Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2024 | May 02, 2024 | |
Document and Entity Information | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2024 | |
Document Transition Report | false | |
Entity File Number | 001-36294 | |
Entity Registrant Name | uniQure N.V. | |
Entity Incorporation, State or Country Code | P7 | |
Entity Tax Identification Number | 00-0000000 | |
Entity Address, Address Line One | Paasheuvelweg 25 | |
Entity Address, City or Town | Amsterdam | |
Entity Address, Country | NL | |
Entity Address, Postal Zip Code | 1105 BP | |
City Area Code | 31 | |
Local Phone Number | 20-240-6000 | |
Title of 12(b) Security | Ordinary Shares, par value €0.05 | |
Trading Symbol | QURE | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 48,549,437 | |
Entity Central Index Key | 0001590560 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q1 | |
Amendment Flag | false |
UNAUDITED CONSOLIDATED BALANCE
UNAUDITED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current assets | ||
Cash and cash equivalents | $ 243,062 | $ 241,360 |
Current investment securities | 312,621 | 376,532 |
Accounts receivable | 10,717 | 4,193 |
Inventories, net | 7,672 | 12,024 |
Prepaid expenses | 18,839 | 15,089 |
Other current assets and receivables | 3,092 | 2,655 |
Total current assets | 596,003 | 651,853 |
Non-current assets | ||
Property, plant and equipment, net of accumulated depreciation of $57.8 million as of March 31, 2024 and $55.7 million as of December 31, 2023 | 44,554 | 46,548 |
Operating lease right-of-use assets | 27,695 | 28,789 |
Intangible assets, net, including in-process research and development asset of $57.8 million as of March 31, 2024 and $59.1 million as of December 31, 2023 | 59,111 | 60,481 |
Goodwill | 25,795 | 26,379 |
Deferred tax assets, net | 11,594 | 12,276 |
Other non-current assets | 5,298 | 5,363 |
Total non-current assets | 174,047 | 179,836 |
Total assets | 770,050 | 831,689 |
Current liabilities | ||
Accounts payable | 5,231 | 6,586 |
Accrued expenses and other current liabilities | 22,658 | 30,534 |
Current portion of contingent consideration | 27,587 | 28,211 |
Current portion of operating lease liabilities | 7,997 | 8,344 |
Total current liabilities | 63,473 | 73,675 |
Non-current liabilities | ||
Long-term debt | 102,120 | 101,749 |
Liability from royalty financing agreement | 405,398 | 394,241 |
Operating lease liabilities, net of current portion | 26,983 | 28,316 |
Contingent consideration, net of current portion | 14,625 | 14,795 |
Deferred tax liability, net | 7,376 | 7,543 |
Other non-current liabilities | 3,321 | 3,700 |
Total non-current liabilities | 559,823 | 550,344 |
Total liabilities | 623,296 | 624,019 |
Commitments and contingencies | ||
Shareholders' equity | ||
Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of March 31, 2024 and December 31, 2023 and 48,492,357 and 47,833,830 ordinary shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively | 2,919 | 2,883 |
Additional paid-in-capital | 1,155,904 | 1,148,749 |
Accumulated other comprehensive loss | (56,042) | (53,553) |
Accumulated deficit | (956,027) | (890,409) |
Total shareholders' equity | 146,754 | 207,670 |
Total liabilities and shareholders' equity | $ 770,050 | $ 831,689 |
UNAUDITED CONSOLIDATED BALANC_2
UNAUDITED CONSOLIDATED BALANCE SHEETS (Parenthetical) $ in Thousands | Mar. 31, 2024 USD ($) shares | Mar. 31, 2024 € / shares | Dec. 31, 2023 USD ($) shares | Dec. 31, 2023 € / shares |
Accumulated depreciation | $ | $ 57,800 | $ 55,700 | ||
Intangible assets, net | $ | $ 59,111 | $ 60,481 | ||
Ordinary shares, par value (in euros per share) | € / shares | € 0.05 | € 0.05 | ||
Ordinary shares, authorized | shares | 80,000,000 | 80,000,000 | ||
Ordinary shares, issued | shares | 48,492,357 | 47,833,830 | ||
Ordinary shares, outstanding | shares | 48,492,357 | 47,833,830 | ||
In-process research & development | ||||
Intangible assets, net | $ | $ 57,800 | $ 59,100 |
UNAUDITED CONSOLIDATED STATEMEN
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Total revenues | $ 8,485 | $ 5,325 |
Operating expenses: | ||
Cost of license revenues | (150) | |
Cost of contract manufacturing revenues | (9,076) | (2,435) |
Research and development expenses | (40,692) | (60,809) |
Selling, general and administrative expenses | (13,937) | (17,848) |
Total operating expenses | (63,855) | (81,092) |
Other income | 1,376 | 1,811 |
Other expense | (234) | (216) |
Loss from operations | (54,228) | (74,172) |
Interest income | 6,508 | 1,669 |
Interest expense | (16,097) | (3,562) |
Foreign currency losses, net | (1,145) | (2,369) |
Loss before income tax (expense) / benefit | (64,962) | (78,434) |
Income tax (expense) / benefit | (656) | 1,207 |
Net loss | (65,618) | (77,227) |
Other comprehensive (loss) / income: | ||
Foreign currency translation (loss) / gains, net | (2,524) | 5,797 |
Defined benefit pension gain, net of taxes | 35 | |
Total comprehensive loss | $ (68,107) | $ (71,430) |
Earnings per ordinary share - basic | ||
Basic net (loss) / income per ordinary share | $ (1.36) | $ (1.63) |
Earnings per ordinary share - diluted | ||
Diluted net (loss) / income per ordinary share | $ (1.36) | $ (1.63) |
Weighted average shares - basic | 48,384,510 | 47,436,335 |
Weighted average shares - diluted | 48,384,510 | 47,436,335 |
License revenues | ||
Total revenues | $ 1,202 | $ 0 |
Contract manufacturing revenues | ||
Total revenues | 3,990 | 4,937 |
Collaboration revenues | ||
Total revenues | $ 3,293 | $ 388 |
UNAUDITED CONSOLIDATED STATEM_2
UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS' EQUITY - USD ($) $ in Thousands | Ordinary shares | Additional paid-in capital | Accumulated other comprehensive income /(loss) | Accumulated deficit | Total |
Beginning balance at Dec. 31, 2022 | $ 2,838 | $ 1,113,393 | $ (58,291) | $ (581,931) | $ 476,009 |
Beginning balance (in shares) at Dec. 31, 2022 | 46,968,032 | ||||
Increase (decrease) in shareholders' equity | |||||
Loss for the period | (77,227) | (77,227) | |||
Other comprehensive income (loss), net | 5,797 | 5,797 | |||
Exercises of share options | $ 1 | 86 | 87 | ||
Exercises of share options (in shares) | 10,055 | ||||
Restricted and performance share units distributed during the period | $ 30 | (30) | |||
Restricted and performance share units distributed during the period (in shares) | 566,091 | ||||
Share-based compensation expense | 8,061 | 8,061 | |||
Issuance of ordinary shares relating to employee stock purchase plan | 44 | 44 | |||
