What changed in this filing

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Item 1. Financial Statements

2024-05-07 compared with 2023-11-07 · 113 added, 177 removed, 296 unchanged (49% of the section changed)

1 unchanged sentence

UNAUDITED CONSOLIDATED BALANCE SHEETS

September 30,

(in thousands, except share and per share amounts)

2 unchanged sentences

Current investment securities

Accounts receivable and contract asset

Accounts receivable

Inventories, net

Prepaid expenses

2 unchanged sentences

Non-current assets

Property, plant and equipment, net of accumulated depreciation of $ 51.6 million as of September 30, 2023 and $ 44.1 million as of December 31, 2022

Non-current investment securities

Property, plant and equipment, net of accumulated depreciation of $ 57.8 million as of March 31, 2024 and $ 55.7 million as of December 31, 2023

Operating lease right-of-use assets

Intangible assets, net, including in-process research and development asset of $ 56.6 million as of September 30, 2023 and $ 57.3 million as of December 31, 2022

Intangible assets, net, including in-process research and development asset of $ 57.8 million as of March 31, 2024 and $ 59.1 million as of December 31, 2023

Deferred tax assets, net

19 unchanged sentences

Ordinary shares, € 0.05 par value:

80,000,000 shares authorized as of September 30, 2023 and December 31, 2022 and 47,810,291 and 46,968,032 ordinary shares issued and outstanding as of September 30, 2023 and December 31, 2022, respectively

80,000,000 shares authorized as of March 31, 2024 and December 31, 2023 and 48,492,357 and 47,833,830 ordinary shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively

Additional paid-in-capital

6 unchanged sentences

COMPREHENSIVE LOSS

Three months ended September 30,

Nine months ended September 30,

(in thousands, except share and per share amounts)

Three months ended March 31,

(in thousands, except share and per share amounts)

4 unchanged sentences

Operating expenses:

Cost of license revenues

Cost of contract manufacturing revenues

6 unchanged sentences

Interest expense

Foreign currency gains / (losses), net

Other non-operating gains, net

Loss before income tax benefit

Income tax benefit

Other comprehensive loss:

Foreign currency translation adjustments

Foreign currency losses, net

Loss before income tax (expense) / benefit

Income tax (expense) / benefit

Other comprehensive (loss) / income:

Foreign currency translation (loss) / gains, net

Defined benefit pension gain, net of taxes

Total comprehensive loss

3 unchanged sentences

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE THREE MONTHS ENDED SEPTEMBER 30, 2023 AND 2022

Ordinary shares

comprehensive

shareholders’

(in thousands, except share and per share amounts)

Balance at June 30, 2022

Loss for the period

Other comprehensive loss

Exercises of share options

Restricted share units distributed during the period

Share-based compensation expense

Issuance of ordinary shares relating to employee stock purchase plan

Balance at September 30, 2022

Balance at June 30, 2023

Loss for the period

Other comprehensive loss

Exercises of share options

Restricted and performance share units distributed during the period

Share-based compensation expense

Issuance of ordinary shares relating to employee stock purchase plan

Balance at September 30, 2023

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

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE NINE MONTHS ENDED SEPTEMBER 30, 2023 AND 2022

FOR THE THREE MONTHS ENDED MARCH 31, 2024 AND 2023

Ordinary shares

4 unchanged sentences

Loss for the period

Other comprehensive loss

Other comprehensive income

Exercises of share options

2 unchanged sentences

Issuance of ordinary shares relating to employee stock purchase plan

Balance at September 30, 2022

Balance at March 31, 2023

Balance at December 31, 2023

Loss for the period

Other comprehensive loss

Exercises of share options

Restricted and performance share units distributed during the period

Other comprehensive loss, net

Restricted share units distributed during the period

Share-based compensation expense

Issuance of ordinary shares relating to employee stock purchase plan

Balance at September 30, 2023

Balance at March 31, 2024

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

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS

Nine months ended September 30,

Three months ended March 31,

(in thousands)

1 unchanged sentence

Adjustments to reconcile net loss to net cash used in operating activities:

