What changed in this filing

/stocks — the workspaceLOADING

Item 1. Financial Statements

2025-05-09 compared with 2024-11-05 · 171 added, 258 removed, 292 unchanged (60% of the section changed)

1 unchanged sentence

UNAUDITED CONSOLIDATED BALANCE SHEETS

September 30,

(in thousands, except share and per share amounts)

3 unchanged sentences

Accounts receivable

Inventories, net

Prepaid expenses

2 unchanged sentences

Non-current assets

Property, plant and equipment, net of accumulated depreciation of $ 30.8 million as of September 30, 2024 and $ 55.7 million as of December 31, 2023

Property, plant and equipment, net of accumulated depreciation of $ 36.1 million as of March 31, 2025 and $ 31.7 million as of December 31, 2024

Other investments

Operating lease right-of-use assets

Intangible assets, net, including in-process research and development asset of $ 59.8 million as of September 30, 2024 and $ 59.1 million as of December 31, 2023

Intangible assets, net, including in-process research and development assets of $ 58.0 million as of March 31, 2025 and $ 55.6 million as of December 31, 2024

Deferred tax assets, net

4 unchanged sentences

Accrued expenses and other current liabilities

Current portion of contingent consideration

Current portion of operating lease liabilities

4 unchanged sentences

Operating lease liabilities, net of current portion

Contingent consideration, net of current portion

Contingent consideration

Deferred tax liability, net

3 unchanged sentences

Commitments and contingencies

Shareholders' equity

Shareholders' equity / (deficit)

Ordinary shares, € 0.05 par value:

80,000,000 shares authorized as of September 30, 2024 and December 31, 2023 and 48,738,874 and 47,833,830 ordinary shares issued and outstanding as of September 30, 2024 and December 31, 2023, respectively

80,000,000 shares authorized as of March 31, 2025 and December 31, 2024 and 54,698,479 and 48,988,087 ordinary shares issued and outstanding as of March 31, 2025 and December 31, 2024, respectively

Additional paid-in-capital

2 unchanged sentences

( 1,173,602 )

Total shareholders' equity

Total liabilities and shareholders' equity

( 1,129,965 )

Total shareholders' equity / (deficit)

Total liabilities and shareholders' equity / (deficit)

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

1 unchanged sentence

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)

14 unchanged sentences

Foreign currency gains / (losses), net

Loss before income tax (expense) / benefit

Income tax benefit / (expense)

Loss before income tax expense

Income tax expense

Other comprehensive (loss):

Foreign currency translation loss, net

Foreign currency translation losses, net

Defined benefit pension gain, net of taxes

Total comprehensive loss

Earnings per ordinary share - basic and diluted

Basic and diluted net loss per ordinary share

Weighted average shares used in computing basic and diluted net loss per ordinary share

Weighted average shares - basic and diluted

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

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE THREE MONTHS ENDED SEPTEMBER 30, 2024 AND 2023

FOR THE THREE MONTHS ENDED MARCH 31, 2025 AND 2024

Ordinary shares

1 unchanged sentence

shareholders’

(deficit) / equity

(in thousands, except share and per share amounts)

Balance at June 30, 2023

Loss for the period

Other comprehensive loss, net

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, 2023

Balance at June 30, 2024

( 1,012,326 )

Balance at December 31, 2023

Loss for the period

Other comprehensive loss, net

Exercises of share options

Restricted share units distributed during the period

Share-based compensation expense

Balance at September 30, 2024

( 1,056,704 )

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, 2024 AND 2023

Ordinary shares

comprehensive

shareholders’

(in thousands, except share and per share amounts)

Balance at March 31, 2024

Balance at December 31, 2024

( 1,129,965 )

Loss for the period

Other comprehensive loss, net

Follow-on public offering

Exercises of share options

1 unchanged sentence

Share-based compensation expense

Issuance of ordinary shares relating to employee stock purchase plan

Balance at September 30, 2023

Balance at December 31, 2023

Loss for the period

Other comprehensive loss, net

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, 2024

Balance at March 31, 2025

( 1,173,602 )

1 unchanged sentence

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS

Nine months ended September 30,

Three months ended March 31,

(in thousands)

4 unchanged sentences

Share-based compensation expense

Royalty financing agreement interest expense

Deferred tax expense / (income)

