What changed in this filing

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Item 9A. Controls and Procedures

2023-02-27 compared with 2022-02-25 · 324 added, 376 removed, 1074 unchanged (39% of the section changed)

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Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our chief executive officer (“CEO”, our principal executive officer) and chief financial officer (“CFO”, our principal financial officer), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”)) as of December 31, 2021.

Based on such evaluation, our CEO and CFO have concluded that as of December 31, 2022, our disclosure controls and procedures were effective.

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Other Information

Disclosure Regarding Foreign Jurisdictions that Prevent Inspections

Directors, Executive Officers and Corporate Governance

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We have audited the accompanying consolidated balance sheets of uniQure N.V.

and subsidiaries (‘the Company’) as of December 31, 2021 and 2020, the related consolidated statement of operations and comprehensive loss, shareholders’ equity, and cash flows for each of the years in the three-year period ended December 31, 2021, and the related notes (collectively, the consolidated financial statements).

and subsidiaries (the Company) as of December 31, 2022 and 2021, the related consolidated statements of operations and comprehensive loss, shareholders’ equity, and cash flows for each of the years in the three-year period ended December 31, 2022, and the related notes (collectively, the consolidated financial statements).

We also have audited the Company’s internal control over financial reporting as of December 31, 2022, based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission.

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Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.

Critical Audit Matter

The critical audit matter communicated below is a matter arising from the current period audit of the consolidated financial statements that was communicated or required to be communicated to the audit committee and that:

(1) relates to accounts or disclosures that are material to the consolidated f inancial statements and (2) involved our especially challenging, subjective, or complex judgments.

The communication of a critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.

Evaluation of the acquisition of Corlieve as a business combination

As discussed in Notes 2.3.5 and 3 to the consolidated financial statements, the Company acquired 97.7% of the outstanding ordinary shares of Corlieve for a total purchase price of EUR 65.8 million.

The Company applied the applicable accounting guidance which requires the acquirer to assess whether the acquisition should be accounted for as an asset acquisition or a business combination.

We identified the evaluation of the acquisition as a business combination to be a critical audit matter.

This evaluation required significant auditor judgement to assess whether or not substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets based on the similarities of risks associated with managing and creating outputs from the assets, including the acquired workforce and identification of intangible assets.

The following are the primary procedures we performed to address this critical audit matter:

‒ We evaluated the design and tested the operating effectiveness of certain internal controls related to the business combination process, including controls related to technical accounting review and over the completeness of assets and liabilities acquired.

‒ We read the sale and purchase agreement and performed inquires with management and business development personnel to understand the business rationale for acquiring Corlieve.

‒ We read the employment terms and inquired of management and business development personnel to challenge management’s judgement that the employees acquired represent an organized workforce.

‒ We involved valuation professionals with specialized skills and knowledge, who assisted us in evaluating the Company’s identification of intangible assets acquired.

Critical Audit Matters

Critical audit matters are matters arising from the current period audit of the consolidated financial statements that were communicated or required to be communicated to the audit committee and that:

(1) relate to accounts or disclosures that are material to the consolidated f inancial statements and (2) involved our especially challenging, subjective, or complex judgments.

We determined that there are no critical audit matters.

/s/ KPMG Accountants N.V.

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Cash and cash equivalents

Current investment securities

Accounts receivable and contract asset

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Property, plant and equipment, net

Non-current investment securities

Operating lease right-of-use assets

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Accrued expenses and other current liabilities

Current portion of contingent consideration

Current portion of operating lease liabilities

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Operating lease liabilities, net of current portion

Contingent consideration

Contingent consideration, net of current portion

Deferred tax liability, net

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Ordinary shares, € 0.05 par value:

80,000,000 shares authorized as of December 31, 2021 and 60,000,000 shares authorized as of December 31, 2020 and 46,298,635 and 44,777,799 ordinary shares issued and outstanding as of December 31, 2021 and December 31, 2020, respectively

80,000,000 shares authorized as of December 31, 2022 and December 31, 2021 and 46,968,032 and 46,298,635 ordinary shares issued and outstanding as of December 31, 2022 and December 31, 2021, respectively

Additional paid-in-capital

Accumulated other comprehensive (loss) / income

Accumulated other comprehensive loss

Accumulated deficit

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License revenues

License revenues from related party

License revenues from related parties

Contract manufacturing revenues

Collaboration revenues

Collaboration revenues from related party

Collaboration revenues from related parties

Total revenues

Operating expenses:

Cost of contract revenues

Cost of license revenues

Cost of contract manufacturing revenues

Research and development expenses

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Other expense

Income / (loss) from operations

(Loss) / income from operations

Interest income

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Foreign currency gains / (losses), net

Other non-operating (losses) / gains, net

Income / (loss) before income tax (expense) / benefit

Income tax (expense) / benefit

Net income / (loss)

Other comprehensive (loss) / gain:

Other non-operating gains / (losses), net

(Loss) / income before income tax benefit / (expense)

Income tax benefit / (expense)

Net (loss) / income

Other comprehensive loss:

Foreign currency translation adjustments

Total comprehensive gain / (loss)

Total comprehensive (loss) / gain

Earnings per ordinary share - basic

Basic net income / (loss) per ordinary share

Basic net (loss) / income per ordinary share

Earnings per ordinary share - diluted

Diluted net income / (loss) per ordinary share

Diluted net (loss) / income per ordinary share

Weighted average shares - basic

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shareholders’

(in thousands, except share data)

income / (loss)

(in thousands, except share and per share amounts)

Balance at December 31, 2019

Loss for the period

Other comprehensive loss

Follow-on public offering

Hercules warrants exercise

Other comprehensive income

Exercises of share options

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Balance at December 31, 2020

Loss for the period

Other comprehensive income

Exercise of share options

Income for the period

Other comprehensive loss

Issuance of ordinary shares

Income tax benefit of past share issuance cost

Exercises of share options

Restricted and performance share units distributed during the period

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Balance at December 31, 2021

Income for the period

Loss for the period

Other comprehensive loss

Issuance of ordinary shares

Income tax benefit of past share issuance cost

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Cash flows from operating activities

Net income / (loss)

Adjustments to reconcile net income / (loss) to net cash generated from / (used in) operating activities:

Net (loss) / income

Adjustments to reconcile net (loss) / income to net cash (used in) / generated from operating activities:

Depreciation and amortization expense

Share-based compensation expense

Deferred tax expense / (income)

Changes in fair value of contingent consideration and derivative financial instruments

Deferred tax (income) / expense

Changes in fair value of contingent consideration and derivative financial instrument, net

Unrealized foreign exchange (gains) / losses, net

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Changes in operating assets and liabilities:

Contract asset related to CSL Behring milestone payments

Accounts receivable, prepaid expenses, and other current assets and receivables

Contract asset related to CSL Behring milestone payments

Accounts payable

Accrued expenses, other liabilities, and operating leases

Net cash generated from / (used in) operating activities

Net cash (used in) / generated from operating activities

Cash flows from investing activities

Investment in investment securities

Purchases of property, plant, and equipment

Acquisition of Corlieve, net of cash acquired

Purchases of intangible assets

Purchases of property, plant, and equipment

Net cash used in investing activities

Cash flows from financing activities

Proceeds from issuance of ordinary shares related to employee stock option and purchase plans

Proceeds from loan increment, net of debt issuance costs

Proceeds from public offering of shares, net of issuance costs

Proceeds from issuance of ordinary shares

Proceeds from issuance of ordinary shares related to employee stock option and purchase plans

Repayment of debt acquired through acquisition of Corlieve

Share issuance costs from issuance of ordinary shares

Proceeds from exercise of warrants

Repayment of debt acquired through acquisition of Corlieve

Net cash generated from financing activities

Currency effect on cash, cash equivalents and restricted cash

Net increase / (decrease) in cash, cash equivalents and restricted cash

Net (decrease) / increase in cash, cash equivalents and restricted cash

Cash, cash equivalents and restricted cash at beginning of period

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Cash paid for interest

Non-cash increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment

Non-cash (decrease) / increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment

The accompanying notes are an integral part of these consolidated financial statements.

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The consolidated financial statements have been prepared under the historical cost convention, except for derivative financial instruments and contingent consideration, which are recorded at fair value through profit or loss.

The consolidated financial statements are presented in U.S.

dollars, except where otherwise indicated.

The consolidated financial statements are presented in United States (“U.S.”) dollars ($), except where otherwise indicated.

Transactions denominated in currencies other than U.S.

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GAAP and Securities and Exchange Commission (“SEC”) rules and regulations, 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 consolidated financial statements and reported amounts of revenues and expenses during the reporting period.

Estimates and assumptions are primarily made in relation to the treatment of the share and purchase agreement (“SPA”) entered into on June 21, 2021 to acquire all of the outstanding ordinary shares of Corlieve Therapeutics SAS (“Corlieve”), a privately held French gene therapy company (“Corlieve Transaction”), the treatment of the commercialization and license agreement entered into (“CSL Behring Agreement”) between the Company and CSL Behring LLC (“CSL Behring”), the assessment of a valuation allowance on the Company’s deferred tax assets in the Netherlands and the U.S., and the December 1, 2020, amendment (“amended BMS CLA”) of the 2015 collaboration and license agreement (“BMS CLA”) between the Company and Bristol-Myers Squibb (“BMS”).

Estimates and assumptions are primarily made in relation to contingent consideration related to the acquisition of Corlieve Therapeutics SAS (“Corlieve”), the treatment of revenue to be recognized under the commercialization and license agreement entered into (“CSL Behring Agreement”) between the Company and CSL Behring LLC (“CSL Behring”), and the assessment of a valuation allowance on the Company’s deferred tax assets in the Netherlands and the U.S.

If actual results differ from the Company’s estimates, or to the extent these estimates are adjusted in future periods, the Company’s results of operations could either benefit from, or be adversely affected by, any such change in estimate.

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The functional currency of uniQure Inc.

dollar ($) and the functional currency of Corlieve AG is the Swiss Franc.

dollar ($) and the functional currency of Corlieve AG is the Swiss Franc (CHF).

The consolidated financial statements are presented in U.S.

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2.3.5 Corlieve transaction

On June 21, 2021, we entered into a share and purchase agreement (“SPA”) to acquire all of outstanding ordinary shares of Corlieve, a privately held French gene therapy company (“Corlieve Transaction”).

On July 30, 2021 (“Acquisition Date”), the Company acquired Corlieve.

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Goodwill is not amortized but is evaluated for impairment on an annual basis and between annual tests if we become aware of any events occurring or changes in circumstances that would more likely than not reduce the fair value of the reporting unit below its carrying amount.