Depreciation and amortization expense

Amortization of premium/discount on investment securities

Depreciation and amortization

Amortization of discount on investment securities

Share-based compensation expense

Royalty financing agreement interest expense

Deferred tax income

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

Unrealized foreign exchange losses / (gains), net

Deferred tax expense / (income)

Changes in fair value of contingent consideration

Unrealized foreign exchange losses, net

Other items, net

Changes in operating assets and liabilities:

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

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

Accounts payable

Accrued expenses, other liabilities, and operating leases

Contingent consideration milestone payment

Net cash used in operating activities

Cash flows from investing activities

Investment in investment securities

Proceeds on maturity of investment securities

Proceeds on maturity of debt securities

Investment in debt securities

Purchases of property, plant, and equipment

Acquisition of uniQure France SAS (formerly Corlieve Therapeutics SAS), net of cash acquired

Net cash used in investing activities

Net cash generated from investing activities

Cash flows from financing activities

Proceeds from royalty financing agreement

Payment of debt issuance costs

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

Contingent consideration milestone payment

Net cash generated from financing activities

10 unchanged sentences

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

NOTES TO CONSOLIDATED FINANCIAL STATEMENTS

1 General business information

(the “Company”) was incorporated on January 9, 2012, initially as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid ) under the laws of the Netherlands.

(the “Company”) was incorporated on January 9, 2012, as a private company with limited liability ( besloten vennootschap met beperkte aansprakelijkheid ) under the laws of the Netherlands.

The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results.

2 unchanged sentences

acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT.

Effective February 10, 2014, in connection with its initial public offering on the Nasdaq Global Select Market, the Company converted into a public company with limited liability ( naamloze vennootschap ) and changed its legal name from uniQure B.V.

Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability ( naamloze vennootschap ) and changed its legal name from uniQure B.V.

to uniQure N.V.

The Company is registered in the trade register of the Chamber of Commerce ( Kamer van Koophandel ) in Amsterdam, the Netherlands under number 54385229.

The Company is registered in the trade register of the Dutch Chamber of Commerce ( Kamer van Koophandel ) in Amsterdam, the Netherlands under number 54385229.

The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25a, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

2 unchanged sentences

2.1 Basis of preparation

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S.

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States (“U.S.

GAAP”) and applicable rules and regulations of the United States Securities and Exchange Commission (the “SEC”) regarding interim financial reporting.

9 unchanged sentences

GAAP have been omitted.

The results of operations for the three and nine months ended September 30, 2023, are not necessarily indicative of the results to be expected for the full year ending December 31, 2023, or for any other future year or interim period.

The results of operations for the three months ended March 31, 2024, are not necessarily indicative of the results to be expected for the full year ending December 31, 2024, or for any other future year or interim period.

The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 filed by the Company with the SEC on February 28, 2024 (the “Annual Report”).

1 unchanged sentence

The preparation of the financial statements in conformity with U.S.

GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods.

Actual results could differ from those estimates.

2 unchanged sentences

statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2023, and the notes thereto, which are included in the Annual Report .

There have been no material changes in the Company’s significant accounting policies during the nine months ended September 30, 2023, except as noted below.

Royalty Financing Agreement

In May 2023, uniQure biopharma B.V.

(“uniQure biopharma”), a wholly-owned subsidiary of the Company entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV (the “Purchaser”) to sell certain current and future royalties due to uniQure biopharma from CSL Behring LLC (“CSL Behring”) under the Commercialization and License Agreement (the “CSL Behring Agreement”) by and between uniQure biopharma and CSL Behring from the net sales of HEMGENIX®.

Refer to Note 9 “Royalty Financing Agreement” for further details of the Royalty Financing Agreement.

The Company determined that the Royalty Financing Agreement should be accounted for as debt in accordance with topic ASC 470, Debt .

The Company initially recognized the debt at fair value.

The Company subsequently records the debt at amortized cost and determines the effective interest rate based on its projection of contractual cash flows.