Royalty financing agreement interest expense, net of interest paid

Deferred tax expense

Changes in fair value of contingent consideration

Provision for inventory write-downs

Unrealized foreign exchange (gains) / losses, net

4 unchanged sentences

Accrued expenses, other liabilities, and operating leases

Contingent consideration milestone payment

Net cash used in operating activities

2 unchanged sentences

Investment in debt securities

Divestment of commercial manufacturing facility

Purchases of property, plant, and equipment

Net cash generated from / (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 public offering of shares in connection with follow on, net of issuance costs

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

Repayment of long-term debt

Contingent consideration milestone payment

Net cash generated (used in) / generated from financing activities

Net cash generated from financing activities

Currency effect on cash, cash equivalents and restricted cash

26 unchanged sentences

Any reference in these notes to applicable guidance is meant to refer to authoritative U.S.

GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update of the Financial Accounting Standards Board.

GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Updates of the Financial Accounting Standards Board.

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

4 unchanged sentences

The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented.

There has been a reclassification of $ 2.2 million from Other non-current assets to Other investments within the comparative consolidated balance sheets at December 31, 2023 to improve the consistency and comparability of the consolidated financial statements.

Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S.

GAAP have been omitted.

The results of operations for the three and nine months ended September 30, 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 results of operations for the three months ended March 31, 2025, are not necessarily indicative of the results to be expected for the full year ending December 31, 2025, 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, 2024 filed by the Company with the SEC on February 27, 2025 (the “Annual Report”) .

4 unchanged sentences

2.4 Accounting policies

The principal accounting policies applied in the preparation of these unaudited consolidated financial

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

There have been no material changes in the Company’s significant accounting policies during the nine months ended September 30, 2024 except as described within Note 3 “ Divestiture of commercial manufacturing activities ” and Note 5 “ CSL Behring collaboration .”

The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2024, and the notes thereto, which are included in the Annual Report .

There have been no material changes in the Company’s significant accounting policies during the three months ended March 31, 2025.

2.5 Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 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.

Divestiture of commercial manufacturing activities

Description of transaction

On June 29, 2024, affiliates of the Company agreed with Genezen Holdings Inc.

and its affiliate Genezen MA, Inc.

(together “Genezen”) to sell the Company’s commercial manufacturing activities located in Lexington, MA (the “Lexington Transaction”).

The Lexington Transaction closed on July 22, 2024 (the “Closing”).

Genezen extended offers of employment to a significant majority of the Company’s employees located at the Lexington facility (the “Lexington Facility”), with the remaining employees terminated effective August 30, 2024.

and uniQure biopharma B.V., both wholly owned subsidiaries of the Company, entered into an Asset Purchase Agreement (“APA”) with Genezen on June 29, 2024.

Pursuant to the APA, Genezen agreed to acquire the manufacturing equipment with a carrying value of $ 5.7 million and related manufacturing operations, inventory with a carrying value of $ 8.8 million and certain other assets with a carrying value of $ 12.3 million associated with the Lexington Facility on Closing.

As consideration, the Company received (i) shares of newly issued Series C preferred stock of Genezen Holdings Inc.

which are convertible into Genezen common stock and will accrue an 8.0 % per annum cumulative dividend, (ii) a convertible promissory note with a nominal amount of $ 12.5 million, bearing interest at 8.0 % per annum and maturing 63 months following the date of issuance and (iii) a right to purchase HEMGENIX® at terms considered favorable to market terms.

The Company recorded the Series C preferred stock in Genezen Holdings Inc.

issued to the Company at Closing at its fair market value of $ 12.5 million.

The Company subsequently measures these non-marketable equity securities at cost less any impairment, adjusted to fair value if there are observable price changes in orderly transactions for an identical or similar investment of the Genezen Holdings Inc., in accordance with topic ASC 321, Investments – Equity Securities.

The value of the Series C preferred stock was $ 12.5 million as of September 30, 2024.

The convertible promissory note was recognized at its fair value of $ 13.3 million at Closing.

The convertible promissory note is classified as not held for sale and subsequently measured at amortized cost, net of allowance for credit losses in accordance with topic ASC 310, Receivables as it did not meet the definition of a debt security under topic ASC 320, Investments – Debt Securities.

The Company accrues interest income on its convertible promissory note using the effective interest method over the convertible promissory note’s contractual term.

The convertible promissory note had a balance of $ 13.4 million and nil as of September 30, 2024 and December 31, 2023, respectively.