As of December 31, 2021, the Company has not recognized any impairment charges related to goodwill.

As of December 31, 2022 and 2021, the Company has not recognized any impairment charges related to goodwill.

Refer to Note 3 “ Corlieve transaction ” for further detail.

Acquired research and development

The Company identified various licenses that combined with the results of the research and development activities conducted in relation to AMT-260 since incorporation of Corlieve in 2019 constitute an In-process research and development intangible asset (“IPR&D Intangible Asset”).

The Company identified various licenses that combined with the results of the research and development activities conducted in relation to its target candidate for the treatment of temporal lobe epilepsy (“AMT-260”) since incorporation of Corlieve in 2019 constitute an In-process research and development intangible asset (“IPR&D Intangible Asset”).

The IPR&D Intangible Asset is considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts and is not amortized.

If and when development is completed, which generally occurs when regulatory approval to market a product is obtained, the associated asset would be deemed finite-lived and would then be amortized based on its respective useful life at that point in time.

As of December 31, 2021, the Company has not recognized any impairment charges related to the IPR&D Intangible Asset.

As of December 31, 2022 and 2021, the Company has not recognized any impairment charges related to the IPR&D Intangible Asset.

In case of abandonment, the IPR&D Intangible Asset will be written-off.

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The Company views its operations and manages its business as one operating segment, which comprises the discovery, development, and commercialization of innovative gene therapies.

2.3.8 Net income / (loss) per share

2.3.8 Net (loss) / income per share

The Company follows the provisions of ASC 260, Earnings Per Share .

In accordance with these provisions, net income / (loss) per share is calculated by dividing net income / (loss) by the weighted average number of ordinary shares outstanding during the period.

Diluted net income / (loss) per share reflects the dilution that would occur if share options or warrants to issue ordinary shares were exercised, performance or restricted share units were distributed, or shares under the employee share purchase plan were issued.

In accordance with these provisions, net (loss) / income per share is calculated by dividing net (loss) / income by the weighted average number of ordinary shares outstanding during the period.

Diluted net (loss) / income per share reflects the dilution that would occur if share options or warrants to issue ordinary shares were exercised, performance or restricted share units were distributed, or shares under the employee share purchase plan were issued.

However, potential ordinary shares are excluded if their effect is anti-dilutive.

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Fair value is determined through various valuation techniques, including discounted cash flow models, quoted market values, and third-party independent appraisals, as considered necessary.

The Company performs the same quantitative analysis discussed above for long-lived assets and finite-lived intangible assets

Refer to Note 2.3.5 “ Corlieve transaction ” for information on impairment testing related to goodwill and acquired research and development intangible assets.

2.3.10 Investment securities

Investment securities consist of sovereign debt with residual maturities of less than 12 months (presented as current) and beyond (presented as non-current).

The Company classifies these securities as held-to-maturity.

Held-to-maturity securities are those securities in which the Company has the ability and intent to hold the security until maturity.

Held-to-maturity securities are recorded at amortized cost, adjusted for the amortization or accretion of premiums or discounts.

Premiums and discounts are amortized or accreted over the term of the related held-to-maturity security as an adjustment to yield using the effective interest rate method.

Investments securities with original maturities of less than three months when purchased are presented within cash and cash equivalents (December 13, 2022:

$ 21.2 million, December 31, 2021:

A decline in the market value of any investment security below cost that is deemed to be other than temporary results in a reduction in the carrying amount to fair value.

The impairment is charged to operations and a new cost base for the security is established.

Other-than-temporary impairment charges are included in interest and other income (expense), net.

Interest income is recognized when earned.

Refer to Note 5 “ Investment securities ” for further information.

2.3.11 Accounts receivable

Accounts receivables include amounts due from services provided to the Company’s licensing and collaboration partners as well as unconditional rights to consideration from its licensing and collaboration partners.

2.3.12 Inventories

The Company started producing commercial materials in April 2022 to supply CSL Behring with the Product in accordance with the June 2020 Development and Commercial Supply Agreement between the Company and CSL Behring.

From this date onwards, the Company presents the costs associated with the aforementioned activities as cost of contract manufacturing.

Refer to Note 4, “Collaboration arrangements and concentration of credit risk” for further detail.

Per ASC 330, Inventory, inventory is stated at the lower of cost or estimated net realizable value, on a first-in, first-out basis.

The Company capitalizes raw materials to the extent these can be used in the manufacturing of the Product.

The Company uses standard costs, approximating average costs to determine its cost basis for work in progress and finished goods.

The Company’s assessment of recoverability value requires the use of estimates regarding the net realizable value of its inventory balances, including an assessment of excess or obsolete inventory.

As applicable, write-downs resulting from adjustments to net realizable value will be recorded to cost of contract manufacturing.

2.3.13 Prepaid expenses

Prepaid expenses are amounts paid in the period, for which the benefit has not been realized, and include payments made for insurance and research and clinical contracts.

The related expense will be recognized in the subsequent period as incurred.

2.3.14 Other (non) current assets

Deposits paid are either presented as other current assets or as other non-current assets based on duration of the underlying contractual arrangement.

Deposits are classified as restricted cash and primarily relate to facility leases.

Contract assets are presented in current assets or as non-current assets based on the timing of the right to consideration.

2.3.15 Property, plant, and equipment

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2.3.16 Leases

The Company adopted ASC 842, Leases using the modified retrospective approach with an effective date as of the beginning of the Company’s fiscal year, January 1, 2019, to operating leases that existed on that date.

The Company measured lease liabilities at the present value of the future lease payments as of January 1, 2019.

The Company used an incremental borrowing rate to discount the lease payments.

The Company derived the discount rate, adjusted for differences such as in the term and payment patterns, from the Company’s loan from Hercules Technology Growth Capital, Inc (“Hercules Capital”), which was refinanced immediately prior to the January 1, 2019 adoption date in December 2018.

The right-of-use asset is valued at the amount of the lease liability reduced by the remaining December 31, 2018 balance of lease incentives received.

The lease liability is subsequently measured at the present value of the future lease payments as of the reporting date with a corresponding adjustment to the right-to-use asset.

Absent a lease modification, the Company will continue to utilize the January 1, 2019, incremental borrowing rate.

For leases recognized after the adoption date, the Company determines if an arrangement is a lease at inception.

The Company records leases in accordance with ASC 842, Leases and determines if an arrangement is a lease at inception.

Operating lease right-of-use assets and lease liabilities are initially recognized based on the present value of future minimum lease payments over the lease term at commencement date calculated using an incremental borrowing rate applicable to the lease asset, unless the implicit rate is readily available.

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The Company presents lease payments within cash flows from operations within the Consolidated statements of cash flows.

2.3.12 Other (non) current assets

Deposits paid are either presented as other current assets or as other non-current assets based on duration of the underlying contractual arrangement.

Deposits are classified as restricted cash and primarily relate to facility leases.

Contract assets are presented in other current assets or as other non-current assets based on the timing of the right to consideration.

2.3.13 Prepaid expenses

Prepaid expenses are amounts paid in the period, for which the benefit has not been realized, and include payments made for insurance and research and clinical contracts.

The related expense will be recognized in the subsequent period as incurred.

2.3.14 Accounts receivable

2.3.17 Accounts payable and accrued expenses

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Accrued expenses are recognized for goods or services that have been acquired in the ordinary course of business.

Contract liabilities are presented in accounts payable and accrued expenses.

Contract liabilities, if any, are presented in accrued expenses.

2.3.18 Long-term debt

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The Company concluded that CSL Behring is a customer in accordance with ASC 606, Revenue from Contracts with Customers and identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License Sale”);

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

(i) Sale of the exclusive global rights to etranacogene dezaparvovec, our investigational gene therapy for patients with hemophilia B (the “Product”) (“License Sale”);

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of the Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

These performance obligations are considered distinct from one another, as CSL Behring can benefit from the identified service either on its own or together with other resources that are readily available to CSL Behring, and as the performance obligations are separately identifiable from other performance obligations in the CSL Behring Agreement.

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Bristol-Myers Squib collaboration

The Company initially entered into collaboration, research, and license agreements with BMS in 2015 and amended them in 2020.

The Company evaluated the initial BMS CLA and determined that its performance obligations were as follows:

The Company initially entered into collaboration, research, and license agreements with Bristol-Myers Squibb (“BMS”) in 2015 (“BMS CLA”) and amended them in 2020 (“amended BMS CLA”).

The agreement terminated on February 21, 2023 (“Termination Date”).

The Company evaluated the initial BMS CLA and determined that its performance obligations were as follows as of the amended BMS CLA:

● Providing pre-clinical research activities (“Collaboration Revenue”);

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As further discussed in Note 4, “ Collaboration arrangements and concentration of credit risk ”, as a result of the December 2020 amended BMS CLA, the Company’s performance obligation related to License Revenues was materially completed as of the date of the amendment effective date of December 1, 2020.

The Company may still be required to provide pre-clinical research activities or clinical and commercial manufacturing services when BMS exercises its options for those services.

License Revenue

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Collaboration and Manufacturing Revenue

The Company recognizes Collaboration Revenues associated with optional work orders it receives from BMS to provide analytical development and process development activities that are reimbursable by BMS in accordance with the BMS CLA as well as the amended BMS CLA.

BMS and the Company entered into a Master Clinical Supply Agreement in April 2017 for the Company to supply gene therapy products during the clinical phase as well as into a binding term sheet to supply gene therapy products during the commercial phase to BMS.

In December 2020, BMS and the Company also entered into a Research Supply Agreement.

Revenues from product sales will be recognized when earned.

The Company will provide these services as it receives optional work orders from BMS in relation to such services.

The Company recognized Collaboration Revenue associated with optional work orders it received from BMS to provide analytical development and process development activities that were reimbursable by BMS in accordance with the BMS CLA as well as the amended BMS CLA.

2.3.22 Other income, other expense

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2.3.25 Recently Adopted Accounting Pronouncements

Recent Accounting Pronouncements Not Yet Effective

Government Assistance

In November 2018, the FASB issued ASU 2021-10, Government Assistance (Topic 832) which discusses the requirements for disclosures related to transactions with a government.

In November 2018, the FASB issued ASU 2021-10, Government Assistance (Topic 832) which discussed the requirements for disclosures, to be applied prospectively or retrospectively, related to transactions with a government.

ASU 2021-10 is effective for fiscal years beginning after December 15, 2021.