Interest expense (presented as “Interest Expense” in the consolidated statements of operations and comprehensive loss) is recorded over the projected repayment period using the effective interest method.

The Company periodically assesses and adjusts the effective interest rate to reflect changes in projected cash flows.

The Company prospectively applies the adjusted effective interest rate following the date of change.

In accordance with topic ASC 835, Interest , debt issuance costs incurred in relation to the Royalty Financing Agreement are presented as a reduction of carrying amount of the debt.

Debt issuance cost is amortized together with the interest expense recorded.

There have been no material changes in the Company’s significant accounting policies during the three months ended March 31, 2024.

2.5 Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 2.3.25 of the Annual Report , which could be expected to materially impact the Company’s unaudited consolidated financial statements.

There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2024, as compared to the recent accounting pronouncements described in Note 2.3.27 of the Annual Report , which could be expected to materially impact the Company’s unaudited consolidated financial statements.

CSL Behring collaboration

On June 24, 2020, uniQure biopharma B.V.

entered into the CSL Behring Agreement with CSL Behring, pursuant to which CSL Behring received exclusive global rights to HEMGENIX® .

entered into a commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”), pursuant to which CSL Behring received exclusive global rights to HEMGENIX® .

The transaction became fully effective on May 6, 2021.

License revenue

The Company recognized $ 0.5 million and $ 1.3 million of royalty revenue in each of the three and nine months ended September 30, 2023, compared to nil in the three and nine months ended September 30, 2022.

Royalties on the sale of the HEMGENIX® are recorded once earned and are presented as license revenue.

The Company recognized $ 1.2 million of royalty revenue in the three months ended March 31, 2024 , compared to nil in the three months ended March 31, 2023 .

Royalties on the sale of HEMGENIX® are recorded once earned and are presented as license revenue.

Accounts receivable and contract asset

As of December 31, 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 for a milestone due from CSL Behring following the first sale of HEMGENIX® in the U.S.

, which was collected in July 2023.

As of September 30, 2023, the Company had accounts receivable of $ 1.6 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue.

As of December 31, 2023, the Company recorded accounts receivable of $ 4.0 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue.

As of March 31, 2024 , the Company had accounts receivable of $ 10.6 million from CSL Behring related to collaboration services, contract manufacturing revenue and royalty revenue.

Investment securities

The following tables summarize the Company’s investments in sovereign debt as of September 30, 2023 and December 31, 2022:

At September 30, 2023

Amortized cost, as adjusted

The following tables summarize the Company’s investments in sovereign debt as of March 31, 2024 and December 31, 2023:

At March 31, 2024

Amortized cost

Gross unrealized holding gains

5 unchanged sentences

At December 31, 2023

Amortized cost, as adjusted

Amortized cost

Gross unrealized holding gains

4 unchanged sentences

Government debt securities (held-to-maturity)

Non-current investments:

Government debt securities (held-to-maturity)

The Company invests in short-term U.S.

3 unchanged sentences

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

The following table summarizes the inventory balances as of September 30, 2023 and December 31, 2022:

September 30,

Inventories, net

The following table summarizes the inventories, net balances as of March 31, 2024 and December 31, 2023:

(in thousands)

2 unchanged sentences

Finished goods

The Company recorded write downs to net realizable value of $ 1.8 million for the three months ended March 31, 2024 and nil for the same period in 2023.

The costs are recognized as Cost of Contract Manufacturing Revenues.

As at March 31, 2024, and December 31, 2023, the Company recorded an allowance for inventory of $ 2.7 million and $ 1.6 million, respectively.

Fair value measurement

9 unchanged sentences

The carrying amount of cash and cash equivalents, accounts receivable from licensing and collaboration partners, other assets, accounts payable, accrued expenses and other current liabilities reflected in the consolidated balance sheets approximate their fair values due to their short-term maturities.