Issuance of ordinary shares relating to employee stock purchase plan (in shares) | 2,495 | ||||
Ending balance at Mar. 31, 2023 | $ 2,869 | 1,121,554 | (52,494) | (659,158) | 412,771 |
Ending balance (in shares) at Mar. 31, 2023 | 47,546,673 | ||||
Beginning balance at Dec. 31, 2023 | $ 2,883 | 1,148,749 | (53,553) | (890,409) | $ 207,670 |
Beginning balance (in shares) at Dec. 31, 2023 | 47,833,830 | 47,833,830 | |||
Increase (decrease) in shareholders' equity | |||||
Loss for the period | (65,618) | $ (65,618) | |||
Other comprehensive income (loss), net | (2,489) | (2,489) | |||
Restricted and performance share units distributed during the period | $ 36 | (36) | |||
Restricted and performance share units distributed during the period (in shares) | 658,527 | ||||
Share-based compensation expense | 7,191 | 7,191 | |||
Ending balance at Mar. 31, 2024 | $ 2,919 | $ 1,155,904 | $ (56,042) | $ (956,027) | $ 146,754 |
Ending balance (in shares) at Mar. 31, 2024 | 48,492,357 | 48,492,357 |
UNAUDITED CONSOLIDATED STATEM_3
UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Cash flows from operating activities | ||
Net loss | $ (65,618) | $ (77,227) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 2,629 | 2,527 |
Amortization of discount on investment securities | (3,695) | (925) |
Share-based compensation expense | 7,191 | 8,061 |
Royalty financing agreement interest expense | 12,362 | |
Deferred tax expense / (income) | 656 | (1,207) |
Changes in fair value of contingent consideration | 165 | 975 |
Unrealized foreign exchange losses, net | 1,389 | 1,193 |
Other items, net | 2,778 | 456 |
Changes in operating assets and liabilities: | ||
Accounts receivable, prepaid expenses, and other current assets and receivables | (10,417) | (936) |
Inventories | 2,236 | (553) |
Accounts payable | (701) | (1,661) |
Accrued expenses, other liabilities, and operating leases | (9,550) | (9,005) |
Net cash used in operating activities | (60,575) | (78,302) |
Cash flows from investing activities | ||
Proceeds on maturity of debt securities | 150,107 | 5,330 |
Investment in debt securities | (83,778) | |
Purchases of property, plant, and equipment | (2,344) | (2,342) |
Net cash generated from investing activities | 63,985 | 2,988 |
Cash flows from financing activities | ||
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans | 131 | |
Net cash generated from financing activities | 131 | |
Currency effect on cash, cash equivalents and restricted cash | (1,725) | 1,034 |
Net increase / (decrease) in cash, cash equivalents and restricted cash | 1,685 | (74,149) |
Cash, cash equivalents and restricted cash at beginning of period | 244,544 | 231,173 |
Cash, cash equivalents and restricted cash at the end of period | 246,229 | 157,024 |
Cash and cash equivalents | 243,062 | 153,851 |
Restricted cash related to leasehold and other deposits | 3,167 | 3,173 |
Total cash, cash equivalents and restricted cash | 246,229 | 157,024 |
Supplemental cash flow disclosures: | ||
Cash paid for interest | (4,761) | (3,057) |
Non-cash decrease in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment | $ (577) | $ (753) |
General business information
General business information | 3 Months Ended |
Mar. 31, 2024 | |
General business information | |
General business information | 1 uniQure N.V. (the “Company”) was incorporated on January 9, 2012, as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid naamloze vennootschap The Company is registered in the trade register of the Dutch Chamber of Commerce ( Kamer van Koophandel The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”. |
Summary of significant accounti
Summary of significant accounting policies | 3 Months Ended |
Mar. 31, 2024 | |
Summary of significant accounting policies | |
Summary of significant accounting policies | 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 (“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 United States (“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, 2024, are not necessarily indicative of the results to be expected for the full year ending December 31, 2024, 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 period. Actual results could differ from those estimates. 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, 2023, and the notes thereto, which are included in the Annual Report 2.5 Recent accounting pronouncements There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2024, as compared to the recent accounting pronouncements described in Note 2.3.27 of the Annual Report |
CSL Behring collaboration
CSL Behring collaboration | 3 Months Ended |
Mar. 31, 2024 | |
CSL Behring collaboration | |
CSL Behring collaboration | 3 CSL Behring collaboration On June 24, 2020, uniQure biopharma B.V. entered into a commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”), pursuant to which CSL Behring received exclusive global rights to HEMGENIX® The transaction became fully effective on May 6, 2021. License revenue The Company recognized $1.2 million of royalty revenue in the three months ended , compared to nil in the three months ended . Royalties on the sale of HEMGENIX® are recorded once earned and are presented as license revenue. Accounts receivable and contract asset As of December 31, 2023, the Company recorded accounts receivable of $4.0 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue. As of , the Company had accounts receivable of $10.6 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue. |
Investment securities
Investment securities | 3 Months Ended |
Mar. 31, 2024 | |
Investment securities | |
Investment securities | 4 Investment securities The following tables summarize the Company’s investments in sovereign debt as of March 31, 2024 and December 31, 2023: At March 31, 2024 Amortized cost Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 312,621 $ 12 $ — $ 312,633 Total $ 312,621 $ 12 $ — $ 312,633 At December 31, 2023 Amortized cost Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 376,532 $ 139 $ — $ 376,671 Total $ 376,532 $ 139 $ — $ 376,671 The Company invests in short-term U.S. and European government bonds with the highest investment credit rating. The U.S. and European government bonds are U.S. dollar and euro denominated, respectively. Inputs to the fair value of the investments are considered Level 2 inputs. |
Inventories, net
Inventories, net | 3 Months Ended |
Mar. 31, 2024 | |
Inventories, net | |