The new disclosure requirements will require disclosures around 1) information about the nature of the transactions and the related accounting policy used to account for the transactions, 2) the line items on the balance sheet and income statement that are affected by the transactions, and the amounts applicable to each financial statement line item, and 3) significant terms and conditions of the transactions, including commitments and contingencies.

An entity should apply the updates prospectively or retrospectively.

The Company currently includes information on government grants and does not expect these amendments to have a material impact on the Company’s consolidated financial statements.

The new disclosure requirements required disclosures around 1) information about the nature of the transactions and the related accounting policy used to account for the transactions, 2) the line items on the balance sheet and income statement that are affected by the transactions, and the amounts applicable to each financial statement line item, and 3) significant terms and conditions of the transactions, including commitments and contingencies.

The Company currently includes information on government grants and the adoption of ASU 2021-10 on January 1, 2022 has not had a material impact on the Company’s consolidated financial statements.

Recent Accounting Pronouncements Not Yet Effective

Corlieve transaction

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At the Acquisition Date, Corlieve and its Swiss subsidiary, Corlieve Therapeutics AG, employed seven employees.

Corlieve’s result for the full year 2021 was a $ 7.3 million loss.

The result included in the Company’s consolidated results for the year ended December 31, 2021 is a $ 4.1 million loss.

Corlieve’s result for the full year 2021 was a $ 7.3 million loss of which $ 4.1 million was included in the Company’s consolidated results.

The Company evaluated the Corlieve transaction as to whether or not the transaction should be accounted for as a business combination or asset acquisition by first applying a screen test to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets.

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The excess of the fair value of the consideration transferred over the fair value of the net assets acquired was recorded as goodwill.

The following table summarizes the fair values assigned to assets acquired and the liabilities assumed by the Company, along with the resulting goodwill, as of the Acquisition Date:

(in thousands)

Consideration

Contingent consideration

Liability related to Mandatorily Redeemable Shares (see below)

Fair value of total consideration

Recognized amounts of identifiable assets acquired and liabilities assumed

Current assets including € 2.8 million of cash

Property, plant and equipment

Identifiable intangible asset

Current liabilities

Deferred tax liability, net

Other non-current liabilities

Fair value of net assets acquired

Consideration

On the Acquisition Date, the Company acquired 97.7 % of the outstanding ordinary shares of Corlieve in return for EUR 44.9 million ($ 53.3 million as of the Acquisition Date).

As is contractually required the Company acquired the remaining outstanding ordinary shares on February 9, 2022 following the expiration of a minimum holding period.

The Company recorded a liability related to these Mandatorily Redeemable Shares for an amount of EUR 0.7 million ($ 0.9 million) as of the Acquisition Date.

The Company financed the Corlieve Transaction from its cash on hand.

The Company acquired the remaining outstanding ordinary shares in February, July and September 2022 for a total of EUR 1.8 million ($ 1.9 million).

In addition to the payments to acquire 100 % of the outstanding ordinary shares, Corlieve’s former shareholders are eligible to receive up to EUR 40.0 million ($ 42.8 million as of December 31, 2022) upon achievement of certain development milestones through Phase I/II and EUR 160.0 million ($ 171.3 million as of December 31, 2022) upon achievement of certain milestones associated with Phase III development and obtaining approval to commercialize Corlieve’s target candidate for the treatment of temporal lobe epilepsy (“AMT-260” or “TLE”) in the United States of America and the European Union.

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As of the Acquisition Date, the Company recorded EUR 20.2 million ($ 24.0 million) as a contingent liability (presented as “Non-current liability”) for the fair value of these milestone payments.

The fair value of the contingent liability as of December 31, 2021 amounted to EUR 26.0 million ($ 29.5 million).

Changes in fair value of the contingent liability are recognized within research and development expenses in the consolidated statements of operations.

Identified intangible assets

The Company determined the fair value of the IPR&D Intangible Asset using a present value model based on expected cash flows.

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On May 6, 2021, a day after the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, the CSL Behring Agreement became fully effective (“Closing”).

Pursuant to the CSL Behring Agreement, the Company received a $ 450.0 million upfront cash payment and $ 12.4 million in other payments related to the Closing and the transfer of the license.

The Company is eligible to receive up to $ 1.6 billion in additional payments based on the achievement of regulatory and commercial milestones.

The CSL Behring Agreement also provides that the Company will be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of net sales of the Product based on sales thresholds.

On the Signing Date, the Company and CSL Behring entered into a development and commercial supply agreement, pursuant to which, among other things, the Company will supply the Product to CSL Behring at an agreed-upon price commensurate with the SSP.

The Company will be responsible for supplying development and commercial Product until such time that these capabilities may be transferred to CSL Behring or a designated contract manufacturing organization.

The Company will be completing the HOPE-B clinical trial and the validation of the manufacturing process on behalf of CSL Behring, as well as provide further development services if requested by CSL Behring.

Activities related to on-demand development services as well as activities related to the completing the HOPE-B clinical trial will be reimbursed by CSL Behring at an agreed full-time-employee rate (“FTE-rate”) and CSL Behring will also reimburse agreed third-party expenses incurred in relation to performing these activities.

The validation of the manufacturing process as well as Manufacturing Development will be reimbursed through a future milestone payment.

If completed after certain contractually agreed upon dates, the milestone payment will be reduced in accordance with a pre-specified mechanism.

The Company concluded that CSL Behring is a customer in accordance with Topic 606.

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The Additional Covenants are not considered distinct from the performance obligation to sell the license to CSL Behring as CSL Behring could not benefit from the Additional Covenants on their own, or have these activities be performed with readily available resources.

The Company determined that the fixed upfront payment of $ 450.0 million and the $ 12.4 million that the Company received in relation to the Additional Covenants should be allocated to the License Sale.

The Company determined that the fixed upfront payment of $ 450.0 million and the $ 12.4 million that the Company received in May 2021 in relation to the Additional Covenants should be allocated to the License Sale.

In addition, the Company concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well.

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The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.

Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments are constrained as of Closing.

Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments were constrained as of Closing.

The Company determined that it would recognize revenue related to these payments only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter.

Similarly, the Company will record expenses related to its existing license and other agreements as well as its financial advisor for a high single digit percentage of any such revenue recognized associated to meeting a milestone.

The Company determined that achievement of a total of $ 55.0 million of milestone payments related to the submissions of a biologics license application (“BLA”) and market authorization application (“MAA”) was probable as of February 25, 2022, the time of filing the 2021 financial statements, and hence recorded these as license revenue in the year ended December 31, 2021.

In March and April 2022, the global regulatory submissions were submitted and the Company received the $ 55.0 million owed to it from CSL Behring.

The Company recorded $ 100.0 million in variable milestone revenue related to a first sale of HEMGENIX™ in the U.S.

during the year ended December 31, 2022 as the Company considers the occurrence of this event to be probable following the November 2022 BLA approval of HEMGENIX™.

Despite the approval of the MAA for HEMGENIX™ by the Europeans Medicines Agency (“EMA”) in February 2023, the Company did not record the $ 75.0 million variable milestone payment related to a first sale of HEMGENIX™ in the one of five major European countries, namely France, Germany, Italy, Spain, and the United Kingdom, as license revenue in the year ended December 31, 2022.

The Company considers that the milestone is only owed if achieved prior to July 2, 2023, which is contingent on factors outside the Company’s control and thus the Company determined that occurrence was not probable as of December 31, 2022.

The Company is also eligible to receive up to $ 1.3 billion in additional payments based on the achievement of commercial milestones.

The Company will include payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of Product once these have been earned.

The Company determined that achievement of a total of $ 55.0 million of milestone payments related to the submissions of a biologics license application (“BLA”) and market authorization application (“MAA”) is probable as of the time of filing these financial statements and hence recorded these as license revenue in the year ended December 31, 2021.

In making the determination, the Company considered that after Closing, it believes to successfully have completed the validation of its manufacturing process for the Product in December 2021 and that CSL Behring announced the accomplishment of the primary clinical endpoint for the Product in December 2021.

The Company recognized $ 517.4 million of revenues related to the License Sale in the year ended December 31, 2021.

The Company determined that the variable milestone payment related to Manufacturing Development should be allocated to the Manufacturing Development performance obligation.

The Company concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin.

The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan.

The variable consideration will be reduced if the Company does not complete the development by pre-agreed dates.

The Company recognized $ 100.0 million and $ 517.4 million of revenues related to the License Sale in the years ended December 31, 2022 and December 31, 2021, respectively ( nil in 2020).

The Company records expenses related to its existing license and other agreements as well as its financial advisor for a high single digit percentage of any such revenue recognized associated to meeting a milestone.

Manufacturing Development

The Company determined that the $ 50.0 million variable milestone payment related to Manufacturing Development should be allocated to the Manufacturing Development performance obligation.

The Company concluded that this milestone payment represents the stand-alone selling price (“SSP”) of the services based on the estimated cost of providing the services including a reasonable margin.

Manufacturing Development includes providing information regarding a next generation manufacturing process of the Product to CSL Behring.

CSL Behring did not request such services during the year ended December 31, 2022.

The variable consideration will be reduced if the Company does not complete the development by pre-agreed dates following BLA, respectively MAA approval.

The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.

Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes.

Since the Company cannot control the timing or outcome of any regulatory decisions, the Company concluded that it would recognize revenue related to this payment when it becomes probable that the milestone has been achieved.

The Company has not recognized any revenue related to Manufacturing Development.

The Company recognized $ 2.4 million of collaboration revenue in the year ended December 31, 2021, respectively, compared to nil in the same periods in 2020 and 2019.

The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring.

CSL Behring may request additional development services or request the Company to support the transfer of manufacturing to a party designated by CSL Behring.

These collaboration services will be reimbursed at the pre-agreed FTE-rate.

As of December 31, 2022, the Company has not recognized any revenue related to the Manufacturing Development milestone.

Contract manufacturing

On September 6, 2022, CSL Behring notified the Company of its intent to transfer manufacturing technology related to the Product in the coming years to a third-party contract manufacturer designated by CSL Behring.

CSL Behring also requested that the Company continue to serve as a manufacturer of the Product after the Company completes the technology transfer to a third party.

The Company and CSL Behring are in the process of negotiating the terms of the transfer of manufacturing responsibility pursuant to the CSL Behring Agreement.

The Company generated $ 1.7 million contract manufacturing revenue from sales to CSL Behring.

The Company recognizes contract manufacturing revenue when CSL Behring obtains control of the Product.

The Company incurred $ 2.1 million of cost in relation to its contract manufacturing activities during the year ended December 31, 2022.

