ended March 31, 2022 was ( 1.3 %) as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands. ​ ​ 10 Basic and diluted earnings per share ​ Diluted earnings per share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. As the Company has incurred a loss, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share. The ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of March 31, 2023 and March 31, 2022, respectively. ​ The potentially dilutive ordinary shares are summarized below: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three months ended March 31, ​ ​ 2023 ​ 2022 Anti-dilutive ordinary share equivalents ​ ​ ​ ​ ​ ​ Stock options under 2014 Plans and previous plan ​ ​ 5,150,690 ​ ​ 4,139,698 Non-vested RSUs and PSUs ​ ​ 2,772,146 ​ ​ 2,287,969 ESPP ​ ​ 801 ​ ​ 745 Total anti-dilutive ordinary share equivalents ​ ​ 7,923,637 ​ ​ 6,428,412 ​ ​ 11 Subsequent events ​ None. ​ ​ 13 Table of Contents