Inventories, net | 5 Inventories, net The following table summarizes the inventories, net balances as of March 31, 2024 and December 31, 2023: March 31, December 31, 2024 2023 (in thousands) Raw materials $ 6,331 $ 7,157 Work in progress 1,341 4,109 Finished goods — 758 Inventories $ 7,672 $ 12,024 The Company recorded write downs to net realizable value of $1.8 million for the three months ended and nil for the same period in 2023. The costs are recognized as Cost of Contract Manufacturing Revenues. |
Fair value measurement
Fair value measurement | 3 Months Ended |
Mar. 31, 2024 | |
Fair value measurement | |
Fair value measurement | 6 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 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, 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, 2024 and December 31, 2023 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, 2023 Assets: Cash and cash equivalents $ 241,360 $ — $ — $ 241,360 Cash and cash equivalents Restricted cash 3,184 — — 3,184 Other non-current assets Total assets $ 244,544 $ — $ — $ 244,544 Liabilities: Contingent consideration — — 43,006 43,006 Contingent consideration Consideration for post-acquisition services — — 457 457 Other non-current liabilities Total liabilities $ — $ — $ 43,463 $ 43,463 At March 31, 2024 Assets: Cash and cash equivalents $ 243,062 $ — $ — $ 243,062 Cash and cash equivalents Restricted cash 3,167 — — 3,167 Other non-current assets Total assets $ 246,229 $ — $ — $ 246,229 Liabilities: Contingent consideration — — 42,212 42,212 Contingent consideration Consideration for post-acquisition services — — 498 498 Other non-current liabilities Total liabilities $ — $ — $ 42,710 $ 42,710 Contingent consideration The Company is required to pay up to EUR 178.8 million (or $193.0 million based on the foreign exchange rate on March 31, 2024) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of contractually defined milestones in connection with the Company’s July 2021 acquisition of uniQure France SAS. The fair value of the contingent consideration as of March 31, 2024 was $42.2 million (December 31, 2023: $43.0 million) using discount rates of approximately 14.8% to 15.6% (December 31, 2023: 15.3% to 15.6%). The Company assumes the probability of achieving a EUR 30.0 million ($32.4 million) milestone payment following the dosing of the first patient in Phase I/II clinical trial to be 100%. If as of March 31, 2024 the Company had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to $74.8 million. If as of March 31, 2024 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income. The following table presents the changes in fair value of contingent consideration between December 31, 2023 and March 31, 2024: Amount of contingent consideration 2024 (in thousands) Balance at December 31, 2023 $ 43,006 Change in fair value (presented within research and development expenses) 165 Currency translation effects (959) Balance at March 31, 2024 $ 42,212 As of March 31, 2024, the Company classified $27.6 million (December 31, 2023: $28.2 million) of the total contingent consideration of $42.2 million (December 31, 2023: $43.0 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 |
Accrued expenses and other curr
Accrued expenses and other current liabilities | 3 Months Ended |
Mar. 31, 2024 | |
Accrued expenses and other current liabilities | |
Accrued expenses and other current liabilities | 7 Accrued expenses and other current liabilities Accrued expenses and other current liabilities include the following items: March 31, December 31, 2024 2023 (in thousands) Personnel related accruals and liabilities $ 9,572 $ 16,263 Accruals for goods received from and services provided by vendors-not yet billed 11,881 12,834 Liability owed to the Purchaser pursuant to the Royalty Financing Agreement 1,205 1,437 Total $ 22,658 $ 30,534 |
Long-term debt
Long-term debt | 3 Months Ended |
Mar. 31, 2024 | |
Long-term debt | |
Long-term debt | 8 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, January 2021, December 2021 (the “2021 Restated Facility”) and on May 12, 2023 (the “2023 Amended Facility”). The total principal outstanding under the 2023 Amended Facility is $100.0 million. The 2023 Amended Facility extends the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”). 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 The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2023 Amended Facility was $103.3 million as of March 31, 2024, compared to $102.9 million as of December 31, 2023, and is recorded net of discount and debt issuance costs. The foreign currency loss on the facility in the three months ended March 31, 2024 was $2.3 million, compared to a foreign currency gain of $0.7 million during the same period in 2023. Interest expense associated with the 2023 Amended Facility during the three months ended March 31, 2024 was $3.7 million, compared to $3.6 million during the same period in 2023. Under the 2023 Amended 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 U.S. 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 2023 Amended 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 $770.1 million, less $6.8 million of cash and cash equivalents and other current assets held by the Company, and $87.7 million of other current assets and investment held by uniQure France SAS as well as receivables sold to the Purchaser. Under the 2023 Amended 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, 2024, the Company was in material compliance with all covenants and provisions. |
Royalty Financing Agreement
Royalty Financing Agreement | 3 Months Ended |
Mar. 31, 2024 | |
Royalty Financing Agreement | |
Royalty Financing Agreement | 9 On May 12, 2023, the Company entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. (the “Purchaser”). Under the terms of the Royalty Financing Agreement the Company received an upfront payment of $375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to the Company. The Company is also eligible to receive an additional $25.0 million milestone payment under the Royalty Financing Agreement if 2024 net sales of HEMGENIX® exceed a pre-specified threshold, as set forth in the Royalty Financing Agreement. The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) and 1.85 times the $25.0 million milestone payment (if paid) until June 30, 2032 (“First Hard Cap Date”) if such thresholds are met or, if such cap is not met by June 30, 2032, up to 2.25 times of the upfront and milestone payment (if paid) through December 31, 2038. If 2024 net sales do not exceed a pre-specified threshold, the Company will be obligated to pay $25.0 million to