During the three and nine months ended September 30, 2024, the Company recognized $ 0.1 million and $ 0.1 million, respectively, of interest income.

As of September 30, 2024, the Company has not recorded an allowance for credit losses related to the convertible promissory note.

uniQure Inc., Genezen and the landlord of the Lexington Facility entered into an agreement for uniQure to assign and Genezen to assume the existing lease agreement between uniQure and the landlord at Closing.

The Company also amended its original July 2013 guarantee to continue guaranteeing rental payments owed by Genezen until the end of the current term on May 31, 2029.

In the event of Genezen’s default related to rental payments owed to the landlord, uniQure is entitled to terminate the assignment agreement and step into the original lease agreement.

On Closing, the Company de-recognized its right of use asset with a carrying amount of $ 11.8 million and related lease liability for the facility, of $ 17.9 million, which were classified as held-for-sale as at June 30, 2024.

Following the Closing, $ 1.7 million of deposits were released and reclassified from restricted cash into cash.

At Closing, uniQure Inc.

entered into a Commercial Supply Agreement (“CSA”) with Genezen.

Pursuant to the terms of the CSA, the parties agreed to subcontract the manufacturing of HEMGENIX® to Genezen.

The CSA includes a minimum term of three years and minimum purchase commitments of HEMGENIX® commercial supplies of $ 43.3 million over the first three years , unless certain contractual provisions are triggered.

The CSA provides the Company with rights to purchase HEMGENIX® at terms considered favorable to arms-length market terms.

In accordance with ASC 805 Business Combinations, the Company recorded an intangible asset valued at $ 16.7 million with respect to these favorable terms.

The intangible asset will be amortized on a straight-line basis over a three-year term, commencing at Closing.

Amortization expense of $ 1.2 million (presented within Other expense in the Consolidated Statement of Operations and Comprehensive Loss) was recorded during the three and nine-month periods ended September 30, 2024.

The Company’s obligations with respect to the supply of HEMGENIX® to CSL Behring pursuant to the Development and Commercial Supply Agreement between uniQure biopharma B.V.

and CSL Behring Inc (the “DCSA”) remain in effect notwithstanding the subcontracting to Genezen.

The Company expects to resell HEMGENIX® material purchased from Genezen via its minimum commitments to CSL Behring at a loss.

In accordance with ASC 330 Inventory, the Company, at Closing, recognized a liability of $ 8.8 million related to the net losses expected from the purchase of these minimum commitments.

The liability is accounted for as a reduction of the consideration received.

The Company will subsequently adjust the liability for any changes to the losses expected to be incurred.

Changes to the liability are recorded as Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.

As of September 30, 2024, the Company classified $ 2.4 million of the liability as accrued expenses and other current liabilities and $ 5.5 million within Other non-current liabilities in the Consolidated Balance Sheets.

The Lexington Transaction was accounted for as a divestment of the Company’s commercial manufacturing activities.

The net fair value of consideration received of $ 25.4 million, less costs associated with the sale of $ 3.3 million, exceeded the $ 20.9 million fair market value of the net assets transferred (including allocated goodwill) by $ 1.2 million.

The excess over the fair market value was recognized as a net gain in Other income on the Company’s Consolidated Statement of Operations and Comprehensive Loss.

At Closing, the Company paid a total of $ 8.3 million to Genezen and a third party related to adjustments of working capital and to obtain consent to proceed with the divestment.

Additionally, uniQure biopharma B.V.

entered into a development and other manufacturing services agreement (“DMSA”) with Genezen at Closing.

Pursuant to the DMSA, the Company is entitled to receive, as a preferred customer, manufacturing and development services to support the Company’s investigational gene therapy programs and other services related to HEMGENIX® (other than its manufacturing and supply obligations under the CSA).

The DMSA has a minimum term of three years and requires the Company to purchase services for a total minimum of $ 14.0 million.

Restructuring

As part of the Lexington Transaction, the Company terminated certain employees that did not transfer to Genezen.

The Company incurred $ 1.4 million of expenses related to termination benefits offered to these employees, which were recorded as Research and Development Expense and Selling, General and Administrative Expense during the nine-month period ended September 30, 2024.

On August 1, 2024, the Company announced an organizational restructuring (the “Restructuring”).