The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of September 30, 2023, and December 31, 2022:

The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of March 31, 2024 and December 31, 2023 :

Quoted prices

12 unchanged sentences

Total liabilities

At September 30, 2023

At March 31, 2024

Cash and cash equivalents

8 unchanged sentences

Contingent consideration

The Company is required to pay up to EUR 178.8 million (or $ 189.1 million based on the foreign exchange rate on September 30, 2023) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of contractually defined milestones in connection with the Company’s July 2021 acquisition of uniQure France SAS.

In September 2023, a milestone payment of EUR 10.0 million ($ 10.6 million) was paid, of which EUR 8.9 million ($ 9.6 million) related to contingent consideration.

The fair value of the contingent consideration as of September 30, 2023 was $ 40.7 million (December 31, 2022:

The Company is required to pay up to EUR 178.8 million (or $ 193.0 million based on the foreign exchange rate on March 31, 2024) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of contractually defined milestones in connection with the Company’s July 2021 acquisition of uniQure France SAS.

The fair value of the contingent consideration as of March 31, 2024 was $ 42.2 million (December 31, 2023:

$ 43.0 million) using discount rates of approximately 14.8 % to 15.6 % (December 31, 2023:

15.3 % to 15.6 %).

Following the clearance of an Investigational New Drug (“IND”) application for AMT-260 in August 2023, the Company increased the probability of achieving a EUR 30.0 million ($ 31.7 million) milestone payment due to the first patient dosed in Phase I/II clinical trial from 66.0 % to 100.0 %.

This also resulted in an increase of, the probability that AMT-260 may advance to late-stage development and commercialization.

The Company recorded $ 14.2 million and $ 15.4 million in expenses related to the changes in the fair value of the contingent consideration in the three and nine months ended September 30, 2023, respectively, compared to $ 5.5 million and $ 7.5 million during the same period in 2022.

If as of September 30, 2023 the Company had assumed a 100 % likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to $ 72.0 million.

If as of September 30, 2023 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would be released to income.

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

The following table presents the changes in fair value of contingent consideration between December 31, 2022 and September 30, 2023:

The Company assumes the probability of achieving a EUR 30.0 million ($ 32.4 million) milestone payment following the dosing of the first patient in Phase I/II clinical trial to be 100 %.

If as of March 31, 2024 the Company had assumed a 100 % likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to $ 74.8 million.

If as of March 31, 2024 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.

The following table presents the changes in fair value of contingent consideration between December 31, 2023 and March 31, 2024:

consideration

2 unchanged sentences

Change in fair value (presented within research and development expenses)

Contingent consideration milestone payment

Currency translation effects

Balance at September 30, 2023

As of September 30, 2023, the Company classified $ 26.7 million of the total contingent consideration of $ 40.7 million as current liabilities.

Balance at March 31, 2024

As of March 31, 2024, the Company classified $ 27.6 million (December 31, 2023:

$ 28.2 million) of the total contingent consideration of $ 42.2 million (December 31, 2023:

$ 43.0 million) as current liabilities.

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

Investment securities

Refer to Note 4 “ Investment securities ” for the fair value of the investment securities as of September 30, 2023 and December 31, 2022.

Refer to Note 4 “ Investment securities ” for the fair value of the investment securities as of March 31, 2024 and December 31, 2023.

Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:

September 30,

(in thousands)

Accruals for goods received from and services provided by vendors-not yet billed

Personnel related accruals and liabilities

Accruals for goods received from and services provided by vendors-not yet billed

Liability owed to the Purchaser pursuant to the Royalty Financing Agreement

Accrued contract fulfillment costs and costs to obtain a contract

8 Long-term debt

5 unchanged sentences

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 % or (ii) 7.95 % plus the prime rate less 3.25 % per annum.

The Company paid a $ 2.5 million back-end fee in June 2023.

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 2023 Amended Facility, the Company owes a back-end fee of $ 4.9 million on December 1, 2025 and a back-end fee of $ 1.3 million on the Maturity Date.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2023 Amended Facility was $ 102.5 million as of September 30, 2023, compared to $ 103.8 million as of December 31, 2022, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the facility in the three and nine months ended September 30, 2023 was $ 3.0 million and $ 1.4 million, respectively, compared to a foreign currency loss of $ 6.6 million and $ 15.0 million during the same period in 2022.