the Purchaser but only to the extent that the Company achieves a future sales milestone under the CSL Behring Agreement. If such milestone payment is not due from CSL Behring, the Company is not obligated to pay any amounts to the Purchaser. The Company has retained the rights to all other royalties, as well as contractual milestones totaling up to $1.3 billion, under the terms of the CSL Behring Agreement. Net proceeds from the Royalty Financing Agreement, after deducting professional and financial advisory fees related to the transaction of $4.9 million, were $370.1 million. The Company initially recorded these net proceeds as “Liability from royalty financing agreement” at their fair market value on its balance sheet as of closing of the transaction on June 5, 2023. Following the initial recognition, the Company records the debt at amortized cost. The Company expects to satisfy its commitment to the Purchaser prior to the First Hard Cap Date. The Company will record the difference of $323.7 million between the total expected payments of $693.8 million to the Purchaser and the $370.1 million net proceeds as interest expense using the effective interest rate method. The Company determined the effective interest rate based on the projected cash flows up to the First Hard Cap Date. Based on the Company’s projections the effective interest rate is expected to be within a range of 12.0% per annum to 13.5% per annum. The Company would have recorded between $12.1 million and $13.7 million of interest expense through the three months ended March 31, 2024 (nil for the three months ended March 31, 2023) if it had used 12.0% or 13.5%, instead of the $12.4 million recorded in the three months ended March 31, 2024 (nil for the three months ended March 31, 2023). The Company will prospectively update the effective interest rate at each reporting date based on updated projections. The liability was initially recognized at fair value and inputs were considered Level 3 inputs. The following table presents the movement in the liability related to the Royalty Financing Agreement between the December 31, 2023 and March 31, 2024: Amount of liability (in thousands) Balance as of December 31, 2023 (includes $1.4 million presented as "Accrued expenses and other current liabilities) $ 395,678 Royalty payments to Purchaser (1,437) Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") (1,205) Interest expense for the period 12,362 Liability related to the royalty financing agreement $ 405,398 |
Share-based compensation
Share-based compensation | 3 Months Ended |
Mar. 31, 2024 | |
Share-based compensation | |
Share-based compensation | 10 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 14 ,351,471 . In June 2018, the Company’s shareholders adopted and approved an employee share purchase plan (the “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, as amended. 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, 2024 2023 (in thousands) Cost of manufacturing services revenue $ 345 $ 24 Research and development 3,425 4,305 Selling, general and administrative 3,421 3,732 Total $ 7,191 $ 8,061 Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows: Three months ended March 31, 2024 2023 (in thousands) Award type/ESPP Share options $ 2,913 $ 3,274 Restricted share units 4,370 4,630 Performance share units (99) 150 Employee share purchase plan 7 7 Total $ 7,191 $ 8,061 As of March 31, 2024, 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 $ 20,743 2.56 Restricted share units 27,981 1.99 Performance share units 80 0.61 Total $ 48,804 2.23 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% of each grant 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 2014 Plans for the three months ended March 31, 2024: Options Number of Weighted average ordinary shares exercise price Outstanding at December 31, 2023 4,974,030 23.25 Granted 930,900 $ 5.59 Forfeited (71,437) $ 21.26 Expired (11,584) $ 37.61 Outstanding at March 31, 2024 5,821,909 $ 20.42 Thereof, fully vested, and exercisable on March 31, 2024 3,054,242 $ 25.70 Thereof, outstanding and expected to vest after March 31, 2024 2,767,667 $ 14.60 Outstanding and expected to vest after December 31, 2023 2,098,557 $ 23.38 Total weighted average grant date fair value of options issued during the period (in $ millions) $ 3.0 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 March 31, Assumptions 2024 2023 Expected volatility 70% 70% Expected terms 10 years 10 years Risk free interest rate 4.32% 4.10% Expected dividend yield 0% 0% RSUs The following table summarizes the RSU activity for the three months ended March 31, 2024: RSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2023 2,264,369 $ 18.07 Granted 1,160,800 $ 5.59 Vested (659,240) $ 20.30 Forfeited (58,401) $ 17.39 Non-vested at March 31, 2024 2,707,528 $ 12.19 Total weighted average grant date fair value of RSUs granted during the period (in $ millions) $ 6.5 RSUs generally vest over one PSUs The following table summarizes the PSU activity for the three months ended March 31, 2024: PSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2023 222,550 $ 28.09 Forfeited (17,030) $ 27.12 Non-vested at March 31, 2024 205,520 $ 27.99 The Company granted ordinary shares to certain employees in December 2021 and at various dates during the year ended December 31, 2022 that will be earned upon achievement of defined milestones. Earned 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. As of March 31, 2024, two milestones had been achieved and vested in either 2022 or 2023. Additionally, another two milestones are considered probable as of December 31, 2023 and March 31, 2024. The ESPP During the three months ended March 31, 2024, nil ordinary shares were issued under the ESPP compared to 2,495 during the same period in 2023. As of March 31, 2024, 96,862 ordinary shares remain available for issuance under the ESPP compared to a total of 113,565 as of March 31, 2023. |
Income taxes
Income taxes | 3 Months Ended |
Mar. 31, 2024 | |
Income taxes | |
Income taxes | 11 Income taxes The Company recorded $0.7 million deferred tax expense in relation to its operations in the U.S. during the three months ended March 31, 2024. The Company recorded $1.2 million deferred tax benefit in relation to its operations in the U.S. and France during the three months ended March 31, 2023. The effective income tax rate of 1.0% during the three ended March 31, 2024 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 and a partial a valuation allowance against its net deferred tax assets in France. The effective income tax rate during the three months ended March 31, 2023 was (1.5%), as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands. |