Collaboration services

Following Closing, the Company was facilitating the completion of the HOPE-B clinical trial on behalf of CSL Behring until CSL Behring took over the execution of the clinical trials in December 2022.

Activities related to on-demand development services as well as activities related to the completing the HOPE-B clinical trial are reimbursed by CSL Behring at an agreed full-time-employee rate (“FTE-rate”) and CSL Behring also reimburses agreed third-party expenses incurred in relation to performing these activities.

The Company concluded that these rights at Closing do not represent material rights.

The Company incurred $ 2.1 million of expenses for obligations related to the CSL Behring Agreement that had not been satisfied as of December 31, 2020.

The Company capitalized these expenses as contract fulfillment costs (presented within Other current assets).

As of December 31, 2020, the Company also recognized a $ 2.1 million receivable (presented within Accounts receivable) from CSL Behring for expenses for which it has a right of reimbursement as well as a contract liability (presented within Accrued expenses and other current liabilities) for the same amount.

In accordance with ASC 606 the Company could not recognize any license revenue related to the CSL Behring Agreement in the period ended December 31, 2020.

Following the Closing, the Company collected the $ 2.1 million of accounts receivable related to reimbursable contract fulfillment costs that was outstanding as of December 31, 2020 .

As of December 31, 2021, the Company has recorded accounts receivable of $ 2.9 million from CSL Behring related to clinical development services as well as a contract asset of $ 55.0 million related to BLA and MAA submission milestone payments considered probable.

The Company recognized $ 3.0 million of collaboration revenue in the year ended December 31, 2022, compared to $ 2.4 million and nil in the same periods in 2021 and 2020.

Accounts r eceivable and c ontract 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 expected to be received from CSL Behring following the first sale of HEMGENIX™ in the U.S in 2023.

As of December 31, 2021, the Company recorded accounts receivable of $ 2.9 million from CSL Behring related to collaboration services as well as a contract asset of $ 55.0 million associated with milestone payments due upon CSL Behring’s global regulatory submissions for HEMGENIX™.

Bristol-Myers Squibb collaboration

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The initial four-year research term under the collaboration terminated on May 21, 2019.

During the initial research term of the BMS CLA, the Company supported BMS in discovery, non-clinical, analytical and process development efforts in respect of the Collaboration Targets.

For any Collaboration Targets that may be advanced, the Company will be responsible for manufacturing of clinical and commercial supplies.

BMS reimbursed the Company for all its research and development costs in support of the collaboration, and will lead development, regulatory and commercial activities for any Collaboration Targets that may be advanced.

The BMS CLA initially provided that the Company and BMS could potentially have collaborated on up to ten Collaboration Targets in total.

2020 amendment

On December 1, 2020, the Company and BMS entered into the amended BMS CLA.

Under the amended BMS CLA, BMS is limited to four Collaboration Targets.

Under the amended BMS CLA, BMS was limited to four Collaboration Targets.

For a period of one-year from the effective date of the amended BMS CLA, BMS was able to replace up to two of the four active Collaboration Targets with two new targets in the field of cardiovascular disease.

The Company continues to be eligible to receive research, development, and regulatory milestone payments of up to $ 217.0 million for each Collaboration Target, if defined milestones are achieved.

Since the December 2020 amendment, BMS is no longer entitled to designate the fifth to tenth Collaboration Targets and as such the Company’s remaining obligations under the amended BMS CLA are substantially reduced.

The Company is also no longer entitled to receive up to an aggregate $ 16.5 million in target designation payments for the research, development and regulatory milestone payments associated with the fifth to tenth Collaboration Targets.

For as long as any of the four Collaboration Targets are being advanced, BMS may place a purchase order to be supplied with research, clinical and commercial supplies.

Subject to the terms of the amended BMS CLA, BMS has the right to terminate the research, clinical and commercial supply relationships, and has certain remedies for failures of supply, up to and including technology transfer for any such failure that otherwise cannot be reasonably resolved.

Both BMS and the Company may agree to a technology transfer of manufacturing capabilities pursuant to the terms of the amended BMS CLA.

The amended BMS CLA does not extend the initial four-year research term that expired in May 2019.

BMS may place purchase orders to provide limited services primarily related to analytical and development efforts in respect of the four Collaboration Targets.

BMS may request such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) November 30, 2023, if no replacement targets are designated.

BMS continues to reimburse the Company for these services.

During the year ended December 31, 2020, the Company evaluated the impact of the amendment of the BMS CLA had in relation to its performance obligation related to License Revenue.

The Company did not identify any new distinct performance obligations and determined the amended BMS CLA did not represent a separate contract in accordance with ASC 606.

The Company evaluated the effect the modification had on its measure of progress towards the completion of its performance obligation related to License Revenue and determined that its remaining performance obligation under the amended BMS CLA was immaterial and recognized the remaining balance of unrecognized License Revenue as of November 30, 2020.

Services to BMS are rendered by the Dutch operating entity.

The Company continued to be eligible to receive research, development, and regulatory milestone payments of up to $ 217.0 million for each Collaboration Target, if defined milestones had been achieved.

Since the December 2020 amendment, BMS no longer was entitled to designate a fifth to tenth Collaboration Targets and as such the Company’s remaining obligations under the amended BMS CLA were substantially reduced.

The Company also no longer was entitled to receive up to an aggregate $ 16.5 million in target designation payments for the research, development and regulatory milestone payments associated with the fifth to tenth Collaboration Targets.

For as long as any of the four Collaboration Targets were being advanced, BMS might have placed a purchase order to be supplied with research, clinical and commercial supplies.

The amended BMS CLA did not extend the initial four-year research term that expired in May 2019.

BMS could place purchase orders to be provided with limited services primarily related to analytical and development efforts in respect of the four Collaboration Targets.

BMS could have requested such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) November 30, 2023.

BMS continued to reimburse the Company for these services.

2022 Termination

On November 21, 2022, the Company received written notice that BMS is terminating the BMS CLA as amended effective February 21, 2023.

Services to BMS were rendered by the Dutch operating entity.

Total collaboration and license revenue generated with BMS are as follows (presented as revenue from a related party up until the effective date of the amended BMS CLA and presented as revenue after the effective date):

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Bristol Myers Squibb

License Revenue

The Company recognized no License Revenue for the year ended December 31, 2022 (December 31, 2021:

nil million, December 31, 2020:

$ 33.0 million).

In 2015 the Company received $ 75.1 million of payments that it allocated to License Revenue.

The Company recognized License Revenue over the expected performance period based on its measure of progress towards the completion of certain activities related to its services.

The Company did not identify any new distinct performance obligations and determined the amended BMS CLA did not represent a separate contract in accordance with ASC 606.

The Company included variable consideration related to any research, development, and regulatory milestone payments, in the transaction price once it considered it probable that including these payments in the transaction price would not result in the reversal of cumulative revenue recognized.

Due to the significant uncertainty surrounding the development of gene-therapy product candidates and the dependence on BMS’s performance and decisions, the Company with the exception of a $ 4.4 million research milestone payment received and recorded as License Revenue in December 2020 did not consider this probable as of December 31, 2021.

No variable consideration became due prior to the termination of the amended BMS CLA on February 21, 2023.

Under the amended BMS CLA, the Company would have recognized License Revenue related to product sales by BMS from any of the Collaboration Targets when the sales would have occurred.

The Company was eligible to receive net sales-based milestone payments and tiered mid-single to low double-digit royalties on product sales.

Collaboration Revenue

The Company recognizes collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that are reimbursable by BMS under the BMS CLA and the amended BMS CLA as well as other related agreements.

Collaboration Revenue related to these contracted services is recognized when performance obligations are satisfied.

The Company recognized collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that were reimbursable by BMS under the BMS CLA and the amended BMS CLA as well as other related agreements.

Collaboration Revenue related to these contracted services was recognized when performance obligations were satisfied.

The Company generated $ 1.8 million collaboration revenue for the year ended December 31, 2022 (December 31, 2021:

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$ 0.2 million).

License Revenue

The Company recognized no License Revenue for the year ended December 31, 2021 (December 31, 2020:

$ 33.0 million, December 31, 2019:

$ 5.0 million).

On May 21, 2015, the Company recorded a $ 60.1 million upfront payment and in August 2015 it recorded a $ 15.0 million payment it received from BMS in relation to the designation of the second, third and fourth Collaboration Targets.

The Company recognized License Revenue over the expected performance period based on its measure of progress towards the completion of certain activities related to its services.

The Company determined such progress by comparing activities performed at the end of each reporting period with total activities expected to be performed.

The Company estimated total expected activities using several unobservable inputs, such as the probability of BMS designating additional targets, the probability of successfully completing each phase and estimated time required to provide services during the various development stages.

The estimation of total services at the end of each reporting period involves considerable judgment.

The amount of services the Company expects to provide is significantly impacted by the number of Collaboration Targets that it estimates BMS would pursue.

As a result of the December 1, 2020 amendment of the BMS CLA the Company no longer is required to potentially provide any services in relation to six additional targets that BMS might have designated.

The Company determined its remaining performance obligation is immaterial.

The Company adjusted its measure of progress towards the completion of its activities related to its services as of the December 1, 2020 modification date accordingly.

The Company recognized the remaining balance of unrecognized License Revenue as of November 30, 2020 of $ 27.8 million in profit and loss during the year ended December 31, 2020 as License Revenue from a related party.

The Company includes variable consideration related to any research, development, and regulatory milestone payments, in the transaction price once it is considered probable that including these payments in the transaction price would not result in the reversal of cumulative revenue recognized.

Due to the significant uncertainty surrounding the development of gene-therapy product candidates and the dependence on BMS’s performance and decisions, the Company does not currently consider this probable.

However, there was a milestone that was recorded as license revenue in the year ended December 31, 2020 (see below).

On December 17, 2020 BMS designated one of the four Collaboration Targets as a candidate to advance into Investigational New Drug-enabling studies (“IND-enabling studies”) entitling the Company to receive a $ 4.4 million research milestone payment.

The Company recorded the $ 4.4 million as License Revenue in the year ended December 31, 2020.

The Company recognizes License Revenue related to product sales by BMS from any of the Collaboration Targets when the sales occur.

The Company is eligible to receive net sales-based milestone payments and tiered mid-single to low double-digit royalties on product sales.

The royalty term is determined on a licensed-product-by-licensed-product and country-by-country basis and begins on the first commercial sale of a licensed product in a country and ends on the expiration of the last to expire of specified patents or regulatory exclusivity covering such licensed product in such country or, with a customary royalty reduction, ten years after the first commercial sale if there is no such exclusivity.