Interest expense associated with the 2023 Amended Facility during the three and nine months ended September 30, 2023 was $ 3.7 million and $ 10.9 million, respectively, compared to $ 3.0 million and $ 8.1 million during the same period in 2022.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2023 Amended Facility was $ 103.3 million as of March 31, 2024, compared to $ 102.9 million as of December 31, 2023, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the facility in the three months ended March 31, 2024 was $ 2.3 million, compared to a foreign currency gain of $ 0.7 million during the same period in 2023.

Interest expense associated with the 2023 Amended Facility during the three months ended March 31, 2024 was $ 3.7 million, compared to $ 3.6 million during the same period in 2023.

Under the 2023 Amended Facility the Company must remain current in its periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the U.S.

3 unchanged sentences

In combination with other covenants, the 2023 Amended Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers, and directors, and to make dividend payments and other distributions to its shareholders.

The Company secured the facilities by directly or indirectly pledging its total assets of $ 871.6 million, less $ 1.0 million of cash and cash equivalents and other current assets held by the Company, $ 85.0 million of other current assets and investment held by uniQure France SAS as well as receivables sold to the Purchaser.

The Company secured the facilities by directly or indirectly pledging its total assets of $ 770.1 million, less $ 6.8 million of cash and cash equivalents and other current assets held by the Company, and $ 87.7 million of other current assets and investment held by uniQure France SAS as well as receivables sold to the Purchaser.

Under the 2023 Amended Facility, the occurrence of a material adverse effect, as defined therein, would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable.

As of September 30, 2023, the Company was in material compliance with all covenants and provisions.

As of March 31, 2024, the Company was in material compliance with all covenants and provisions.

9 Royalty Financing Agreement

On May 12, 2023, the Company entered into the Royalty Financing Agreement with the Purchaser.

On May 12, 2023, the Company entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P.

(the “Purchaser”).

Under the terms of the Royalty Financing Agreement the Company received an upfront payment of $ 375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to the Company.

The Company is also eligible to receive an additional $ 25.0 million milestone payment under the Royalty Financing Agreement if 2024 net sales of HEMGENIX® exceed certain thresholds, as set forth in the Royalty Financing Agreement.

The Company is also eligible to receive an additional $ 25.0 million milestone payment under the Royalty Financing Agreement if 2024 net sales of HEMGENIX® exceed a pre-specified threshold, as set forth in the Royalty Financing Agreement.

The Purchaser will receive 1.85 times the upfront payment (or $ 693.8 million) and 1.85 times the $ 25.0 million milestone payment (if paid) until June 30, 2032 (“First Hard Cap Date”) if such thresholds are met or, if such cap is not met by June 30, 2032, up to 2.25 times of the upfront and milestone payment (if paid) through December 31, 2038.

If 2024 net sales do not exceed the pre-specified threshold, the Company will be obligated to pay $ 25.0 million to the Purchaser but only to the extent that the Company achieves a future sales milestone under the CSL Behring Agreement.

If 2024 net sales do not exceed a pre-specified threshold, the Company will be obligated to pay $ 25.0 million to the Purchaser but only to the extent that the Company achieves a future sales milestone under the CSL Behring Agreement.

If such milestone payment is not due from CSL Behring, the Company is not obligated to pay any amounts to the Purchaser.

1 unchanged sentence

Net proceeds from the Royalty Financing Agreement, after deducting professional and financial advisory fees related to the transaction of $ 4.9 million, were $ 370.1 million.

The Company initially recorded these net proceeds as “Liability from royalty financing agreement” on its balance sheet as of closing of the transaction on June 5, 2023.

The Company initially recorded these net proceeds as “Liability from royalty financing agreement” at their fair market value on its balance sheet as of closing of the transaction on June 5, 2023.

Following the initial recognition, the Company records the debt at amortized cost.

3 unchanged sentences

Based on the Company’s projections the effective interest rate is expected to be within a range of 12.0 % per annum to 13.5 % per annum.

The Company would have recorded between $ 14.6 million and $ 16.5 million of interest expense through the nine months ended September 30, 2023 ($ 11.5 million and $ 13.0 million, respectively, for the three months ended September 30, 2023) if it had used 12.0 % or 13.5 %, instead of the $ 14.9 million recorded in the nine months ended September 30, 2023 ($ 11.8 million recorded through the three months ended September 30, 2023).

The Company would have recorded between $ 12.1 million and $ 13.7 million of interest expense through the three months ended March 31, 2024 ( nil for the three months ended March 31, 2023) if it had used 12.0 % or 13.5 %, instead of the $ 12.4 million recorded in the three months ended March 31, 2024 ( nil for the three months ended March 31, 2023).

The Company will prospectively update the effective interest rate at each reporting date based on updated projections.

The liability was initially recognized at fair value and inputs were considered Level 3 inputs.

The following table presents the movement in the liability related to the Royalty Financing Agreement between the closing of the transaction on June 5, 2023 and September 30, 2023:

The following table presents the movement in the liability related to the Royalty Financing Agreement between the December 31, 2023 and March 31, 2024:

Amount of liability

(in thousands)

Gross proceeds from royalty financing agreement on June 5, 2023

Debt issuance costs paid

Balance as of December 31, 2023 (includes $ 1.4 million presented as "Accrued expenses and other current liabilities)

Royalty payments to Purchaser

Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities")

Interest expense for the period June 5, 2023 to September 30, 2023

Liability owed to the Purchaser (presented as "Accrued expense and other current liabilities")

Interest expense for the period

Liability related to the royalty financing agreement

2 unchanged sentences

The number of shares authorized for issuance under the 2014 Plan is 14 ,351,471 .

The 2014 Plan expires on January 9, 2024.

In June 2018, the Company’s shareholders adopted and approved an employee share purchase plan (the “ESPP”) allowing the Company to issue up to 150,000 ordinary shares.

4 unchanged sentences

Share-based compensation expense recognized by classification included in the Consolidated Statements of Operations and Comprehensive Loss in relation to the 2014 Plans and the ESPP for the periods indicated below was as follows:

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Three months ended March 31,

(in thousands)

3 unchanged sentences

Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows:

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Three months ended March 31,

(in thousands)

4 unchanged sentences

Employee share purchase plan

As of September 30, 2023, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

As of March 31, 2024, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

Weighted average

8 unchanged sentences

Certain grants to non-executive directors vest in full after one year .

All share options must be exercised by the tenth anniversary of the initial grant date.

The following tables summarize share option activity under the 2014 Plans for the nine months ended September 30, 2023:

Any options that vest must be exercised by the tenth anniversary of the initial grant date.

The following tables summarize option activity under the 2014 Plans for the three months ended March 31, 2024:

Weighted average

2 unchanged sentences

Outstanding at December 31, 2023

Outstanding at September 30, 2023

Thereof, fully vested, and exercisable on September 30, 2023

Thereof, outstanding and expected to vest after September 30, 2023

Outstanding at March 31, 2024

Thereof, fully vested, and exercisable on March 31, 2024

Thereof, outstanding and expected to vest after March 31, 2024

Outstanding and expected to vest after December 31, 2023

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

Proceeds from option sales during the period (in $ millions)

The fair value of each share option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions:

Three months ended September 30,

Nine months ended September 30,

The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions:

Three months ended March 31,

Expected volatility

1 unchanged sentence

Risk free interest rate

3.71 % - 4.00 %

3.71 % - 4.10 %

2.12 % - 4.16 %

Expected dividend yield

The following table summarizes the RSU activity for the nine months ended September 30, 2023:

The following table summarizes the RSU activity for the three months ended March 31, 2024:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2023

Non-vested at September 30, 2023

Non-vested at March 31, 2024

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

RSUs vest over one to three years , as specified when granted.

RSUs generally vest over one to three years .

RSUs granted to non-executive directors vest one year from the date of grant.

The following table summarizes the PSU activity for the nine months ended September 30, 2023:

The following table summarizes the PSU activity for the three months ended March 31, 2024:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2023

Non-vested at September 30, 2023

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

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

Non-vested at March 31, 2024

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

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

In addition, portions of the ordinary shares granted in December 2021 to executives and other members of senior management are subject to achieving a minimum total shareholder return relative to the NASDAQ Biotechnology Index.

The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable.

As of September 30, 2023, two milestones had been achieved.

Additionally, another milestone is considered probable as of September 30, 2023.

During the nine months ended September 30, 2023, 12,270 ordinary shares were issued under the ESPP compared to 9,305 during the same period in 2022.

As of September 30, 2023, 103,790 ordinary shares remain available for issuance under the ESPP compared to a total of 117,997 as of September 30, 2022.

The Company recorded $ 0.1 million and $ 1.1 million deferred tax benefit in relation to its operations in the U.S.

and France during the three and nine months ended September 30, 2023, respectively.

The Company recorded $ 0.3 million and $ 1.3 million deferred tax benefit in relation to its operations in the U.S.

and France during the three and nine months ended September 30, 2022, respectively.

The effective income tax rate of ( 0.1 %) and ( 0.5 %) during the three and nine months ended September 30, 2023 is substantially lower than the enacted rate of 25.8 % in the Netherlands as the Company records a valuation allowance against its net deferred tax assets in the Netherlands.

The effective income tax rate during the three and nine months ended September 30, 2022 was ( 0.7 %) and ( 0.9 %), respectively, as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands.

As of March 31, 2024, two milestones had been achieved and vested in either 2022 or 2023.

Additionally, another two milestones are considered probable as of December 31, 2023 and March 31, 2024.

During the three months ended March 31, 2024, nil ordinary shares were issued under the ESPP compared to 2,495 during the same period in 2023.

As of March 31, 2024, 96,862 ordinary shares remain available for issuance under the ESPP compared to a total of 113,565 as of March 31, 2023.

The Company recorded $ 0.7 million deferred tax expense in relation to its operations in the U.S.

during the three months ended March 31, 2024.

The Company recorded $ 1.2 million deferred tax benefit in relation to its operations in the U.S.

and France during the three months ended March 31, 2023.

The effective income tax rate of 1.0 % during the three ended March 31, 2024 is substantially lower than the enacted rate of 25.8 % in the Netherlands as the Company records a valuation allowance against its net deferred tax assets in the Netherlands and a partial a valuation allowance against its net deferred tax assets in France.

The effective income tax rate during the three months ended March 31, 2023 was ( 1.5 %), as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands.

14 Basic and diluted earnings per share

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

As the Company has incurred a loss in the three and nine months ended September 30, 2023, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share for the three and nine months ended September 30, 2023.

The ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of September 30, 2023 and September 30, 2022, respectively.

As the Company has incurred a loss in the three months ended March 31, 2024, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share for the three months ended March 31, 2024.

The ordinary shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of March 31, 2024 and March 31, 2023, respectively.

The potentially dilutive ordinary shares are summarized below:

Three months ended September 30,

Nine months ended September 30,

Three months ended March 31,

Anti-dilutive ordinary share equivalents

3 unchanged sentences

13 Subsequent events

On October 5, 2023, the Company announced a reorganization plan (the “Reorganization”) pursuant to which the Company’s global workforce will be reduced by approximately 20 %.

As part of the Reorganization, more than half of the Company’s research and technology projects will be discontinued and a research lab in Lexington, Massachusetts will be closed.

The Company estimated that it will incur charges of approximately $ 2.3 million in connection with the reorganization related to cash expenditures for employee severance costs.

The Company also anticipates that there could be costs associated with the lab to be closed in Lexington and associated fixed assets if the carrying value is determined to not be recoverable as of the cease-use date, which is expected to be later in the fourth quarter of 2023.

The Company anticipates the costs associated with the reorganization will be incurred in the fourth quarter of 2023.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1. Financial Statements