What changed in this filing

/stocks — the workspaceLOADING

Item 1. Financial Statements

2025-11-10 compared with 2025-07-29 · 235 added, 150 removed, 356 unchanged (52% of the section changed)

1 unchanged sentence

UNAUDITED CONSOLIDATED BALANCE SHEETS

September 30,

(in thousands, except share and per share amounts)

7 unchanged sentences

Non-current assets

Property, plant and equipment, net of accumulated depreciation of $ 41.3 million as of June 30, 2025 and $ 31.7 million as of December 31, 2024

Property, plant and equipment, net of accumulated depreciation of $ 43.5 million as of September 30, 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 assets of $ 62.9 million as of June 30, 2025 and $ 55.6 million as of December 31, 2024

Intangible assets, net, including in-process research and development assets of $ 62.9 million as of September 30, 2025 and $ 55.6 million as of December 31, 2024

Deferred tax assets, net

4 unchanged sentences

Accrued expenses and other current liabilities

Liability related to pre-funded warrants

Income taxes payable

Current portion of operating lease liabilities

10 unchanged sentences

Commitments and contingencies

Shareholders' (deficit) / equity

Shareholders' equity / (deficit)

Ordinary shares, € 0.05 par value:

80,000,000 shares authorized as of June 30, 2025 and December 31, 2024 and 54,865,673 and 48,988,087 ordinary shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively

80,000,000 shares authorized as of September 30, 2025 and December 31, 2024 and 62,165,900 and 48,988,087 ordinary shares issued and outstanding as of September 30, 2025 and December 31, 2024, respectively

Additional paid-in-capital

3 unchanged sentences

( 1,129,965 )

Total shareholders' (deficit) / equity

Total liabilities and shareholders' (deficit) / equity

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 June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

(in thousands, except share and per share amounts)

14 unchanged sentences

Interest expense

Foreign currency gains / (losses), net

Foreign currency (losses) / gains, net

Other non-operating losses, net

Loss before income tax expense

Income tax expense

Income tax (expense) / benefit

Other comprehensive loss:

Foreign currency translation losses, net

Foreign currency translation gains / (losses), net

Defined benefit pension gain, net of taxes

Total comprehensive loss

Earnings per ordinary share - basic and diluted

Loss per ordinary share - basic and diluted

Basic and diluted net loss per ordinary share

1 unchanged sentence

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

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ (DEFICIT) / EQUITY

FOR THE THREE MONTHS ENDED JUNE 30, 2025 AND 2024

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY / (DEFICIT)

FOR THE THREE MONTHS ENDED SEPTEMBER 30, 2025 AND 2024

Ordinary shares

1 unchanged sentence

shareholders’

(deficit) / equity

equity / (deficit)

(in thousands, except share and per share amounts)

Balance at March 31, 2024

Balance at June 30, 2024

( 1,012,326 )

Loss for the period

Other comprehensive loss, net

Exercise 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 June 30, 2024

Balance at September 30, 2024

( 1,056,704 )

Balance at March 31, 2025

Balance at June 30, 2025

( 1,211,321 )

Loss for the period

Other comprehensive loss, net

Other comprehensive gain, net

Follow-on public offering

Tax benefit related to historical share issuance costs

Exercises of share options

1 unchanged sentence

Share-based compensation expense

Balance at June 30, 2025

Balance at September 30, 2025

( 1,291,850 )

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ (DEFICIT) / EQUITY

FOR THE SIX MONTHS ENDED JUNE 30, 2025 AND 2024

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

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY / (DEFICIT)

FOR THE NINE MONTHS ENDED SEPTEMBER 30, 2025 AND 2024

Ordinary shares

1 unchanged sentence

shareholders’

(deficit) / equity

equity / (deficit)

(in thousands, except share and per share amounts)