Basic and diluted earnings per
Basic and diluted earnings per share | 3 Months Ended |
Mar. 31, 2024 | |
Basic and diluted earnings per share | |
Basic and diluted earnings per share | 14 Basic and diluted earnings per share The potentially dilutive ordinary shares are summarized below: Three months ended March 31, 2024 2023 Anti-dilutive ordinary share equivalents Stock options under 2014 Plans and previous plan 5,821,909 5,150,690 Non-vested RSUs and PSUs 2,913,048 2,772,146 ESPP 3,140 801 Total anti-dilutive ordinary share equivalents 8,738,097 7,923,637 |
Subsequent events
Subsequent events | 3 Months Ended |
Mar. 31, 2024 | |
Subsequent events | |
Subsequent events | 13 None. |
Summary of significant accoun_2
Summary of significant accounting policies (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Summary of significant accounting policies | |
Basis of preparation | 2.1 Basis of preparation The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States (“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 United States (“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. |
Unaudited interim financial information | 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, 2024, are not necessarily indicative of the results to be expected for the full year ending December 31, 2024, 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 |
Use of estimates | 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 period. Actual results could differ from those estimates. |
Accounting policies | 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, 2023, and the notes thereto, which are included in the Annual Report |
Recent accounting pronouncements | 2.5 Recent accounting pronouncements There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2024, as compared to the recent accounting pronouncements described in Note 2.3.27 of the Annual Report |
Investment securities (Tables)
Investment securities (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Investment securities | |
Schedule of investments in sovereign debt | At March 31, 2024 Amortized cost Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 312,621 $ 12 $ — $ 312,633 Total $ 312,621 $ 12 $ — $ 312,633 At December 31, 2023 Amortized cost Gross unrealized holding gains Gross unrealized holding losses Estimated fair value (in thousands) Current investments: Government debt securities (held-to-maturity) $ 376,532 $ 139 $ — $ 376,671 Total $ 376,532 $ 139 $ — $ 376,671 |
Inventories, net (Tables)
Inventories, net (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Inventories, net | |
Schedule of inventories, net balances | March 31, December 31, 2024 2023 (in thousands) Raw materials $ 6,331 $ 7,157 Work in progress 1,341 4,109 Finished goods — 758 Inventories $ 7,672 $ 12,024 |
Fair value measurement (Tables)
Fair value measurement (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Fair value measurement | |
Schedule of assets and liabilities measured at fair value on recurring basis | 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, 2023 Assets: Cash and cash equivalents $ 241,360 $ — $ — $ 241,360 Cash and cash equivalents Restricted cash 3,184 — — 3,184 Other non-current assets Total assets $ 244,544 $ — $ — $ 244,544 Liabilities: Contingent consideration — — 43,006 43,006 Contingent consideration Consideration for post-acquisition services — — 457 457 Other non-current liabilities Total liabilities $ — $ — $ 43,463 $ 43,463 At March 31, 2024 Assets: Cash and cash equivalents $ 243,062 $ — $ — $ 243,062 Cash and cash equivalents Restricted cash 3,167 — — 3,167 Other non-current assets Total assets $ 246,229 $ — $ — $ 246,229 Liabilities: Contingent consideration — — 42,212 42,212 Contingent consideration Consideration for post-acquisition services — — 498 498 Other non-current liabilities Total liabilities $ — $ — $ 42,710 $ 42,710 |
Schedule of changes in fair value of contingent consideration | Amount of contingent consideration 2024 (in thousands) Balance at December 31, 2023 $ 43,006 Change in fair value (presented within research and development expenses) 165 Currency translation effects (959) Balance at March 31, 2024 $ 42,212 |
Accrued expenses and other cu_2
Accrued expenses and other current liabilities (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Accrued expenses and other current liabilities | |
Schedule of accrued expenses and other current liabilities | March 31, December 31, 2024 2023 (in thousands) Personnel related accruals and liabilities $ 9,572 $ 16,263 Accruals for goods received from and services provided by vendors-not yet billed 11,881 12,834 Liability owed to the Purchaser pursuant to the Royalty Financing Agreement 1,205 1,437 Total $ 22,658 $ 30,534 |
Royalty financing agreement (Ta
Royalty financing agreement (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Royalty Financing Agreement | |
Schedule of movement in the liability related to royalty financing agreement | Amount of liability (in thousands) Balance as of December 31, 2023 (includes $1.4 million presented as "Accrued expenses and other current liabilities) $ 395,678 Royalty payments to Purchaser (1,437) Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") (1,205) Interest expense for the period 12,362 Liability related to the royalty financing agreement $ 405,398 |
Share-based compensation (Table
Share-based compensation (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Share-based compensation | |
Schedule of share-based compensation expense by classification included in consolidated statements of operations and comprehensive loss | Three months ended March 31, 2024 2023 (in thousands) Cost of manufacturing services revenue $ 345 $ 24 Research and development 3,425 4,305 Selling, general and administrative 3,421 3,732 Total $ 7,191 $ 8,061 |
Schedule of share-based compensation expense | Three months ended March 31, 2024 2023 (in thousands) Award type/ESPP Share options $ 2,913 $ 3,274 Restricted share units 4,370 4,630 Performance share units (99) 150 Employee share purchase plan 7 7 Total $ 7,191 $ 8,061 |
Schedule of unrecognized compensation cost related to unvested awards | Unrecognized Weighted average share-based remaining compensation period for expense recognition (in thousands) (in years) Award type Share options $ 20,743 2.56 Restricted share units 27,981 1.99 Performance share units 80 0.61 Total $ 48,804 2.23 |