Investment securities

The following table summarizes the Company’s investments into sovereign debt as of December 31, 2022:

At December 31, 2022

Amortized cost, as adjusted

Gross unrealized holding gains

Gross unrealized holding losses

Estimated fair value

(in thousands)

Current investments:

Obligations of governmental agencies (held-to-maturity)

Non-current investments:

Obligations of governmental agencies (held-to-maturity)

No investments classified as held-to-maturity were purchased in the prior years.

Inputs to the fair value of the investments are considered Level 2 inputs.

The following table summarizes the inventory balances as of December 31, 2022:

(in thousands)

Raw materials

Work in progress

Finished goods

Fair value measurement and Other non-operating (losses) / gains

The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting.

The carrying amount of cash and cash equivalents, accounts receivable from collaborators, 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 Company’s only material financial assets measured at fair value using Level 1 inputs is cash and cash equivalents and restricted cash.

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 Company’s material financial assets include cash and cash equivalents, restricted cash and investment securities.

Cash and cash equivalents and restricted cash are measured at fair value using Level 1 inputs.

Restricted cash is included within “Other non-current assets” within the consolidated balance sheets.

Investment securities are measured at amortized cost.

The following table sets forth the balances and changes in fair values of liabilities that are measured at fair value using Level 3 inputs:

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Balance at December 31, 2019

Net losses recognized in profit or loss

Exercise of Hercules warrants

Currency translation effects

Balance at December 31, 2019

Net gains recognized in profit or loss

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Balance at December 31, 2021

Net losses recognized in profit or loss

Currency translation effects

Balance at December 31, 2022

Contingent consideration

The Company is required to pay up to EUR 178.8 million ($ 191.4 million at the December 31, 2022 foreign exchange rate) to the former shareholders of Corlieve upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve (refer to Note 3 “Corlieve transaction” ).

The Company recorded a liability for the fair market value of the contingent consideration of EUR 20.2 million ($ 24.0 million) at the Acquisition Date.

The fair market value was determined using unobservable initial inputs with respect to (i) the probability of achieving the relevant milestones, or POS, (ii) the estimated timing of achieving such milestones, and (iii) the interest rate used to discount the payments.

The Company determined the fair market value of the contingent consideration by calculating the probability-adjusted payments based on each milestone’s probability of achievement.

The probability-adjusted payments were then discounted to present value using a discount rate representing the Company’s credit risk.

This discount rate was determined using the effective interest rate of the Company’s existing debt facility adjusted for difference in maturity dates based on market data on effective yields for U.S.

bonds with a CCC credit rating.

The fair value of the contingent consideration as of December 31, 2022 was $ 35.3 million (2021:

$ 29.5 million) using discount rates ranging from 14.0 % to 14.4 % (December 31, 2021:

10.9 % to 11.9 %) as well as a 66.0 % (December 31, 2021:

55.0 %) likelihood of AMT-260 advancing into clinical development by no later than late 2023.

If as of December 31, 2022 the Company had assumed a 100 % likelihood of AMT-260 advancing into clinical development, then the fair value of the contingent consideration would have increased to $ 48.8 million.

If as of December 31, 2022 the Company assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would be released to income.

Changes in fair value of the contingent liability are recognized within research and development expenses in the consolidated statements of operations.

As of December 31, 2022, the Company classified $ 26.0 million of the total contingent consideration of $ 35.3 million as current liabilities.

The balance sheet classification between current and non-current liabilities is based upon the Company’s best estimate of the timing of settlement of the remaining relevant milestones.

Derivative financial instruments

The Company issued derivative financial instruments related to its collaboration with BMS and in relation to the issuance of the Hercules loan facility.

The Company recorded the following results in other non-operating (losses) / gains related to the changes in the fair value of derivative financial instruments.

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Derivative losses

Total other non-operating losses:

Other non-operating (losses) / gains, net

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On December 1, 2020, the Company derecognized the warrants when these were terminated in accordance with the amended BMS CLA.

Pursuant to the terms of the BMS CLA the exercise price in respect of each warrant was equal to the greater of (i) the product of (A) $ 33.84 , multiplied by (B) a compounded annual growth rate of 10 % (or approximately $ 57.32 as of November 30, 2020) and (ii) the product of (A) 1.10 multiplied by (B) the weighted average volume price (“VWAP”) for the 20 trading days ending on the date that is five trading days prior to the date of a notice of exercise delivered by BMS.

The fair value of the warrants as of December 31, 2019 was $ 3.1 million.

During the year ended December 31, 2020, the Company recognized a $ 3.1 million gain in non-operating (losses) / gains (December 31, 2019:

$ 2.3 million loss) related to fair value changes of the BMS warrants.

The gain recognized in the year ended December 31, 2020 includes $ 0.8 million from the derecognition of the BMS warrants on December 1, 2020.

The Company used Monte-Carlo simulations to determine the fair market value of the BMS warrants.

The valuation model incorporated several inputs, the risk-free rate adjusted for the period affected, an expected volatility based on historical Company volatility, the expected yield on any dividends and management’s expectations on the timelines of reaching certain defined trigger events for the exercising of the warrants, as well as management’s expectations regarding the number of ordinary shares that would be issued upon exercise of the warrants.

All of these represent Level 3 inputs.

Additionally, the model assumed BMS would exercise the warrants only if it was financially rational to do so.

The warrants could only have been exercised following the occurrence of events contractually defined in the warrant agreements.

The probability of the occurrence of these events represented another significant unobservable input used in the calculation of the fair value of the warrants.

On December 1, 2020, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four Collaboration Targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $ 70.0 million, provided that (x) if $ 70.0 million is greater than five percent ( 5.0 %) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $ 70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”).

The Company has not consummated any change of control transaction as of December 31, 2021 that would obligate it to make a CoC-payment.

The Company determined that the CoC-payment should be recorded as a derivative financial liability as of December 1, 2020 and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss.

The fair market value of the derivative financial liability is materially impacted by probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment.

During the year ended December 31, 2020, the Company recognized a $ 3.1 million gain in non-operating (losses) / gains related to the fair value changes of the BMS warrants, which includes $ 0.8 million from the derecognition of the BMS warrants on December 1, 2020.

On December 1, 2020, as part of the amended BMS CLA, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four Collaboration Targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $ 70.0 million, provided that (x) if $ 70.0 million is greater than five percent ( 5.0 %) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $ 70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”).

The Company determined that the CoC-payment should be recorded as a derivative financial liability as of the December 1, 2020 initial recognition and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss.

The fair market value of the derivative financial liability is materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment.

This probability represents an unobservable input.

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The expected cash flows are materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction within the biotechnology industry.

The Company estimated this unobservable input using the best information available as of December 1, 2020, and December 31, 2020 and 2021, respectively.

The Company estimated this unobservable input using the best information available as of December 2021.

The Company obtained reasonably available market information that it believed market participants would use in determining the likelihood of the occurrence of a change-of control transaction within the biotechnology industry.

Selecting and evaluating market information involves considerable judgment and uncertainty.

Based on all such information and its judgment, the Company estimated that the fair market value of the derivative financial liability (presented within “Other non-current liabilities”) as of December 31, 2021 was $ 2.8 million (December 1, 2020 and December 31, 2020:

The Company recorded a $ 0.2 million loss in the year ended December 31, 2021 related to an increase in the fair market value of the derivative financial liability and a $ 2.6 million loss in the year ended December 31, 2020 related to the initial recognition of this derivative financial liability

The Company determined the fair market value of the derivative financial liability to be nil as of December 31, 2022 as no change of control transaction had been consummated prior to the termination of the amended BMS CLA on February 21, 2023.

The Company considered the probability of a change of control transaction occurring before the Termination Date to be remote.

This resulted in the derecognition of the derivative financial liability for the year ended December 31, 2022.

Accordingly, the Company recorded a $ 2.8 million gain within “Other non-operating (losses) / gains” in the year ended December 31, 2022

As of December 31, 2022, the Company recorded $ 0.3 million liability related to consideration for post-acquisition services, presented within Other non-current liabilities in connection with the Company’s acquisition of Corlieve (December 31, 2021:

$ 0.8 million).

The Company recorded a $ 2.6 million loss within “Other non-operating (losses) / gains” in the year ended December 31, 2020 related to the initial recognition of this derivative financial liability.

The increase of the fair market value of the derivative financial liability of $ 0.2 million in the year ended December 31, 2021 was recorded in Other non-operating (losses) / gains.

Hercules loan facility

In 2013 the Company entered into a venture debt loan facility with Hercules (see Note 10, “ Long-term debt ”) which included a warrant maturing on February 5, 2019.

The warrant was not closely related to the host contract and was accounted for separately as a derivative financial liability measured at fair value though profit or loss.

The Hercules warrants were exercised as of February 1, 2019.

The Company issued 37,175 ordinary shares at $ 34.25 following the exercise of all Hercules warrants and receipt of $ 0.5 million from Hercules.

During the years ended December 31, 2021 and 2020, respectively, the Company recognized no more gains or losses in Other non-operating (losses) / gains related to fair value changes of the Hercules warrants (December 31, 2019:

$ 0.2 million loss).

Contingent consideration

Corlieve transaction

The Company is required to pay up to EUR 178.8 million ($ 202.8 million at the December 31, 2021 foreign exchange rate) to the former shareholders of Corlieve upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve (refer to Note 3 “Corlieve transaction” ).

The Company recorded a liability for the fair market value of the contingent consideration of EUR 20.2 million ($ 24.0 million) at the Acquisition Date.

The Company determined the fair market value of the contingent consideration by calculating the probability-adjusted payments based on each milestone’s probability of achievement.

The probability-adjusted payments were then discounted to present value using a discount rate representing the Company’s credit risk.

This discount rate was determined using the effective interest rate of the Company’s existing debt facility adjusted for difference in maturity dates based on CCC yield curve.

The fair value of the contingent consideration as of December 31, 2021 was $ 29.5 million using discount rates ranging from 10.9 % to 11.9 % as well as a 55 % likelihood of AMT-260 advancing into clinical development by no later than early 2024.

The Company increased the likelihood of advancing into clinical development from 40 % to 55 % following the designation of a lead candidate in late October 2021, which resulted in EUR 5.0 million ($ 5.8 million) increase of the contingent liability.

If as of December 31, 2021 the Company had assumed a 100 % likelihood of AMT-260 advancing into clinical development, then the fair value of the contingent consideration would have increased to $ 47.0 million.