2 unchanged sentences

Other comprehensive loss, net

Exercises of share options

Restricted share units distributed during the period

1 unchanged sentence

Issuance of ordinary shares relating to employee stock purchase plan

Balance at June 30, 2024

Balance at September 30, 2024

( 1,056,704 )

3 unchanged sentences

Other comprehensive loss, net

Follow-on public offering

Follow-on public offerings

Tax benefit related to historical share issuance costs

Exercises of share options

1 unchanged sentence

Share-based compensation expense

Balance at June 30, 2025

Balance at September 30, 2025

( 1,291,850 )

1 unchanged sentence

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS

Six months ended June 30,

Nine months ended September 30,

(in thousands)

7 unchanged sentences

Changes in fair value of contingent consideration

Unrealized foreign exchange (gains) / losses, net

Provision for inventory write-downs

Changes in fair value of liability related to pre-funded warrants

Unrealized foreign exchange gains, net

Allowance for inventory write-downs

Other items, net

2 unchanged sentences

Accounts payable

Income tax payable

Accrued expenses, other liabilities, and operating leases

3 unchanged sentences

Investment in debt securities

Divestment of commercial manufacturing facility

Purchases of property, plant, and equipment

Net cash generated from investing activities

Cash flows from financing activities

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

Net cash generated from financing activities

Cash flows generated from / (used in) financing activities

Proceeds from follow-on public offerings of ordinary shares, net of issuance costs

Proceeds from issuance of pre-funded warrants, net of issuance costs

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

Repayment of long-term debt

Net cash generated from / (used in) financing activities

Currency effect on cash, cash equivalents and restricted cash

19 unchanged sentences

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.

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

The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.

13 unchanged sentences

GAAP have been omitted.

The results of operations for the three and six months ended June 30, 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 results of operations for the three and nine months ended September 30, 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”) .

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 period.

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

Actual results could differ from those estimates.

1 unchanged sentence

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 six months ended June 30, 2025.

There have been no material changes in the Company’s significant accounting policies during the nine months ended September 30, 2025, except as noted below:

Liability related to pre-funded warrants

As part of the Company’s September 2025 follow-on public offering, the Company issued pre-funded warrants (the “Pre-Funded Warrants”), denominated in U.S.

As the Pre-Funded Warrants are not denominated in the Company’s functional currency, they are not indexed to the Company’s ordinary shares in accordance with ASC 815-40, Derivatives and Hedging, Contracts in Entity’s Own Equity .

The obligation to issue ordinary shares at an offering price of $ 47.50 per ordinary share, less a $ 0.0001 per ordinary share exercise price, is classified as a liability.

The liability related to pre-funded warrants is accounted for at fair value in accordance with ASC 820, Fair Value Measurement .

Changes in the fair value of the liability related to pre-funded warrants are recognized within Other non-operating items, net in the Company’s Consolidated Statements of Operations and Comprehensive Loss.

The liability related to pre-funded warrants is classified as current due to the Pre-Funded Warrant holder’s right to exercise at any time.

The Company made an accounting policy election to exclude the Pre-Funded Warrants from the denominator in the computation of basic net loss per ordinary share, as such instruments are not considered outstanding ordinary shares.

2.5 Recent accounting pronouncements

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

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

2 unchanged sentences

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

The convertible promissory note had a balance of $ 14.1 million as of June 30, 2025 (December 31, 2024:

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

In the three and six months ended June 30, 2025, the Company recognized $ 0.3 million and $ 0.5 million of interest income, respectively, compared to nil during the same periods in 2024.

As of June 30, 2025, the Company has not recorded an allowance for credit losses related to the convertible promissory note.

The convertible promissory note had a balance of $ 14.4 million as of September 30, 2025 (December 31, 2024:

$ 13.7 million) and is presented within Other investments on the Company’s Consolidated Balance Sheets.

In the three and nine months ended September 30, 2025, the Company recognized $ 0.2 million and $ 0.7 million of interest income, respectively, compared to nil during the same periods in 2024.