Schedule of weighted-average assumptions for fair value of option issued | Three months ended March 31, Assumptions 2024 2023 Expected volatility 70% 70% Expected terms 10 years 10 years Risk free interest rate 4.32% 4.10% Expected dividend yield 0% 0% |
Summary of RSUs activity | RSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2023 2,264,369 $ 18.07 Granted 1,160,800 $ 5.59 Vested (659,240) $ 20.30 Forfeited (58,401) $ 17.39 Non-vested at March 31, 2024 2,707,528 $ 12.19 Total weighted average grant date fair value of RSUs granted during the period (in $ millions) $ 6.5 |
Summary of PSUs activity | PSUs Weighted average Number of grant-date fair ordinary shares value Non-vested at December 31, 2023 222,550 $ 28.09 Forfeited (17,030) $ 27.12 Non-vested at March 31, 2024 205,520 $ 27.99 |
2014 Plan | |
Share-based compensation | |
Summary of option activity | Options Number of Weighted average ordinary shares exercise price Outstanding at December 31, 2023 4,974,030 23.25 Granted 930,900 $ 5.59 Forfeited (71,437) $ 21.26 Expired (11,584) $ 37.61 Outstanding at March 31, 2024 5,821,909 $ 20.42 Thereof, fully vested, and exercisable on March 31, 2024 3,054,242 $ 25.70 Thereof, outstanding and expected to vest after March 31, 2024 2,767,667 $ 14.60 Outstanding and expected to vest after December 31, 2023 2,098,557 $ 23.38 Total weighted average grant date fair value of options issued during the period (in $ millions) $ 3.0 |
Basic and diluted earnings pe_2
Basic and diluted earnings per share (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Basic and diluted earnings per share | |
Schedule of potential dilutive common shares | Three months ended March 31, 2024 2023 Anti-dilutive ordinary share equivalents Stock options under 2014 Plans and previous plan 5,821,909 5,150,690 Non-vested RSUs and PSUs 2,913,048 2,772,146 ESPP 3,140 801 Total anti-dilutive ordinary share equivalents 8,738,097 7,923,637 |
CSL Behring collaboration (Deta
CSL Behring collaboration (Details) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Collaboration arrangements | |||
Revenue | $ 8,485 | $ 5,325 | |
CLS | |||
Collaboration arrangements | |||
Accounts receivable | 10,600 | $ 4,000 | |
License revenues | |||
Collaboration arrangements | |||
Revenue | $ 1,202 | $ 0 |
Investment securities (Details)
Investment securities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current investments: | ||
Amortized cost | $ 312,621 | $ 376,532 |
Gross unrealized holding gains | 12 | 139 |
Estimated fair value | 312,633 | 376,671 |
Government debt securities (held-to-maturity) | ||
Current investments: | ||
Amortized cost | 312,621 | 376,532 |
Gross unrealized holding gains | 12 | 139 |
Estimated fair value | $ 312,633 | $ 376,671 |
Inventories, net (Details)
Inventories, net (Details) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Inventories, net | |||
Raw materials | $ 6,331 | $ 7,157 | |
Work in progress | 1,341 | 4,109 | |
Finished goods | 758 | ||
Inventories | 7,672 | 12,024 | |
Inventory write downs | 1,800 | $ 0 | |
Allowance for inventory | $ 2,700 | $ 1,600 |
Fair value measurement - Assets
Fair value measurement - Assets and liabilities measured on a recurring basis (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 |
Assets: | |||
Restricted cash | $ 3,167 | $ 3,173 | |
Recurring Fair Value Measurements | |||
Assets: | |||
Cash and cash equivalents | 243,062 | $ 241,360 | |
Restricted cash | 3,167 | 3,184 | |
Total assets | 246,229 | 244,544 | |
Liabilities: | |||
Contingent consideration | 42,212 | 43,006 | |
Consideration for post-acquisition services | 498 | 457 | |
Total liabilities | 42,710 | 43,463 | |
Recurring Fair Value Measurements | Fair value hierarchy Level 1 | |||
Assets: | |||
Cash and cash equivalents | 243,062 | 241,360 | |
Restricted cash | 3,167 | 3,184 | |
Total assets | 246,229 | 244,544 | |
Recurring Fair Value Measurements | Fair value hierarchy Level 3 | |||
Liabilities: | |||
Contingent consideration | 42,212 | 43,006 | |
Consideration for post-acquisition services | 498 | 457 | |
Total liabilities | $ 42,710 | $ 43,463 |
Fair value measurement - Contin
Fair value measurement - Contingent Consideration (Details) $ in Thousands, € in Millions | 3 Months Ended | 12 Months Ended | |||
Mar. 31, 2024 USD ($) | Mar. 31, 2024 EUR (€) | Mar. 31, 2023 USD ($) | Dec. 31, 2023 USD ($) | Mar. 31, 2024 EUR (€) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |||||
Contingent consideration, net of current portion | $ 14,625 | $ 14,795 | |||
Changes in fair value of contingent consideration | 165 | $ 975 | |||
Contingent consideration | 42,200 | 43,000 | |||
Current portion of contingent consideration | $ 27,587 | 28,211 | |||
One hundred percent likelihood of AMT-260 advancing into clinical development | |||||
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |||||
Percentage of increase in Probability of Success | 100% | 100% | |||
Changes in fair value of contingent consideration | $ 74,800 | ||||
uniQure France [S.A.S] | |||||
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |||||
Contingent consideration, net of current portion | $ 42,212 | $ 43,006 | |||
Percentage of increase in Probability of Success | 100% | 100% | |||
Milestone payment achieving due to increase In Probability Of Success In Clinical Development | $ 32,400 | € 30 | |||
Changes in fair value of contingent consideration | 165 | ||||
uniQure France [S.A.S] | Maximum | |||||
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |||||
Contingent consideration payable upon achievement of contractually defined milestones | $ 193,000 | € 178.8 | |||
Contingent consideration discount rate percentage | 15.60% | 15.60% | 15.60% | ||
uniQure France [S.A.S] | Minimum | |||||
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |||||
Contingent consideration discount rate percentage | 14.80% | 14.80% | 15.30% |
Fair value measurement - Change
Fair value measurement - Changes in fair value of contingent consideration (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | ||
Beginning Balance | $ 14,795 | |
Change in fair value (presented within research and development expenses) | 165 | $ 975 |
Ending Balance | 14,625 | |
uniQure France [S.A.S] | ||
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | ||
Beginning Balance | 43,006 | |
Change in fair value (presented within research and development expenses) | 165 | |
Currency translation effects | (959) | |
Ending Balance | $ 42,212 |