If as of December 31, 2021 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 liability are recognized within research and development expenses in the consolidated statements of operations.

As of December 31, 2021, the Company recorded $ 0.8 million related to consideration for post-acquisition services, presented within Other non-current liabilities in connection with the Company’s acquisition of Corlieve.

Investment securities

Refer to Note 5 “ Investment securities ” for the fair value of the investment securities as of December 31, 2022.

Property, plant, and equipment, net

11 unchanged sentences

$ 5.7 million).

Depreciation expense is allocated to research and development expenses to the extent it relates to the Company’s manufacturing facility and equipment and laboratory equipment.

Depreciation expense is allocated to research and development expenses and cost of contract manufacturing to the extent it relates to the Company’s manufacturing facility and equipment and laboratory equipment.

All other depreciation expenses are allocated to selling, general and administrative expense.

15 unchanged sentences

In December 2021, the Company entered into a new lease for an additional facility in Lexington, Massachusetts, United States of approximately 13,501 square feet of space.

The lease is expected to commence in the second half of 2022, is set for seven years starting from the rent commencement date and is non-cancellable.

The lease commenced in May 2022, is set for seven years and is non-cancellable.

The lease is renewable for one five-year term.

In February 2022, the Company also entered into a new lease for an additional facility in Lexington, Massachusetts, United States of approximately 12,716 square feet.

The lease commenced in November 2022 and is set for a non-cancellable period of seven years and four months .

The lease is renewable for one five-year term.

Amsterdam / The Netherlands

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Operating lease liabilities

The components of lease cost in accordance with the new lease accounting standard were as follows:

The components of lease cost were as follows:

Year ended December 31,

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Total lease cost

The table below presents the lease-related assets and liabilities recorded on the Consolidated balance sheets in accordance with the new lease accounting standard.

The table below presents the lease-related assets and liabilities recorded on the Consolidated balance sheets.

(in thousands)

13 unchanged sentences

Operating lease

1) The Company received $ 1.5 million of landlord incentive payments for the year ended December 31, 2019, which are not included in the cash paid amounts.

2) The Company capitalized $ 19.0 million of operating right-of-use assets upon adoption of ASC 842 Leases on January 1, 2019 that are not included in the movement for the year ended December 31, 2019.

Undiscounted cash flows

18 unchanged sentences

All acquired licenses are owned by uniQure biopharma B.V, a subsidiary of the Company.

The acquired licenses have a weighted average remaining life of 10.8 years as of December 31, 2021.

During the year ended December 31, 2020, the Company capitalized $ 2.2 million of expenditures related to costs incurred in relation to rights to exclusively evaluate certain technologies during a two-year period that commenced on February 1, 2020.

During the same period, the Company disposed of a number of licenses determined to have no alternative future use.

The remaining weighted average life is 11.5 years as of December 31, 2022.

(December 31 , 2021 10.8 years.)

As of December 31, 2022, the estimated future amortization expense for each of the five succeeding years and the period thereafter is as follows:

4 unchanged sentences

$ 4.6 million).

In 2020, the Company disposed of a number of licenses determined to have no alternative future use.

The impairment expense related to licenses for the year ended December 31, 2022 was $ 0.0 million (December 31, 2021:

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Accrued contract fulfillment costs and costs to obtain a contract

Contract liability (see Note 4.

"Collaboration arrangements and concentration of credit risk")

Long-term debt

3 unchanged sentences

The 2018 Amended Facility extended the loan’s maturity date from May 1, 2020 until June 1, 2023.

The interest rate is adjustable and is the greater of (i) 8.85 % and (ii) 8.85 % plus the prime rate less 5.50 % per annum.

Under the 2018 Amended Facility, the Company owes a back-end fee of 4.95 % of the outstanding debt.

In addition, in May 2020 the Company paid a back-end fee of $ 1.0 million in relation to the 2016 Amended Facility.

The interest rate was adjustable and was the greater of (i) 8.85 % and (ii) 8.85 % plus the prime rate less 5.50 % per annum.

In May 2020 the Company paid a back-end fee of $ 1.0 million in relation to the 2016 Amended Facility.

On January 29, 2021, the Company and Hercules amended the 2018 Amended Facility (“2021 Amended Facility”).

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The 2021 Restated Facility extended the loan’s maturity date from June 1, 2023 until December 1, 2025.

The interest-only period is extended from January 1, 2023 to December 1, 2024, or December 1, 2025 if, prior to June 30, 2024, either (a) the BLA for AMT-061 is approved by the FDA or (b) AMT-130 is advanced into a pivotal trial.

The interest-only period was extended from January 1, 2023 to December 1, 2024, or December 1, 2025 if, prior to June 30, 2024, either (a) the BLA for AMT-061 is approved by the U.S.

Food and Drug Administration (“FDA”) or (b) AMT-130 is advanced into a pivotal trial.

On November 22, 2022, the FDA approved the BLA for AMT-061 resulting in the extension of the interest-only period to 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 4.85 % of the outstanding debt.

The Company is required to repay the facility in equal monthly installments of principal and interest between the end of the interest-only period and the maturity date.

The Company continues to owe a $ 2.5 million back-end fee related to the 2021 Amended Facility which is due on June 1, 2023.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2021 Amended Facility was $ 101.6 million as of December 31, 2021, compared to an amortized cost of $ 35.9 million for the 2018 Amended Facility as of December 31, 2020, and is recorded net of discount and debt issuance costs.

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 $ 4.85 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 $ 103.8 million as of December 31, 2022, compared to an amortized cost of $ 101.6 million for the 2021 Restated Facility as of December 31, 2021, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the loan was $ 5.8 million in 2022 (2021:

gain of $ 3.1 million;

loss of $ 5.3 million;

gain of $ 3.1 million).

The fair value of the loan approximates its carrying amount.

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This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company.

The Company, beginning on April 1, 2023, is also required to keep a minimum of unrestricted cash of at least 50 % of the loan amount outstanding.

If, prior to June 30, 2024, either (a) the BLA for AMT-061 is approved by the FDA or (b) AMT-130 is advanced into a pivotal trial, the minimum cash covenant will be lowered to at least 30 % of the loan amount outstanding and its effectiveness will be deferred to April 1, 2024.

Following the approval of the BLA by the FDA for AMT-061 in November 2022, the Company, beginning on April 1, 2024, is required to keep a minimum of unrestricted cash of 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 $ 809.2 million with the exception of $ 103.2 million of cash and cash equivalents and other current assets held by uniQure N.V.

The Company secured the facilities by directly or indirectly pledging its total assets of $ 705.0 million with the exception of $ 63.7 million of cash and cash equivalents and other current assets held by uniQure N.V and $ 85.2 million of other current assets and investment held by Corlieve Therapeutics SAS.

The 2021 Restated Facility contain provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable.

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As of December 31, 2022, the Company’s authorized share capital is € 4.0 million (or $ 4.3 million when translated at an exchange rate as of December 31, 2022, of $ 1.07 / €1.00), divided into 80,000,000 ordinary shares, each with a nominal value of € 0.05 .

The Company’s shareholders, at the 2021 Annual General Meeting of Stockholders held on June 16, 2021, approved an increase in the number of authorized ordinary shares by 20,000,000 to 80,000,000 million.

The Company’s shareholders, at the 2021 Annual General Meeting of Stockholders held on June 16, 2021, approved an increase in the number of authorized ordinary shares by 20,000,000 to 80,000,000 .

All ordinary shares issued by the Company were fully paid.

Besides the minimum amount of share capital to be held under Dutch law, there are no distribution restrictions applicable to the equity of the Company.

As of December 31, 2021, and 2020 and 2019 the Company’s other comprehensive result was restricted for payment of dividends for an accumulated other comprehensive loss of $ 28.9 million in 2021, an accumulated other comprehensive gain of $ 9.9 million in 2020, and an accumulated other comprehensive loss of $ 6.7 million in 2019.

As of December 31, 2022, and 2021 and 2020 the Company’s other comprehensive result was restricted for payment of dividends for an accumulated other comprehensive loss of $ 58.3 million in 2022, an accumulated other comprehensive loss of $ 28.9 million in 2021, and an accumulated other comprehensive gain of $ 9.9 million in 2020.

On March 1, 2021, the Company entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which the Company may, from time to time in its sole discretion, offer and sell through SVB Leerink, acting as agent, its ordinary shares, up to an aggregate offering price of $ 200.0 million.

2 unchanged sentences

The Company defers direct, incremental costs associated to this offering, except for the commission costs to SVB Leerink, which are a reduction to additional paid-in capital, and will deduct these costs from additional paid-in capital in the consolidated balance sheets proportionately to the amount of proceeds raised.

During the year ended December 31, 2021, $ 1.3 million of direct, incremental costs were deducted from additional paid-in capital.

During the year ended December 31, 2021, $ 1.3 million of direct, incremental costs were deducted from additional paid-in capital ( nil for the year ended December 31, 2022).

Following the Closing of the CSL Behring transaction, the Company consumed its tax net operating loss carryforwards from the years 2011 to 2018.

The Company allocated the tax benefit from the release of the valuation allowance related to net operating loss carryforwards generated by share issuance costs incurred in 2014, 2015, 2017 and 2018 to additional paid-in capital.

This resulted in an increase of additional paid-in capital of $ 3.0 million in the year ended December 31, 2021.

On September 10, 2019, the Company completed a follow-on public offering of 4,891,305 ordinary shares at a public offering price of $ 46.00 per ordinary share, and on September 13, 2019, the Company completed the sale of an additional 733,695 ordinary shares at a public offering price of $ 46.00 per ordinary share pursuant to the exercise by the underwriters of the option to purchase additional ordinary shares, resulting in total gross proceeds to the Company of $ 258.8 million.

The net proceeds to the Company from this offering were $ 242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company.

The Company deducted $ 0.6 million of expenses incurred related to this offering from additional paid-in capital in the accompanying consolidated balance sheets and reflected this within the proceeds from public offering of shares, net of issuance costs within the cash flows from financing activities.

In February 2019, the Company issued 37,175 ordinary shares to Hercules pursuant to exercised warrants for $ 0.5 million in aggregate cash consideration.

The Company deemed the sale and issuance of these shares to be exempt from registration under the Securities Act in reliance on Regulation S of the Securities Act, as an offshore offering of securities and such shares were issued as restricted shares.

Hercules represented to us that they were in compliance with the requirements of Regulation S.

This resulted is an increase of additional paid-in capital of $ 3.0 million in the year ended December 31, 2021.

The Company recorded $ 0.8 million increase of additional paid-in capital in the year ended December 31, 2022 resulting from the release of valuation allowance for the tax benefit of share issuance costs incurred in 2018, 2019 and 2021 within the Netherlands.

Share-based compensation

2 unchanged sentences

(in thousands)

Cost of manufacturing services revenue

Research and development

3 unchanged sentences

(in thousands)

Award type/ESPP

Share options

1 unchanged sentence

Performance share units

Employee share purchase plan

As of December 31, 2022, the unrecognized compensation cost related to unvested awards under the various share-based compensation plans were:

4 unchanged sentences

Performance share units

The Company satisfies the exercise of share options and vesting of Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) through newly issued shares.

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.

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 Company previously had a 2012 Equity Incentive Plan (the “2012 Plan”).

As of December 31, 2021, 14,000 fully vested share options are outstanding (December 31, 2020:

As of December 31, 2022, no fully vested share options are outstanding (December 31, 2021:

14,000 ) under the 2012 Plan.

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Outstanding at December 31, 2022

Thereof, fully vested and exercisable at December 31, 2021

Thereof, fully vested and exercisable on December 31, 2022

Thereof, outstanding and expected to vest after December 31, 2022

Outstanding and expected to vest at December 31, 2020

Outstanding and expected to vest after December 31, 2021

Total weighted average grant date fair value of options issued during the period (in $ millions)

54 unchanged sentences

Total weighted average grant date fair value of PSUs granted during the period (in $ millions)

The Company granted shares to certain employees in September and December 2021 that will be earned upon achievement of defined milestones.

The Company granted shares to certain employees in September and December 2021 and various dates during the year ended December 31, 2022 that will be earned upon achievement of defined milestones.

Earned shares will vest upon the later of a minimum service period of one year or three years , or the achievement of defined milestones, subject to the grantee’s continued employment.

1 unchanged sentence

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 and one of the five defined milestones was considered probable as of December 31, 2022.

In January 2018 and January and February 2019, the Company awarded PSUs to its executives and other members of senior management.

These PSUs were earned in January 2019 and January 2020, based on the Board’s assessment of the level of achievement of agreed upon performance targets through December 31, 2018, and December 31, 2019, respectively.

These PSUs were earned in January 2019 and January 2020, based on the Board of Directors’ (the “Board”) assessment of the level of achievement of agreed upon performance targets through December 31, 2018, and December 31, 2019, respectively.

The PSUs awarded for the year ended December 31, 2018 vested in February 2021 and the PSUs awarded for the year ended December 31, 2019 vested in January 2022.

The following table summarizes information about the weighted average grant-date fair value of the PSUs determined as of the date these were earned for the 2018 and 2019 PSUs, and the date of the grant for the 2021 PSUs:

The following table summarizes information about the weighted average grant-date fair value of the PSUs determined as of the date those were earned for the 2019 PSUs, and the date of the grant for the 2021 and 2022 PSUs:

Weighted average

11 unchanged sentences

4,724 and December 31, 2020:

As of December 31, 2021, a total of 127,302 ordinary shares remains available for issuance under the ESPP plan.

As of December 31, 2022, a total of 116,060 ordinary shares remain available for issuance under the ESPP plan.

Expenses by nature

4 unchanged sentences

Laboratory and development expenses

Legal and advisory expenses

Office and housing expenses

Legal and advisory expenses

Other operating expenses

Patent and license expenses

Depreciation and amortization expenses

Fair value loss - Corlieve contingent consideration

Patent and license expenses

Details of employee-related expenses for the years ended December 31 are as follows:

3 unchanged sentences

Share-based compensation expenses

Other employee expenses

Social security costs

Contractor expenses

Health insurance

Contractor expenses

Other employee expenses

Pension costs - defined contribution plans

2 unchanged sentences

The amount recognized in the year ended December 31, 2022 was $ 5.6 million compared to $ 5.3 million in 2021 and $ 1.9 million in 2020.

In addition, other income includes $ 2.6 million of employee retention credits received under the U.S.

In addition, other income included $ 2.6 million of employee retention credits received under the U.S.

Coronavirus Aid, Relief, and Economic Security Act, during the year ended December 31, 2021.

No such income was received in the years ended December 31, 2022 or December 31, 2020.

An additional $ 3.0 million of other income was recorded in the year ended December 31, 2021, related to the receipt by the Company of 69,899 shares of VectorY B.V.

1 unchanged sentence

entered into in April 2021.

No such income was recorded in 2020 and 2019.

In the year ended December 31, 2022, the Company recognized $ 0.3 million of other income related to the equity stake received in VectorY.

No such income was recorded in the year ending December 31, 2020.

In 2022, 2021 and 2020 the Company’s other income also consisted of income from the subleasing of a portion of the Amsterdam facility while other expense consists of expenses incurred in relation to the subleasing income.

Income tax expense / (benefit)

Income tax (benefit) / expense

Due to the uncertainty surrounding the realization of favorable tax attributes in future tax returns, the Company has recorded a valuation allowance against the Company’s net deferred tax assets in the Netherlands.

The Company released full valuation allowance against the Company’s net deferred tax assets in the United States as of December 31, 2020.

The Company released a full valuation allowance against the Company’s net deferred tax assets in the United States as of December 31, 2020.

In connection with the Corlieve acquisition, the Company recognized a deferred tax liability related to acquired identifiable intangible assets and a deferred tax asset for net operating tax loss carryforwards for a net of EUR 11.9 million ($ 14.2 million) as of the Acquisition Date.

There are no significant unrecognized tax benefits as of December 31, 2022 and 2021.

For the years ended December 31, 2021, 2020 and 2019, income / (loss) before income tax (expense) / benefit consists of the following:

For the years ended December 31, 2022, 2021 and 2020, (loss) / income before income tax benefit / (expense) consists of the following:

Years ended December 31,

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Total current tax (expense)

Deferred tax (expense) / benefit

Deferred tax benefit / (expense)

Dutch operations

Total deferred tax (expense) / benefit

Total income tax (expense) / benefit

Total deferred tax benefit / (expense)

Total income tax benefit / (expense)

Tax rate reconciliation

The reconciliation of the amount of income tax (expense) / benefit that would result from applying the Dutch statutory income tax rate to the Company’s reported amount of income tax (expense) / benefit for the years ended December 31, 2021, 2020 and 2019, is as follows:

The reconciliation of the amount of income tax benefit / (expense) that would result from applying the Dutch statutory income tax rate to the Company’s reported amount of (loss) / income before income tax benefit / (expense) for the years ended December 31, 2022, 2021 and 2020, is as follows:

Years ended December 31,

(in thousands)

Income / (loss) before income tax (expense) / benefit for the period

Expected income tax (expense) / benefit at the tax rate enacted in the Netherlands ( 25 %)

(Loss) / income before income tax benefit / (expense) for the period

Expected income tax benefit / (expense) at the tax rate enacted in the Netherlands (2022:

25.8 %, 2021:

25.0 %, 2020:

Non-deductible expenses

3 unchanged sentences

Release of valuation allowance related to expected future taxable income of U.S.

Income tax (expense) / benefit

Non-deductible expenses predominantly relate to share-based compensation expenses and affected the effective tax rate by an amount of $ 6.7 million in 2021 (2020:

Income tax benefit / (expense)

Non-deductible expenses predominantly relate to share-based compensation expenses.

These expenses affected the effective tax rate by an amount of $ 8.5 million in 2022 (2021:

$ 6.7 million;

$ 5.8 million).

The fair value loss on contingent consideration affected the effective tax rate by an amount of $ 2.0 million in 2021 ( nil in 2020 and 2019).

The fair value loss on contingent consideration affected the effective tax rate by an amount of $ 1.9 million in 2022 ($ 2.0 million and nil in 2021 and 2020, respectively).

Significant components of deferred taxes

10 unchanged sentences

Interest carryforwards

Derivative financial instrument

Total deferred tax assets

1 unchanged sentence

Deferred tax assets, net of valuation allowance

Acquired IPR&D Intangible Asset (see Note 3, "Corlieve transaction")

Acquired IPR&D Intangible Asset

Operating lease right-of-use assets

6 unchanged sentences

Changes recorded in the statement of operations

Changes recorded in equity

Increase related to 2021 and 2020 Dutch tax reforms

3 unchanged sentences

The Company released the full valuation allowance against the Company’s net deferred assets in the United States as of December 31, 2020.

Included within changes recorded in the statement of operations for the year ended December 31, 2020 and December 31, 2019 are benefits of $ 1.2 million and $ 0.8 million, respectively, from the utilization of U.S.

Included within changes recorded in the statement of operations for the year ended December 31, 2020 are benefits of $ 1.2 million from the utilization of U.S.

net operating loss carryforwards.

The valuation allowance as of December 31, 2021 is primarily related to net operating loss carryforwards in the Netherlands that, in the judgment of management, are not more-likely than-not to be realized.

The valuation allowance as of December 31, 2022 is primarily related to net operating loss carryforwards in the Netherlands.

As of December 31, 2022, the total amount of net operating losses carried forward under the Dutch tax regime was $ 264.0 million (December 31, 2021:

$ 228.5 million, 2020:

$ 588.2 million).

The Company has historically recorded a full valuation allowance.

The Company evaluates all positive and negative evidence including future income from the CSL Behring Agreement in assessing the need for such a full valuation allowance.

Management considered reversing taxable temporary differences, projected future taxable income and tax-planning strategies in making this assessment.

A valuation allowance was recorded against deferred tax assets if it is more likely than not that some or all the deferred tax assets will not be realized.

As of December 31, 2020, the Company had recorded a full valuation allowance against its Dutch net deferred tax assets.

On May 6, 2021, the CSL Behring Agreement became effective (refer to Note 4 “Collaboration arrangements and concentration of credit risk” ).

The Company recorded $ 462.4 million of license revenue related to closing the transaction.

The Company concluded that as of December 31, 2022, December 31, 2021 and December 31, 2020 it is more likely than not that the remaining deferred tax assets will not be realized.

The Company recorded $ 462.4 million of license revenue in May 2021 after the Closing of the CSL Behring transaction.

The Company recorded such revenue in its Dutch tax return related to the 12-month period ended December 31, 2020, which it filed on February 10, 2022.

1 unchanged sentence

The Company’s remaining Dutch net operating tax losses carried forward relate to 2019 and 2022.

The Dutch government on June 4, 2021 enacted legislation, whereby such net operating tax losses can be carried forward indefinitely.

The Company expects to continue incurring tax losses for the foreseeable future.

As such, the Company retains its valuation allowance related to the deferred assets as of December 31, 2021.

The Company allocated the tax benefit from the release of the valuation allowance related to net operating loss carryforwards generated by share issuance cost incurred in 2014, 2015, 2017 and 2018 to additional paid-in capital.

This resulted in an increase of additional paid-in capital as well as deferred tax expenses of $ 3.0 million.

A portion of the valuation allowance for deferred tax assets relates to follow-on offering costs incurred in 2019 and costs related to the at-the-market offering in 2021.

This resulted in an increase of additional paid-in capital as well as deferred tax expenses of $ 3.0 million during the year ended December 31, 2021.

A portion of the valuation allowance for deferred tax assets recorded as of December 31, 2022 continues to relate to follow-on offering costs incurred in 2019.

Any subsequently recognized tax benefits will be credited directly to contributed capital.

1 unchanged sentence

The Dutch corporate tax rate for fiscal years 2020 and 2021 was 25.0 %.

During 2019, the Dutch government enacted various changes to the corporate income tax rate applicable to future fiscal years.

In September 2020, further changes were enacted that retain the corporate income tax rate at 25 % from 2021 onwards.

In December 2021, even further changes were enacted that raised the corporate income tax rate from 25 % to 25.8 % from 2022 onwards.

A tax reform in December 2018 limited the carryforward of tax losses arising from January 1, 2019, to six years after the end of the respective period.

Tax losses incurred prior to this date continue to expire nine years after the end of the respective period.

In December 2021, further changes were enacted that raised the corporate income tax rate from 25.0 % to 25.8 % from 2022 onwards.

In June 2021 legislation was enacted allowing for an indefinite carryforward from fiscal year 2022 onwards of existing and future net operating loss carryforwards subject to a limit of offsetting taxable profit in excess of EUR 1.0 million to 50% of the taxable profit.

The Dutch fiscal unity as of December 31, 2021 has an estimated $ 228.5 million (2020:

$ 588.2 million;

$ 414.0 million) of taxable losses that are available for carry forward indefinitely.

In the year ended December 31, 2019, unused tax losses of $ 20.7 million expired.

The fiscal periods from 2020 onwards are still open for inspection by the Dutch tax authorities.

6 unchanged sentences

operations generated taxable income in the fiscal years 2018 to 2022.

Based on the current design of the Company’s worldwide operations, the Company expects to continue to generate taxable income in the U.S.

The Company expects to continue to generate taxable income in the U.S.

during the foreseeable future.

7 unchanged sentences

The Company is currently not under examination by the IRS for any tax years.

The French corporate tax rate for fiscal years 2021 was 26.5 %, as of January 1, 2022 the tax rate is decreased to 25 %.

The French corporate tax rate for fiscal year 2022 was 25 %.

In addition, the Company is subject to a surcharge of 3.3 % of the 25.0 % standard corporate tax rate resulting in a combined rate of 25.8 %.

The Company’s French operation has incurred losses since incorporation and is expected to continue incurring tax losses for the foreseeable future.

1 unchanged sentence

Basic and diluted earnings per share

Basic net income / (loss) per ordinary share is computed by dividing net income / (loss) for the period by the weighted average number of ordinary shares outstanding during the period.

Basic net (loss) / income per ordinary share is computed by dividing net (loss) / income for the period by the weighted average number of ordinary shares outstanding during the period.

Diluted earnings per ordinary share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares.

2 unchanged sentences

Year ended December 31,

(in thousands)

Net income / (loss) attributable to ordinary shares

(in thousands, except

share amounts)

Net (loss) / income attributable to ordinary shares

Weighted-average number of ordinary shares outstanding - basic

5 unchanged sentences

Year ended December 31,

(in thousands)

Anti-dilutive ordinary share equivalents

2 unchanged sentences

Employee share purchase plan

BMS warrants (derecognized as of December 1, 2020 - refer to Note 5, "Fair value measurement"

Total anti-dilutive ordinary share equivalents

The anti-dilutive ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that exceeded the price of the Company’s ordinary shares as of December 31, 2022 and December 31, 2020.

In addition, the BMS warrants were not exercisable as of December 31, 2019, since this would have required the prior designation of Collaboration Targets by BMS.

This would have resulted in a lower number of potentially dilutive ordinary shares as some stock option grants as well as the BMS warrants would have been excluded.

Commitments and contingencies

9 unchanged sentences

All transactions subsequent to the effective date of the amended BMS CLA are considered to no longer be with a related party due to the elimination of the potential obligations related to additional Collaboration Targets (see Note 4 “Collaboration arrangements and concentration of credit risk” ) as well as the elimination of the BMS warrants (see Note 7, “Fair value measurement” ).

On October 21, 2021, the Company held an Extraordinary General Meeting of its shareholders and Rachelle Jacques was appointed to the Board of Directors (the “Board”) as a non-executive director.

Jacques will also serve as a member of the Audit Committee of the Board effective as of October 21, 2021.

On June 16, 2021, the Company’s shareholders voted to approve the reappointment of Mr.

David Meek and Ms.

Paula Soteropoulos as non-executive directors of the Board.

Meek has been appointed chairman of the Board.

Philip Astley-Sparke did not stand for reappointment and retired from the Board on June 16, 2021.

On June 15, 2021, Christian Klemt was appointed as Chief Financial Officer.

Klemt was our Chief Accounting Officer from August 2017 to June 2021, and he will continue to serve as general manager of our Amsterdam site.

Matthew Kapusta, who has been our Chief Executive Officer since December 2016 and had been our Chief Financial Officer from January 2015 to June 2021, will continue to serve as our Chief Executive Officer.

In connection with his transition to Chief Financial Officer, Mr.

Klemt will also serve as our Principal Financial Officer.

On May 17, 2021, Pierre Caloz was appointed as Chief Operating Officer.

Caloz oversees all manufacturing operations, global CMC development and innovation, supply chain, and facilities.

On September 14, 2020, the Company appointed Ricardo Dolmetsch, Ph.D.

as President, Research and Development.

Dolmetsch succeeded Sander van Deventer, M.D., Ph.D., the former Executive Vice President, Research and Product Development.

On August 25, 2020, the Company entered into a separation agreement with Robert Gut, M.D., Ph.D., pursuant to which Dr.

Gut transitioned from his role as Chief Medical Officer on October 14, 2020, to be appointed a non-executive director of the Board of Directors.

On December 1, 2020, at an extraordinary general meeting, the Company’s shareholders voted to approve the appointment of Dr.

Gut as a non-executive director on the Board of Directors.

Gut had previously been appointed as a non-executive director on the Board of Directors on June 13, 2018 by the Company’s shareholders and had resigned as a non-executive director on August 20, 2018, to be appointed as the Company’s Chief Medical Officer.

On October 24, 2018, at an extraordinary general meeting, the Company’s shareholders voted to approve the appointment of Dr.

Gut as an executive director on the Board of Directors.

On June 17, 2020, the Company’s shareholders voted to approve the appointment of Leonard E.

Post, Ph.D., as a non-executive director of the Board of Directors.

Post replaced Dr.

David Schaffer, whose term as a non-executive director of the Board of Directors ended on the same date.

Post has also assumed the role of chair of the Company’s Research and Development Committee of the Board of Directors.

Subsequent events

On January 31, 2023, the Company announced that it had entered into a global licensing agreement for a gene therapy for amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 with Apic Bio.

The Company made an initial cash payment of $ 10.0 million.

In addition, the Company will pay Apic Bio up to $ 43.0 million in milestones upon achievement of regulatory approvals in the U.S.

and Europe and pre-specified annual net sales, and a tiered royalty on net sales ranging from the mid-single digits to low double digits.

EXHIBIT INDEX

22 unchanged sentences

001-36294) for the period ending on September 31, 2017 filed with the Securities and Exchange Commission).

Patent License Agreement (L-107-2007), effective as of May 2, 2007, by and between the Company and the National Institutes of Health, as amended on December 31, 2009, May 31, 2013, and November 11, 2013 (incorporated by reference to Exhibit 10.1 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on March 31, 2017 filed with the Securities and Exchange Commission).

Patent License Agreement (L-116-2011), effective as of August 10, 2011, by and between the Company and National Institutes of Health, as amended on May 31, 2013 and November 11, 2013 (incorporated by reference to Exhibit 10.2 of the Company’s quarterly report on Form 10-Q (file no.

001-36294) for the period ending on March 31, 2017 filed with the Securities and Exchange Commission).

Lease relating to 113 Hartwell Avenue, Lexington, Massachusetts, dated as of July 24, 2013, by and between the Company and King113 Hartwell LLC (incorporated by reference to Exhibit 10.28 of the Company's registration statement on Form F-1 (file no.

26 unchanged sentences

001-36294) filed on October 19, 2017 with the Securities and Exchange Commission).

Employment Agreement dated August 20, 2018 by and between uniQure, Inc.

Robert Gut (incorporated by reference to Exhibit 10.38 of the Company’s annual report on Form 10-K for the year ended December 31, 2018 (file no.

0001-36294) filed with the Securities and Exchange commission).

First Amendment Lease relating to 113 Hartwell Avenue, Lexington, Massachusetts, dated as of July 24, 2013, by and between the Company and King113 Hartwell LLC (incorporated by reference to Exhibit 10.1 of the Company's current report on form 8-K (file no.

9 unchanged sentences

001-36294) filed with the Securities and Exchange Commission) filed on September 20, 2019.

Employment Agreement, executed September 17, 2019, by and between the Company and Dr.

Sander van Deventer (incorporated by reference to Exhibit 10.2 of the Company’s current report on form 8-K (file no.

001-36294) filed with the Securities and Exchange Commission) filed on September 20, 2019.

Amended and Restated Employment Agreement, executed March 1, 2020 by and between uniQure biopharma B.V.

40 unchanged sentences

001-36294) for the period ending on September 30, 2021 filed with the Securities and Exchange Commission).

Form of Share Option Agreement, effective December 8, 2021, under the 2014 Share Incentive Plan.

Form of Restricted Stock Unit Award, effective December 8, 2021, under the 2014 Share Incentive Plan.

Form of Performance Stock Unit Award, effective December 8, 2021 under the 2014 Share Incentive Plan.

Third Amended and Restated Loan and Security Agreement as of December 15, 2021, by and among uniQure biopharma B.V., uniQure Inc., uniQure IP B.V., the Company and Hercules Capital Inc.

Form of Share Option Agreement, effective December 8, 2021, under the 2014 Share Incentive Plan (incorporated by reference to Exhibit 10.65 of the Company’s annual report on Form 10-K for the year ended December 31, 2021 (file no.