As of September 30, 2025, the Company has not recorded an allowance for credit losses related to the convertible promissory note.

Investments valued under measurement alternative

1 unchanged sentence

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 June 30, 2025 and December 31, 2024 was $ 15.6 million and $ 13.8 million, respectively, which 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 and six month periods ended June 30, 2025 and June 30, 2024.

The carrying value of these investments as of September 30, 2025 and December 31, 2024 was $ 15.6 million and $ 13.8 million, respectively, as presented within Other Investments on the Company’s Consolidated Balance Sheets.

The Company did not recognize any gains, losses, or impairments relating to investments carried under the measurement alternative in the three and nine month periods ended September 30, 2025 and September 30, 2024.

Investment securities

The following tables summarize the Company’s investments in government debt securities as of June 30, 2025 and December 31, 2024:

At June 30, 2025

The following tables summarize the Company’s investments in government debt securities as of September 30, 2025 and December 31, 2024:

At September 30, 2025

Amortized cost

14 unchanged sentences

The Company invests in short-term U.S.

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

and European government debt securities with high investment credit ratings.

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 (June 30, 2025:

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, 2025:

$ 119.3 million, December 31, 2024:

3 unchanged sentences

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

ASC 820, Fair Value Measurements and Disclosures requires disclosure of methodologies used in determining the reported fair values and establishes a hierarchy of inputs used when available.

ASC 820 , Fair Value Measurement requires disclosure of the methodologies used in determining the reported fair values and establishes a hierarchy of inputs used when available.

The three levels of the fair value hierarchy are described below:

5 unchanged sentences

A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

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

The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of June 30, 2025 and December 31, 2024 :

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 Company’s 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, 2025 and December 31, 2024 :

Quoted prices

2 unchanged sentences

(in thousands)

At December 31, 2024

Balance at December 31, 2024

Cash and cash equivalents

7 unchanged sentences

Total liabilities

At June 30, 2025

Balance at September 30, 2025

Cash and cash equivalents

2 unchanged sentences

Other non-current assets

Liability related to pre-funded warrants

Contingent consideration

4 unchanged sentences

Contingent consideration

The Company is required to pay up to EUR 143.1 million (or $ 168.0 million based on the foreign exchange rate on June 30, 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 June 30, 2025 was $ 15.9 million (December 31, 2024:

The Company is required to pay up to EUR 143.1 million (or $ 167.9 million based on the foreign exchange rate on September 30, 2025) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of the remaining contractually defined milestones related to the development of AMT-260 in connection with the Company’s acquisition of uniQure France SAS.

The fair value of the contingent consideration as of September 30, 2025 was $ 17.8 million (December 31, 2024:

$ 10.9 million) using discount rates of approximately 12.1 % to 12.5 % (December 31, 2024:

15.3 % to 16.2 %).

If, as of June 30, 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 $ 51.4 million.

If, as of June 30, 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 June 30, 2025:

If, as of September 30, 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 $ 57.2 million.

If, as of September 30, 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 September 30, 2025:

consideration

3 unchanged sentences

Currency translation effects

Balance at June 30, 2025

As of June 30, 2025, the Company classified nil (December 31, 2024:

nil ) of the total contingent consideration of $ 15.9 million (December 31, 2024:

$ 10.9 million) as current liabilities.

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

Balance at September 30, 2025

The Company classified the total contingent consideration liability as non-current as of September 30, 2025 and December 31, 2024.

The classification of the contingent consideration within the Company’s Consolidated Balance Sheets 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 June 30, 2025 and December 31, 2024.

Refer to Note 4 “ Investment securities ” for the fair value of the investment securities as of September 30, 2025 and December 31, 2024.

Liability related to pre-funded warrants

In September 2025, the Company completed a follow-on public offering which included the issuance of Pre-Funded Warrants to purchase 526,316 of the Company’s ordinary shares at the public offering price of $ 47.50 per ordinary share, less a $ 0.0001 per ordinary share exercise price for each Pre-Funded Warrant.