Accrued expenses and other cu_3
Accrued expenses and other current liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Accrued expenses and other current liabilities | ||
Personnel related accruals and liabilities | $ 9,572 | $ 16,263 |
Accruals for goods received from and services provided by vendors-not yet billed | 11,881 | 12,834 |
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement | 1,205 | 1,437 |
Total | $ 22,658 | $ 30,534 |
Long-term debt (Details)
Long-term debt (Details) - USD ($) $ in Millions | 3 Months Ended | |||||
Apr. 01, 2024 | Mar. 31, 2024 | Mar. 31, 2023 | Jan. 05, 2027 | Dec. 01, 2025 | Dec. 31, 2023 | |
Long-term Debt | ||||||
Investment, Variable Interest Rate, Type [Extensible Enumeration] | Prime Rate | |||||
2023 Amended Facility | ||||||
Long-term Debt | ||||||
Outstanding debt | $ 100 | |||||
Interest rate (as a percent) | 7.95% | |||||
Amortized cost net of discount and debt issuance costs | $ 103.3 | $ 102.9 | ||||
Foreign currency loss | 2.3 | |||||
Foreign currency gain | $ 0.7 | |||||
Interest expense recorded | $ 3.7 | $ 3.6 | ||||
2023 Amended Facility | Forecast | ||||||
Long-term Debt | ||||||
Back-end fees | $ 1.3 | $ 4.9 | ||||
2023 Amended Facility | Prime Rate | ||||||
Long-term Debt | ||||||
Variable interest rate basis | 7.95% | |||||
Discount rate (as a percent) | 3.25% | |||||
Venture debt loan facility | 2023 Amended Facility | ||||||
Long-term Debt | ||||||
Minimum cash and cash equivalents in U.S. bank accounts | 65% | 100% | ||||
Assets pledged to secure facilities by directly or indirectly | $ 770.1 | |||||
Assets not being pledged to secure facilities by directly or indirectly | 6.8 | |||||
Venture debt loan facility | 2023 Amended Facility | uniQure France | ||||||
Long-term Debt | ||||||
Assets not being pledged to secure facilities by directly or indirectly | $ 87.7 | |||||
Subsequent events. | Venture debt loan facility | 2023 Amended Facility | ||||||
Long-term Debt | ||||||
Minimum cash and cash equivalents in U.S. bank accounts | 30% |
Royalty financing agreement (De
Royalty financing agreement (Details) $ in Thousands | 3 Months Ended | ||||
May 12, 2023 USD ($) | Mar. 31, 2024 USD ($) | Mar. 31, 2023 USD ($) | Dec. 31, 2038 | Jun. 30, 2032 USD ($) | |
Collaboration arrangements | |||||
Royalty financing agreement interest expense | $ 12,362 | ||||
Interest Expense | 16,097 | $ 3,562 | |||
CLS | |||||
Collaboration arrangements | |||||
Maximum rights to all other royalties, as well as contractual milestones retained | 1,300,000 | ||||
Royalty financing agreement | |||||
Collaboration arrangements | |||||
Upfront payment received | $ 375,000 | ||||
Additional milestone payment | $ 25,000 | ||||
Ratio of upfront payment | 1.85 | ||||
Upfront payment payable to purchaser | $ 693,800 | 693,800 | |||
Royalty financing agreement interest expense | 12,362 | ||||
Net proceeds from the Royalty Financing Agreement | 370,100 | ||||
Payment of debt issuance costs | 4,900 | ||||
Interest Expense | $ 12,400 | $ 0 | |||
Royalty financing agreement | Minimum | |||||
Collaboration arrangements | |||||
Effective interest rate (as a percent) | 12% | ||||
Interest Expense | $ 12,100 | ||||
Royalty financing agreement | Maximum | |||||
Collaboration arrangements | |||||
Effective interest rate (as a percent) | 13.50% | ||||
Interest Expense | $ 13,700 | ||||
Royalty financing agreement | First Hard Cap Date | |||||
Collaboration arrangements | |||||
Ratio of upfront payment | 1.85 | ||||
Upfront payment payable to purchaser | $ 25,000 | ||||
Royalty financing agreement interest expense | 323,700 | ||||
Royalty financing agreement | If paid through December 31, 2038 | |||||
Collaboration arrangements | |||||
Ratio of upfront payment | 2.25 | ||||
Royalty financing agreement | If 2024 net sales do not exceed a pre-specified threshold | |||||
Collaboration arrangements | |||||
Upfront payment payable to purchaser | $ 25,000 |
Royalty financing agreement - M
Royalty financing agreement - Movement in the liability (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Collaboration arrangements | ||
Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") | $ (1,205) | $ (1,437) |
Interest expense for the period | 12,362 | |
Liability related to the royalty financing agreement | 405,398 | 394,241 |
Royalty financing agreement | ||
Collaboration arrangements | ||
Balance as of December 31, 2023 (includes $1.4 million presented as "Accrued expenses and other current liabilities) | $ 395,678 | |
Royalty payments to Purchaser | (1,437) | |
Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities") | (1,205) | |
Interest expense for the period | 12,362 | |
Liability related to the royalty financing agreement | $ 405,398 |
Share-based compensation - Summ
Share-based compensation - Summary of share-based compensation expense and unrecognized costs (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | |
Jun. 30, 2018 | Mar. 31, 2024 | Mar. 31, 2023 | |
Share-based compensation | |||
Authorized shares | 14 | ||
2014 Plan | |||
Share-based compensation | |||
Authorized shares | 14 | ||
Share-based compensation expense | $ 7,191 | $ 8,061 | |
Unrecognized compensation costs | $ 48,804 | ||
Weighted-average remaining period for recognition (in years) | 2 years 2 months 23 days | ||
2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | $ 7,191 | 8,061 | |
Research and development expenses | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | 3,425 | 4,305 | |
Selling, general and administrative expense | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | 3,421 | 3,732 | |
Cost of manufacturing services revenues | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | 345 | 24 | |
Share options | 2014 Plan | |||
Share-based compensation | |||
Unrecognized compensation costs | $ 20,743 | ||
Weighted-average remaining period for recognition (in years) | 2 years 6 months 21 days | ||
Share options | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | $ 2,913 | 3,274 | |
Restricted share units ("RSUs") | 2014 Plan | |||
Share-based compensation | |||
Unrecognized compensation costs | $ 27,981 | ||
Weighted-average remaining period for recognition (in years) | 1 year 11 months 26 days | ||
Restricted share units ("RSUs") | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | $ 4,370 | 4,630 | |
Performance Share Awards | 2014 Plan | |||
Share-based compensation | |||
Unrecognized compensation costs | $ 80 | ||
Weighted-average remaining period for recognition (in years) | 7 months 9 days | ||