As a result, the Company recorded $ 4.1 million of severance and other personnel-related expenses during the three-month period ended September 30, 2024.

A summary of the charges for the three and nine months ended September 30, 2024 by major activity type is as follows ( nil in prior periods) :

Three months ended September 30,

Nine months ended September 30,

Severance and other personnel costs

(in thousands)

Research and development

General and administrative

In October 2023, the Company announced a reorganization and recorded a $ 1.0 million liability as of December 31, 2023 related to the termination of certain employees.

A summary of the changes in the severance and other personnel liabilities related to the workforce reduction, which are included within Accrued Expenses and Other Current Liabilities on the Consolidated Balance Sheets, are as follows:

Amount of liability

(in thousands)

Balance as of December 31, 2023

Severance and other personnel costs

Cash payments during the period

Balance as of September 30, 2024

CSL Behring collaboration

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

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

The transaction became fully effective on May 6, 2021.

License revenue

The Company recognized $ 2.1 million and $ 5.2 million of royalty revenue in the three and nine months ended September 30, 2024, respectively, compared to $ 0.5 million and $ 1.3 million of royalty revenue in the three and nine months ended September 30, 2023, respectively.

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

Manufacturing revenue

The Company recognized nil and $ 6.1 million of manufacturing revenue in the three and nine months ended September 30, 2024, respectively, compared to $ 0.3 million and $ 6.6 million of manufacturing revenue recorded in the three and nine months ended September 30, 2023, respectively.

The Company recognized contract manufacturing revenue when ownership transferred to CSL Behring.

Following the Closing of the Lexington Transaction, title to HEMGENIX® supply directly passes from the contract manufacturer, Genezen, to CSL Behring.

The Company does not control HEMGENIX® before it is transferred to CSL Behring.

The Company arranges for HEMGENIX® to be provided by Genezen to CSL Behring.

The Company determined that it is an agent in the sale of HEMGENIX® to CSL Behring.

In accordance with ASC 330 Inventory , at Closing, the Company recognized a liability of $ 8.8 million related to the expected net losses from the resale of HEMGENIX® to be purchased pursuant to its contractual minimum commitments .

As a result of the Company being an agent, the Company recognizes corresponding costs related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring and income related to the release of liabilities associated with expected net losses in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.

Collaboration revenue

The Company recognized $ 0.2 million and $ 10.6 million of collaboration revenue in the three and nine months ended September 30, 2024, respectively, compared to $ 0.6 million and $ 1.3 million of collaboration revenue in each of the three and nine months ended September 30, 2023.

In accordance with the DCSA, certain development and other services rendered by the Company are reimbursed by CSL Behring.

Accounts receivable

As of September 30, 2024 , the Company had accounts receivable of $ 5.0 million from CSL Behring related to collaboration services 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.

There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2025, as compared to the recent accounting pronouncements described in Note 2.3.29 of the Annual Report , which could be expected to materially impact the Company’s unaudited consolidated financial statements.

Other investments

Convertible promissory note

The Company has an investment in a convertible promissory note with a nominal amount of $ 12.5 million, bearing interest at 8.0 % per annum.

The convertible promissory note was issued in July 2024 and matures in October, 2029.

The convertible promissory note had a balance of $ 13.9 million as of March 31, 2025 (December 31, 2024:

$ 13.7 million), and is presented within “Other investments” on the Company’s unaudited consolidated balance sheets.

In the three month period ended March 31, 2025 and March 31, 2024, the Company recognized $ 0.2 million and nil of interest income, respectively.

As of March 31, 2025, the Company has not recorded an allowance for credit losses related to the convertible promissory note.

Investments valued under measurement alternative

The Company has investments in non-publicly traded equity securities without readily determinable fair values.

These investments are accounted for at cost, less impairment and adjusted to fair value if there are observable price changes in orderly transactions for identical or similar investments using the measurement alternative in accordance with ASC 321, Investments – Equity Securities .

The carrying value of these investments as of March 31, 2025 and December 31, 2024 was $ 14.4 million, and are presented within “Other investments” on the Company’s unaudited consolidated balance sheets.

The Company did not recognize any gains, losses, or impairments relating to investments carried under the measurement alternative in the three month period ended March 31, 2025 and March 31, 2024.

Investment securities

The following tables summarize the Company’s investments in sovereign debt as of September 30, 2024 and December 31, 2023:

At September 30, 2024

The following tables summarize the Company’s investments in government debt securities as of March 31, 2025 and December 31, 2024:

At March 31, 2025

Amortized cost

14 unchanged sentences

The Company invests in short-term U.S.

and European government bonds with the highest investment credit rating.

and European government debt securities with the highest investment credit rating.

and European government bonds are U.S.

dollar and euro denominated, respectively.

Investment securities with original maturities of 90 days or less when purchased are presented within cash and cash equivalents and measured at amortized cost (September 30, 2024:

Investment securities with original maturities of 90 days or less when purchased are presented within cash and cash equivalents and measured at amortized cost (March 31, 2025:

$ 43.4 million, December 31, 2024:

$ 15.3 million).

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

Inventories, net

The following table summarizes the inventories, net balances as of September 30, 2024 and December 31, 2023:

September 30,

(in thousands)

Raw materials

Work in progress

Finished goods

The Company recorded write downs to net realizable value of $ 0.5 million and $ 6.3 million in the three and nine months ended September 30, 2024, respectively, compared to nil in the same periods in 2023.

The costs are recognized as Cost of Contract Manufacturing Revenues.

At September 30, 2024, and December 31, 2023, the Company recorded an allowance for inventory of nil and $ 1.6 million, respectively.

The Company sold its inventories as part of the divestment of its commercial manufacturing activities (refer to Note 3 “ Divestiture of commercial manufacturing activities ” and Note 4 “Restructuring ”).

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, 2024 and December 31, 2023 :

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, 2025 and December 31, 2024 :

Quoted prices

12 unchanged sentences

Total liabilities

At September 30, 2024

At March 31, 2025

Cash and cash equivalents

8 unchanged sentences

Contingent consideration

The Company is required to pay up to EUR 178.8 million (or $ 199.6 million based on the foreign exchange rate on September 30, 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 September 30, 2024 was $ 41.4 million (December 31, 2023:

The Company is required to pay up to EUR 143.1 million (or $ 154.8 million based on the foreign exchange rate on March 31, 2025) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of the remaining contractually defined milestones in connection with the Company’s acquisition of uniQure France SAS.

The fair value of the contingent consideration as of March 31, 2025 was $ 12.6 million (December 31, 2024:

$ 10.9 million) using discount rates of approximately 14.8 % to 15.5 % (December 31, 2024:

15.3 % to 16.2 %).

The Company assumes the probability of achieving a EUR 30.0 million (or $ 33.5 million based on the foreign exchange rate on September 30, 2024) milestone payment following the dosing of the first patient in Phase I/II clinical trial of AMT-260 to be 100 %.

If, as of September 30, 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 $ 68.5 million.

If as of September 30, 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 September 30, 2024:

If, as of March 31, 2025, 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 $ 40.5 million.

If, as of March 31, 2025, 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 the contingent consideration between December 31, 2024 and March 31, 2025:

consideration

3 unchanged sentences

Currency translation effects

Balance at September 30, 2024

As of September 30, 2024, the Company classified $ 29.2 million (December 31, 2023:

$ 28.2 million) of the total contingent consideration of $ 41.4 million (December 31, 2023:

Balance at March 31, 2025

As of March 31, 2025, the Company classified nil (December 31, 2024:

nil ) of the total contingent consideration of $ 12.6 million (December 31, 2024:

$ 10.9 million) as current liabilities.

1 unchanged sentence

Investment securities

Refer to Note 6 “ Investment securities ” for the fair value of the investment securities as of September 30, 2024 and December 31, 2023.

Refer to Note 4 “ Investment securities ” for the fair value of the investment securities as of March 31, 2025 and December 31, 2024.

Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:

September 30,

(in thousands)

2 unchanged sentences

Current portion of firm purchase commitment liability

Liability owed to the Purchaser pursuant to the Royalty Financing Agreement

Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 8 "Royalty Financing Agreement")

7 Long-term debt

1 unchanged sentence

(formerly known as Hercules Technology Growth Capital, Inc.) (“Hercules”).

The facility was amended and restated in 2014, 2016, 2018, January 2021, December 2021 (the “2021 Restated Facility”), and on May 12, 2023 (the “2023 Amended Facility”) and July 22, 2024 (the “2024 Amended Facility”).

On July 19, 2024, in connection with the Closing of the Lexington Transaction and the amendment of the 2023 Amended Facility, the Company prepaid $ 50.0 million of the $ 100.0 million of principal outstanding as well as $ 3.1 million in end-of-term fees.

The facility was amended and restated in 2014, 2016, 2018, 2021 and on May 12, 2023 (the “2023 Amended Facility”) and July 22, 2024 (the “2024 Amended Facility”).

In July 2024, in connection with the divestment of the Company’s commercial manufacturing activities located in Lexington, MA and the amendment of the 2023 Amended Facility, the Company prepaid $ 50.0 million of the $ 100.0 million of principal outstanding as well as $ 3.1 million in end-of-term fees.

The 2023 Amended Facility extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”).

The total principal outstanding as of September 30, 2024 under the 2024 Amended Facility was $ 50.0 million.

The Company is required to repay the residual principal balance of $ 50.0 million on the Maturity Date.

The total principal outstanding as of March 31, 2025 under the 2024 Amended Facility was $ 50.0 million.

The Company is required to repay the residual 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.

Pursuant to the terms of the 2024 Amended Facility, the Company owes a back-end fee of $ 2.4 million on December 1, 2025 and a back-end fee of $ 0.6 million on the Maturity Date.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) was $ 51.7 million as of September 30, 2024, compared to $ 102.9 million as of December 31, 2023, and is recorded net of discount and debt issuance costs.

The foreign currency gain on the facility in the three months ended September 30, 2024 was $ 2.6 million and the foreign currency loss on the facility in the nine months ended September 30, 2024 was $ 0.4 million compared to a foreign currency loss of $ 3.0 million and $ 1.4 million, respectively, during the same periods in 2023.

Interest expense during the three and nine months ended September 30, 2024 was $ 3.7 million and $ 11.1 million, respectively, compared to $ 3.7 million and $ 10.9 million during the same periods in 2023.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) was $ 52.1 million as of March 31, 2025, compared to $ 51.9 million as of December 31, 2024, and is recorded net of discount and debt issuance costs.

The foreign currency gain on the facility in the three months ended March 31, 2025 was $ 2.1 million compared to a foreign currency loss of $ 2.3 million during the same period in 2024.

Interest expense during the three months ended March 31, 2025 was $ 1.8 million, compared to $ 3.7 million during the same period in 2024.

Under the 2024 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.

5 unchanged sentences

Under the 2024 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, 2024, the Company was in material compliance with all covenants and provisions.

As of March 31, 2025, the Company was in material compliance with all covenants and provisions of the Amended Facility.

8 Royalty Financing Agreement

1 unchanged sentence

(the “Purchaser”).

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

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

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

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

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

The Company has retained the rights to all other royalties, as well as contractual milestones totaling up to $ 1.3 billion, under the terms of the CSL Behring Agreement.

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

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 LLC’s (“CSL Behring”) worldwide net sales of HEMGENIX® for certain current and future royalties due to the Company.

The Company will be obligated to pay $ 25.0 million of the first worldwide sales milestone payment from CSL Behring, if received, to the Purchaser.

The Purchaser will receive 1.85 times the upfront payment (or $ 693.8 million) 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 through December 31, 2038 (“Second Hard Cap Date”).

If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to the Company.

The Company has no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.

The Company has retained the rights to all other royalties, as well as contractual milestones totaling up to $ 1.3 billion, under the terms of the commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”).

Net proceeds from the Royalty Financing Agreement were $ 370.1 million.

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

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

The Company expects to satisfy its commitment to the Purchaser prior to the First Hard Cap Date.

The Company will record the difference of $ 323.7 million between the total expected payments of $ 693.8 million to the Purchaser and the $ 370.1 million net proceeds as interest expense using the effective interest rate method.

The Company determined the effective interest rate based on the projected cash flows up to the First Hard Cap Date.

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

Interest expense during the three and nine months ended September 30, 2024 was $ 12.8 and $ 37.7 million, respectively, compared to $ 11.8 million and $ 14.9 million, during the same period in 2023.

The Company would have recorded between $ 37.4 million and $ 42.3 million of interest expense through the nine months ended September 30, 2024 ( $ 12.9 million and $ 14.6 million, respectively, for the three months ended September 30, 2024) if it had used 12.0 % or 13.5 % instead.

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

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

The following table presents the movement in the liability related to the Royalty Financing Agreement between the December 31, 2023 and September 30, 2024:

As of March 31, 2025 the Company expects to satisfy its commitment to the Purchaser prior to the Second Hard Cap Date.

The Company recorded the difference of $ 473.7 million between the total expected payments of $ 843.8 million to the Purchaser and the $ 370.1 million net proceeds as interest expense using the effective interest rate method.

The Company determined the effective interest rate based on the projected cash flows up to the Second Hard Cap Date.

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

The Company would have recorded the following amounts of interest expense if it had used the effective interest rate at the lower and upper end of this range:

Three months ended March 31,

(in millions)

Interest expenses at effective interest rate of 12.0 % per annum

Interest recorded in the statement of operations and comprehensive loss and income

Interest expenses at effective interest rate of 13.5 % per annum

The Company prospectively updates the effective interest rate at each reporting date based on updated cash flow projections.

The following table presents the movement in the liability related to the Royalty Financing Agreement between December 31, 2024 and March 31, 2025:

Amount of liability

1 unchanged sentence

Balance as of December 31, 2024 (includes $ 5.0 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")

Royalty payments to the Purchaser

Interest expense for the period

Liability related to the royalty financing agreement

Balance as of March 31, 2025 (includes $ 1.6 million presented as "Accrued expenses and other current liabilities)

9 Shareholders’ Equity

In January 2025, the Company received net proceeds of $ 70.1 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, through a follow-on public offering of 4.4 million ordinary shares at a public offering price of $ 17.00 per ordinary share.

In February 2025, the Company received an additional $ 10.6 million in net proceeds upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.

10 Share-based compensation

The Company’s share-based compensation plans include the amended and restated 2014 Share Incentive Plan (as amended, the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”).

At the annual general meeting of shareholders in June 2024, the Company’s shareholders approved an increase in the number of ordinary shares authorized for issuance under the 2014 Plan from 14,351,471 to 15,851,471 .

At the annual general meeting of shareholders in June 2024, the Company’s shareholders authorized an additional 1,500,000 shares for issuance.

As of March 31, 2025, a total of 3,912,283 ordinary shares remain available for issuance under the 2014 Plan.

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 / (income) 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, 2024, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

As of March 31, 2025, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

Weighted average

4 unchanged sentences

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 will account for the forfeitures of awards to employees impacted by the August 2024 restructuring as of the date employment with the Company ends, which is expected to occur between November 2024 and June 2025.

The Company expects this to reduce the unrecognized share-based compensation expense by approximately $ 3.2 million.

Share options

3 unchanged sentences

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 nine months ended September 30, 2024:

The following table summarizes option activity under the 2014 Plans for the three months ended March 31, 2025:

Weighted average

2 unchanged sentences

Outstanding at December 31, 2024

Outstanding at September 30, 2024

Thereof, fully vested, and exercisable on September 30, 2024

Thereof, outstanding and expected to vest after September 30, 2024

Outstanding at March 31, 2025

Thereof, fully vested, and exercisable on March 31, 2025

Thereof, outstanding and expected to vest after March 31, 2025

Outstanding and expected to vest after December 31, 2024

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

As a result of the August 2024 restructuring, 67,755 options are expected to be forfeited between November 2024 and June 2025.

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

Three months ended September 30,

Nine months ended September 30,

Three months ended March 31,

Expected volatility

1 unchanged sentence

Risk free interest rate

3.71 % - 4.00 %

3.71 % - 4.10 %

Expected dividend yield

The following table summarizes the RSU activity for the nine months ended September 30, 2024:

The following table summarizes the RSU activity for the three months ended March 31, 2025:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2024

Non-vested at September 30, 2024

Non-vested at March 31, 2025

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

1 unchanged sentence

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

As a result of the August 2024 restructuring, 229,113 RSUs are expected to be forfeited between November 2024 and June 2025.

The following table summarizes the PSU activity for the nine months ended September 30, 2024:

Awards of PSUs are subject to the achievement of specified performance objectives.

The fair value of PSUs granted in 2022 and 2024 is determined at the grant date by reference to share-price.

The fair value of PSUs awarded in 2024 and for which the service period precedes the grant date, is being determined by reference to the price of the Company’s ordinary shares as of March 31, 2025.

The following table summarizes the PSU activity for the three months ended March 31, 2025:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2024

Non-vested at September 30, 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, 2024, three milestones had been achieved.

Additionally, another milestone was achieved in October 2024.

During the nine months ended September 30, 2024, 10,150 ordinary shares were issued under the ESPP compared to 12,270 during the same period in 2023.

As of September 30, 2024, 86,712 ordinary shares remain available for issuance under the ESPP compared to a total of 103,790 as of September 30, 2023.

The Company recorded $ 0.0 million deferred tax benefit and $ 1.6 million deferred tax expense in relation to its operations in the U.S.

during the three and nine months ended September 30, 2024, respectively.

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 effective income tax rate of ( 0.1 %) and 1.0 % during the three and nine months ended September 30, 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 and nine months ended September 30, 2023 was ( 0.1 %) and ( 0.5 %), respectively, as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands.

Non-vested at March 31, 2025

PSUs awarded but not yet earned

Total non-vested and discretionary PSUs

In the three months ended March 31, 2025, there was an increase in the number of ordinary shares awarded, of 115,000 shares, related to the PSUs awarded but not yet earned, as a result of the increase of the expected payout from 100 % to 200 %.

During the three months ended March 31, 2025 and 2024, nil ordinary shares were issued under the ESPP.

As of March 31, 2025, 86,712 ordinary shares remain available for issuance under the ESPP compared to a total of 96,862 as of March 31, 2024.

Segment Reporting

The Company is advancing a pipeline of innovative gene therapies seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results.

The Company manages the operations related to these research and development activities within one operating segment because they rely on a common set of infrastructure, resources and technology of the products and production processes, types of customers, distribution methods and regulatory environment.

The leadership team is identified as the Chief Operating Decision Maker (“CODM”).

The CODM allocates resources to research projects and clinical candidates based on scientific data as well as quantitative and qualitative expected risk adjusted returns on investment.

The CODM uses segment operating loss to monitor that cash operating losses remain within the approved budget.

The accounting policies of the operating segment are the same as those described in the summary of significant accounting policies.

Three months ended March 31,

(in thousands)

Employee related expenses

Laboratory and development expenses

Professional fees

Facility expenses

Other segment items (1)

Segment operating loss

Reconciliation

Depreciation and amortization expense

Share-based compensation expense

Fair value loss - contingent consideration

Foreign currency gains / (losses), net

Interest income

Interest expense - Royalty Financing Agreement

Interest expense - Hercules debt facility

Other reconciling items

Consolidated loss before income tax expense

(1) Other segment items included in segment operating loss include costs related to intellectual property, information technology and insurance offset by income related to payments received from European authorities to subsidize the Company’s research and development in the Netherlands and France.

CSL Behring collaboration

In June 2020, uniQure biopharma B.V.

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

Following the divestiture of the Company’s commercial manufacturing activities located in Lexington, MA (“Lexington Transaction”), the Company determined that it is an agent in the sale of HEMGENIX® to CSL Behring.

As a result of the Company being an agent, the Company recognizes corresponding costs related to the purchase of HEMGENIX® from Genezen Holdings Inc.

and its affiliate Genezen MA, Inc.

(together “Genezen”) net of income from the sales of HEMGENIX® to CSL Behring as well as income related to the release of liabilities associated with expected net losses in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.

Accounts receivable related to collaboration services and royalty revenue were as follows:

(in thousands)

Accounts receivable

Other income for the three months ended March 31, 2025 and 2024:

Three months ended March 31,

(in thousands)

Sale of critical reagents to Genezen

Research and development grants from Dutch authorities

Sublease income

In the three months ended March 31, 2025 the Company recognized $ 6.0 million in income related to a one-time sale of critical reagents to Genezen.

There were no sales of critical reagents in the comparative prior period.

The Company recorded $ 0.5 million deferred tax expense in relation to its operations in the U.S.

during the three months ended March 31, 2025.

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 effective income tax rate of 1.1 % during the three months ended March 31, 2025 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 valuation allowance against its net deferred tax assets in France.

The effective income tax rate during the three months ended March 31, 2024 was 1.0 % as the Company had recorded a valuation allowance against its net deferred tax assets in the Netherlands and a partial valuation allowance against its net deferred tax asset in France.

15 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, 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 and nine months ended September 30, 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 September 30, 2024 and September 30, 2023, respectively.

Diluted earnings per share is 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 months ended March 31, 2025, 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, 2025.

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, 2025 and March 31, 2024, 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

4 unchanged sentences

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