The obligation to issue ordinary shares upon the exercise of the Pre-Funded Warrants is classified as a liability related to pre-funded warrants.

The liability related to pre-funded warrants is accounted for at fair value in accordance with ASC 820, Fair Value Measurement , with the total gross proceeds received with respect to the Pre-Funded Warrants determined as reflective of the fair value of the obligation on the date of issuance.

Changes in fair value of the liability related to pre-funded warrants are recognized within Other non-operating losses, net in the Company’s Consolidated Statements of Operations and Comprehensive Loss.

The Pre-Funded Warrants are not exchange-quoted and are measured using a valuation technique whose significant inputs are observable;

therefore the fair value measurement is classified within Level 2 of the fair value hierarchy.

Liability related

to pre-funded

(in thousands)

Balance at December 31, 2024

Issuance of pre-funded warrants in connection with follow-on public offering

Unrealized change in fair value

Balance at September 30, 2025

The fair value of the liability related to pre-funded warrants as of September 30, 2025 was $ 30.7 million (December 31, 2024:

nil ), classified as current.

Classification is based upon the Pre-Funded Warrant holder’s right to exercise at any time.

During the three and nine months ended September 30, 2025, the Company recorded a $ 5.7 million remeasurement in the fair value of the liability related to pre-funded warrants, respectively.

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

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

(As of September 30, 2025, $ 15.7 million (December 31, 2024:

$ 0.1 million) related to cash proceeds from employees stock option exercises)

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

Current portion of firm purchase commitment liability

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

Other current liabilities

7 Long-term debt

On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc.

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

The facility was amended and restated in 2014, 2016, 2018, 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 June 30, 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 $ 52.3 million as of June 30, 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 and six months ended June 30, 2025 was $ 4.3 million and $ 6.4 million, respectively, compared to a foreign currency loss of $ 0.7 million and $ 3.0 million, respectively, during the same period in 2024.

Interest expense during the three and six months ended June 30, 2025 was $ 1.8 million and $ 3.6 million, respectively, compared to $ 3.7 million and $ 7.4 million, respectively, during the same periods 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.

equivalent to the lesser of (i) 65 % of the outstanding balance of principal due or (ii) 100 % of worldwide cash and cash equivalents.

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

The Company is required to keep a minimum of unrestricted cash equal to at least 30 % of the loan amount outstanding.

The 2024 Amended Facility restricts the Company’s ability, among other things, to incur future indebtedness and obtain additional debt financing, make investments in securities or in other companies, transfer assets, perform certain corporate changes, make loans to employees, officers, and directors, and make dividend payments and other distributions to its shareholders.

The 2024 Amended Facility is secured by a direct or indirect pledge of the Company’s total assets of $ 584.9 million, less $ 69.7 million of cash and cash equivalents and other current assets held by the Company, and $ 89.8 million of other current assets and investment held by uniQure France SAS as well as receivables sold to HemB SPV, L.P.

(the “Purchaser”).

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 June 30, 2025, the Company was in material compliance with all covenants and provisions of the 2024 Amended Facility.

(“Hercules”).

The facility has been amended at various times, including on July 22, 2024 (the “2024 Amended Facility”) and most recently on September 22, 2025 (the “2025 Amended Facility), upon which date the amendment closed (the “Amendment Closing Date”).

On the Amendment Closing Date, the Company drew down $ 50.0 million (the “Tranche 1 Advance”) of the 2025 Amended Facility to repay the $ 50.0 million then outstanding under the 2024 Amended Facility.

A facility charge of $ 0.2 million was incurred on the Amendment Closing Date.

The 2024 Amended Facility was due on January 7, 2027 and bore interest at a rate equal to the greater of (i) 7.95 % and (ii) 7.95 % plus the prime rate less 3.25 % per annum, reflecting a floating rate of 11.95 % at the Amendment Closing Date.

Pursuant to the 2025 Amended Facility, additional term loans of $ 125.0 million were made available.

The Company may draw down (i) an additional $ 100.0 million in two increments of at least $ 25.0 million (the “Tranche 2 Advances”), following the approval of a Biologics License Application (“BLA”) for AMT-130 prior to June 15, 2027, provided that confirmatory trials to the extent and in the manner required to support full approval (if applicable) remain ongoing or are being planned, and (ii) an additional $ 25.0 million in a single advance (the “Tranche 3 Advance”), subject to approval by Hercules.

Advances under the 2025 Amended Facility bear interest at a rate equal to the greater of (i) 9.45 % and (ii) the sum of the prime rate and 2.45 %, reflecting a current floating rate of 9.70 %.

The 2025 Amended Facility matures on October 1, 2030, upon which date the Company is required to repay the principal balance and any unpaid interest on advances received.

The interest-only period of the 2025 Amended Facility is effective through October 1, 2028.

The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to June 15, 2027.

The 2025 Amended Facility will be interest-only if certain commercial milestones are met prior to December 31, 2028.

The Company may prepay all or part of the outstanding principal together with a prepayment charge of 1.50 % if repayment occurs within the first 30 months following the Amendment Closing Date, and thereafter, with a prepayment charge of 0.75 %.

Pursuant to the terms of the 2025 Amended Facility, the Company owes a back-end fee on December 1, 2025.

Absent a default or prepayment of the loan, a back-end fee of $ 0.6 million is due on January 5, 2027.

An end-of-term charge of 2.75 % of the aggregate principal outstanding is due if repaid within 18 months of the Amendment Closing Date, otherwise 5.50 % if repaid thereafter.

The total principal outstanding as of September 30, 2025 under the 2025 Amended Facility was $ 50.0 million.

The amortized cost, including interest due presented as part of Accrued expenses and other current liabilities, was $ 52.4 million as of September 30, 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 loan facilities in the three and nine months ended September 30, 2025 was $ 2.8 million and $ 9.2 million, respectively, compared to a foreign currency gain of $ 2.6 million and a foreign currency loss of $ 0.4 million, respectively, during the same periods in 2024.

Interest expense during the three and nine months ended September 30, 2025 was $ 1.8 million and $ 5.4 million, respectively, compared to $ 3.7 million and $ 11.1 million, respectively, during the same periods in 2024.

The Company repaid $ 50.0 million of the outstanding principal amount of the 2024 Amended Facility on July 19, 2024.

Under the 2025 Amended Facility, the Company must remain current in its periodic reporting requirements.

Unless the Company maintains a minimum market capitalization threshold of at least $ 1,200.0 million, the Company, beginning January 1, 2027 (the “Test Date”), is required at all times to keep a minimum cash balance deposited in bank accounts in the U.S.

of at least the lesser of (i) 65 % of the outstanding balance of aggregate principal due, provided that such percentage will be reduced to 50 % upon the occurrence of the approval of a BLA for AMT-130 prior to June 15, 2027, and further reduced to 35 % upon the occurrence of the contractually defined commercial milestones or (ii) 100 % of worldwide cash and cash equivalents.

The 2025 Amended Facility also provides for a certain performance covenant related to AMT-130 net product revenue which would apply nine months past a potential BLA approval of AMT-130, unless the Company maintains a minimum market capitalization threshold of at least $ 1,200.0 million.

The 2025 Amended Facility restricts the Company’s ability, among other things, to incur future indebtedness and obtain additional debt financing, make investments in securities or in other companies, transfer assets, perform certain corporate changes, own or hold certain digital assets, make loans to employees, officers, and directors, and make dividend payments and other distributions to its shareholders.

The 2025 Amended Facility is secured by a direct or indirect pledge of the Company’s total assets of $ 888.4 million, less $ 80.6 million of cash and cash equivalents and other current assets held by the Company, and $ 88.7 million of other current assets and investments held by uniQure France SAS as well as receivables sold to HemB SPV, L.P.

as described in Note 8 “ Royalty Financing Agreement” .

In accordance with the 2025 Amended Facility, the Company was in compliance with all applicable covenants as of September 30, 2025.

8 Royalty Financing Agreement

In May 2023, the Company entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with the Purchaser.

In May 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 LLC’s (“CSL Behring”) worldwide net sales of etranacogene dezaparvovec-drlb (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”).

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 the upfront payment 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.

2 unchanged sentences

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.

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 the date the transaction closed on June 5, 2023.

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

As of June 30, 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.

As of September 30, 2025 the Company expects to satisfy its commitment to the Purchaser prior to the Second Hard Cap Date.

The Company records 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 over the term 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.

As of September 30, 2025 the effective interest rate is expected to be within a range of 10.5 % to 12.0 % per annum, compared to a range of 12.0 % to 13.5 % per annum as of September 30, 2024.

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 June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

(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

Interest expense at effective interest rate of 10.5 % per annum for the three months and nine months ended September 30, 2025, and 12.0 % per annum for the three and nine months ended September 30, 2024, respectively

Interest recorded in the Consolidated Statements of Operations and Comprehensive Loss

Interest expense at effective interest rate of 12.0 % per annum for the three months and nine months ended September 30, 2025, and 13.5 % per annum for the three and nine months ended September 30, 2024, respectively

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 June 30, 2025:

The following table presents the movement in the liability related to the Royalty Financing Agreement between December 31, 2024 and September 30, 2025:

Amount of liability

3 unchanged sentences

Interest expense for the period

Balance as of June 30, 2025 (includes $ 5.2 million presented as "Accrued expenses and other current liabilities)

9 Shareholders' (deficit) / equity

Balance as of September 30, 2025 (includes $ 3.6 million presented as "Accrued expenses and other current liabilities")

9 Shareholders' equity / (deficit)

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 $ 10.4 million in net proceeds after related offering expenses upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.

In July 2025, the Company entered into a Sales Agreement with Leerink Partners LLC (“Leerink Partners”) with respect to an at-the-market (“ATM”) offering program, under which the Company may, from time to time and at the Company’s sole discretion, offer and sell through Leerink Partners, acting as agent, the Company’s ordinary shares, up to an aggregate offering of $ 200.0 million.

As of September 30, 2025, no sales have been made under the ATM offering program.

In September 2025, the Company received net proceeds of $ 323.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by the Company, through a follow-on public offering of 6.7 million ordinary shares, at a public offering price of $ 47.50 per ordinary share, and, in lieu of ordinary shares to certain investors, Pre-Funded Warrants to purchase 0.5 million of the Company’s ordinary shares at the public offering price less a $ 0.0001 per ordinary share exercise price.

The Company has authorized and reserved a sufficient number of ordinary shares to permit the full exercise of all outstanding Pre-Funded Warrants.

The Pre-Funded Warrants are classified as a liability.

In connection with the recognition as taxable income of the $ 375.0 million upfront payment received in relation to the Royalty Financing Agreement, and the consumption of $ 30.3 million of losses carried forward, as well as the release of the valuation allowance related to these losses carried forward (refer to Note 14, “I ncome Taxes” ), the Company allocated the tax benefit from the release of the valuation allowance related to net operating loss carryforwards generated by share issuance costs incurred in 2019 to additional paid-in capital.

This resulted in an increase in additional paid-in capital of $ 0.9 million in the three and nine months ended September 30, 2025.

10 Share-based compensation

1 unchanged sentence

At the annual general meeting of shareholders in June 2025, the Company’s shareholders authorized an additional 2,400,000 shares for issuance under the 2014 Plan.

As of June 30, 2025, a total of 4,484,501 ordinary shares remain available for issuance under the 2014 Plan.

As of September 30, 2025, a total of 4,414,685 ordinary shares remain available for issuance under the 2014 Plan.

In June 2018, the Company’s shareholders adopted and approved the uniQure N.V.

6 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 June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Cost of manufacturing services revenue

Research and development

Selling, general and administrative

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

Three months ended June 30,

Six months ended June 30,

Cost of contract manufacturing revenues

Research and development expenses

Selling, general and administrative expenses

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)

4 unchanged sentences

Performance share units

Employee share purchase plan

As of June 30, 2025, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

As of September 30, 2025, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

Weighted average

9 unchanged sentences

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

The following table summarizes option activity under the 2014 Plans for the six months ended June 30, 2025:

The following table summarizes option activity under the 2014 Plans for the nine months ended September 30, 2025:

Weighted average

2 unchanged sentences

Outstanding at December 31, 2024

Outstanding at June 30, 2025

Thereof, fully vested, and exercisable on June 30, 2025

Thereof, outstanding and expected to vest after June 30, 2025

Outstanding at September 30, 2025

Thereof, fully vested, and exercisable on September 30, 2025

Thereof, outstanding and expected to vest after September 30, 2025

Outstanding and expected to vest after December 31, 2024

1 unchanged sentence

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 June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

Expected volatility

3 unchanged sentences

Expected dividend yield

The following table summarizes the RSU activity for the six months ended June 30, 2025:

The following table summarizes the RSU activity for the nine months ended September 30, 2025:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2024

Non-vested at June 30, 2025

Non-vested at September 30, 2025

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

2 unchanged sentences

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 determined by reference to the price of the Company’s ordinary shares as of June 30, 2025.

The following table summarizes the PSU activity for the six months ended June 30, 2025:

The fair value of PSUs granted in 2022 and 2024 was determined on the grant date by reference to the market price of the Company’s ordinary shares.

The following table summarizes the PSU activity for the nine months ended September 30, 2025:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2024

Non-vested at June 30, 2025

PSUs awarded but not yet granted

Total non-vested and discretionary PSUs

In the six months ended June 30, 2025, there was an increase in the number of ordinary shares awarded, of 115,000 shares, related to the PSUs awarded but not yet granted, as a result of the increase of the expected payout from 100 % to 200 % .

During the six months ended June 30, 2025 nil ordinary shares were issued under the ESPP compared to 10,150 during the same period in 2024.

As of June 30, 2025, 86,712 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 as of June 30, 2024.

Non-vested at September 30, 2025

During the nine months ended September 30, 2025, nil ordinary shares were issued under the ESPP compared to 10,150 ordinary shares issued during the same period in 2024.

As of September 30, 2025, 86,712 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 ordinary shares as of September 30, 2024.

Segment Reporting

5 unchanged sentences

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

Three months ended June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

(in thousands)

10 unchanged sentences

Fair value loss - contingent consideration

Foreign currency gains / (losses), net

Fair value loss - liability related to pre-funded warrants

Foreign currency gains, net

Interest income

6 unchanged sentences

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 in July 2024 (“Lexington Transaction”), the Company determined that it is an agent in the sale of HEMGENIX® to CSL Behring.

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

Following the divestiture of the Company’s commercial manufacturing activities located in Lexington, MA in July 2024 (the “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.

2 unchanged sentences

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

September 30,

(in thousands)

Accounts receivable

Other income for the three and six months ended June 30, 2025 and 2024:

Three months ended June 30,

Six months ended June 30,

Other income for the three and nine months ended September 30, 2025 and 2024:

Three months ended September 30,

Nine months ended September 30,

(in thousands)

3 unchanged sentences

Sublease income

In the three and six months ended June 30, 2025 the Company recognized nil and $ 6.0 million in income, respectively, 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 and $ 0.9 million deferred tax expense in relation to its operations in the U.S.

during the three and six months ended June 30, 2025, respectively.

The Company recorded $ 0.9 million and $ 1.6 million deferred tax expense in relation to its operations in the U.S.

during the three and six months ended June 30, 2024, respectively.

The effective income tax rate of 1.1 % and 1.1 % during the three and six months ended June 30, 2025 (during the three and six months ended June 30, 2024, 1.7 % and 1.3 %, respectively) 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.

15 Basic and diluted earnings per share

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 and six months ended June 30, 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 and six months ended June 30, 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 June 30, 2025 and June 30, 2024, respectively.

Gain on sale of commercial manufacturing activities

In the three and nine months ended September 30, 2025 the Company recognized nil and $ 6.0 million in income, respectively, related to a one-time sale of critical reagents to Genezen.

There were no sales of critical reagents in the comparative period in 2024.

The effective income tax rate of 12.0 % and 6.3 % during the three and nine months ended September 30, 2025 (during the three and nine months ended September 30, 2024, ( 0.1 %) and 1.0 %, respectively) was substantially lower than the enacted rate of 25.8 % in the Netherlands as the Company recorded 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.

Since 2023, the Company has been engaged with the Dutch tax authorities regarding the tax treatment of the $ 375.0 million upfront payment received in exchange for the rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX®.

Refer to Note 8, “ Royalty Financing Agreement” .

In September 2025, the Dutch tax authorities communicated their position that, for Dutch corporate income tax purposes, the upfront payment would be treated as taxable income in 2023.

This would result in a taxable profit instead of a net operating loss for that year.

On a provisional basis, the recognition of the $ 375.0 million upfront payment as taxable income resulted in the consumption of $ 30.3 million of losses carried forward, as well as the release of the associated valuation allowance.

During the three and nine months ended September 30, 2025, the Company recorded, on a discrete basis, an estimate of $ 7.5 million of current tax expense related to the Dutch operations and $ 1.1 million of related interest expense, in accordance with ASC 740, Income Taxes .

These estimates remain subject to change upon finalization of the 2023 filing position.

In connection with the estimate and associated release of the valuation allowance, the Company allocated part of such release to the tax benefit of share issuance costs incurred in 2019.

This resulted in an increase in additional paid-in capital as well as deferred tax expenses of $ 0.9 million.

The following table summarizes the income tax (expense) / benefit for the three and nine months ended September 30, 2025 and 2024, respectively:

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Current tax expense

Dutch operations

Total current income tax expense

Deferred tax (expense) / benefit

Dutch operations

Total deferred tax (expense) / benefit

Total income tax (expense) / benefit

15 Basic and diluted loss per share

Diluted loss 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 incurred a loss in the three and nine months ended September 30, 2025 and September 30, 2024, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of diluted loss per share for the three and nine months ended September 30, 2025 and 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, 2025 and September 30, 2024, respectively.

The potentially dilutive ordinary shares are summarized below:

Three months ended June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

Anti-dilutive ordinary share equivalents

1 unchanged sentence

Non-vested RSUs and PSUs

Pre-Funded Warrants

Total anti-dilutive ordinary share equivalents

16 Subsequent events

As of September 30, 2025, the Company recorded its liability related to pre-funded warrants (refer to Note 5, “ Fair value Measurement ”) at a fair value of $ 30.7 million.

The Company determined the fair value of the liability using the closing market price of its ordinary shares of $ 58.37 per ordinary share as of September 30, 2025.

On November 3, 2025 the Company announced preliminary feedback from the FDA based on discussions at the Company’s pre-BLA meeting.

Following such announcement, there was a decrease of the market price of the Company’s ordinary shares to $ 34.29 per ordinary share as of the close of the market on November 3, 2025.

This reduced the fair value of the liability related to pre-funded warrants by $ 12.7 million to $ 18.0 million.

The Company will determine the fair value of its liability related to pre-funded warrants based on the price of its ordinary shares at the close of the market on December 31, 2025.

The Company will record the difference between the fair value as of September 30, 2025 and December 31, 2025 in income during the twelve month period ended December 31, 2025.

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