Performance Share Awards | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | $ (99) | $ 150 | |
Employee share purchase plan | Employee share purchase plan (ESPP) | |||
Share-based compensation | |||
Ordinary shares available for issue | 150,000 | 96,862 | 113,565 |
Discounted rate for purchase of shares | 85% | ||
Employee share purchase plan | 2014 Plans and ESPP | |||
Share-based compensation | |||
Share-based compensation expense | $ 7 | $ 7 |
Share-based compensation - Opti
Share-based compensation - Option activity and weighted-average assumptions (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Weighted average exercise price | |||
Total weighted average grant date fair value of options issued during the period (in $ millions) | $ 3,000 | ||
Proceeds from Issuance of Shares under Incentive and Share-based Compensation Plans, Including Stock Options | $ 131 | ||
The 2014 Plan [Member] | |||
Weighted average exercise price | |||
Vesting period (in years) | 4 years | ||
The 2014 Plan [Member] | Share options | |||
Options | |||
Outstanding at beginning of the period (in shares) | 4,974,030 | ||
Granted (in shares) | 930,900 | ||
Forfeited (in shares) | (71,437) | ||
Expired (in shares) | (11,584) | ||
Outstanding at end of the period (in shares) | 5,821,909 | 4,974,030 | |
Thereof, fully vested and exercisable at end of period (in shares) | 3,054,242 | ||
Thereof, outstanding and expected to vest after at end of period (in shares) | 2,767,667 | 2,098,557 | |
Weighted average exercise price | |||
Outstanding at beginning of the period (in dollars per share) | $ 23.25 | ||
Granted (in dollars per share) | 5.59 | ||
Forfeited (in dollars per share) | 21.26 | ||
Expired (in dollars per share) | 37.61 | ||
Outstanding at end of period (in dollars per share) | 20.42 | $ 23.25 | |
Thereof, fully vested and exercisable at end of period (in dollars per share) | 25.70 | ||
Outstanding and expected to vest after at end of the period (in dollars per share) | $ 14.60 | $ 23.38 | |
Weighted-average assumptions used to estimate fair value of share options granted during year | |||
Expected volatility (as a percent) | 70% | 70% | |
Expected terms (in years) | 10 years | 10 years | |
Risk free interest rate (as a percent) | 4.32% | 4.10% | |
Expected dividend (as a percent) | 0% | 0% | |
The 2014 Plan [Member] | Non-executive directors | |||
Weighted average exercise price | |||
Vesting period (in years) | 1 year | ||
The 2014 Plan [Member] | One year from grant date | |||
Weighted average exercise price | |||
Vesting percentage per year | 25% | ||
Vesting period (in years) | 1 year |
Share-based compensation - RSU
Share-based compensation - RSU activity (Details) - The 2014 Plan [Member] $ / shares in Units, $ in Millions | 3 Months Ended |
Mar. 31, 2024 USD ($) $ / shares shares | |
Other disclosure | |
Vesting period (in years) | 4 years |
Non-executive directors | |
Other disclosure | |
Vesting period (in years) | 1 year |
Restricted share units ("RSUs") | |
Number of shares | |
Non-vested at beginning of period (in shares) | shares | 2,264,369 |
Granted (in shares) | shares | 1,160,800 |
Vested (in shares) | shares | (659,240) |
Forfeited (in shares) | shares | (58,401) |
Non-vested at end of period (in shares) | shares | 2,707,528 |
Weighted average grant-date fair value | |
Non-vested at beginning of period (in dollars per share) | $ / shares | $ 18.07 |
Granted (in dollars per share) | $ / shares | 5.59 |
Vested (in dollars per share) | $ / shares | 20.30 |
Forfeited (in dollars per share) | $ / shares | 17.39 |
Non-vested at end of period (in dollars per share) | $ / shares | $ 12.19 |
Other disclosure | |
Total weighted average grant date fair value of RSUs granted during the period (in millions) | $ | $ 6.5 |
Restricted share units ("RSUs") | Non-executive directors | |
Other disclosure | |
Vesting period (in years) | 1 year |
Minimum | Restricted share units ("RSUs") | |
Other disclosure | |
Vesting period (in years) | 1 year |
Maximum | Restricted share units ("RSUs") | |
Other disclosure | |
Vesting period (in years) | 3 years |
Share-based compensation - PSU
Share-based compensation - PSU activity (Details) | 3 Months Ended | |
Mar. 31, 2024 Milestone $ / shares shares | Dec. 31, 2023 Milestone | |
Other disclosure | ||
Number of Milestones Achieved | Milestone | 2 | |
Number of Milestones Achievement Considered Probable | Milestone | 2 | 2 |
Minimum | ||
Other disclosure | ||
Service period | 1 year | |
Maximum | ||
Other disclosure | ||
Service period | 3 years | |
Performance Share Awards | 2014 Plan | ||
Number of shares | ||
Non-vested at beginning of period (in shares) | shares | 222,550 | |
Forfeited (in shares) | shares | (17,030) | |
Non-vested at end of period (in shares) | shares | 205,520 | |
Weighted average grant-date fair value | ||
Non-vested at beginning of period (in dollars per share) | $ / shares | $ 28.09 | |
Forfeited (in dollars per share) | $ / shares | 27.12 | |
Non-vested at end of period (in dollars per share) | $ / shares | $ 27.99 |
Share-based compensation - Empl
Share-based compensation - Employee Share Purchase Plan (Details) - Employee share purchase plan (ESPP) - Employee Stock - shares | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Jun. 30, 2018 | |
Share-based compensation | |||
Number of shares issued | 0 | 2,495 | |
Ordinary shares available for issue | 96,862 | 113,565 | 150,000 |
Income taxes - Effective Tax Ra
Income taxes - Effective Tax Rate (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Effective income tax rate | 1% | (1.50%) |
Enacted tax rate | 25.80% | |
U.S | ||
Deferred tax (expense) / benefit | $ (0.7) | |
United States and France | ||
Deferred tax (expense) / benefit | $ 1.2 |
Basic and diluted earnings pe_3
Basic and diluted earnings per share (Details) - shares | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Basic and diluted earnings per share | ||
Total anti-dilutive ordinary share equivalents | 8,738,097 | 7,923,637 |
ESPP | ||
Basic and diluted earnings per share | ||
Total anti-dilutive ordinary share equivalents | 3,140 | 801 |
Employee Stock Option | ||
Basic and diluted earnings per share | ||
Total anti-dilutive ordinary share equivalents | 5,821,909 | 5,150,690 |
Non-vested RSUs and PSUs | ||
Basic and diluted earnings per share | ||
Total anti-dilutive ordinary share equivalents | 2,913,048 | 2,772,146 |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Mar. 31, 2024 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |