What changed in this filing

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Item 1. Financial Statements

2026-05-05 compared with 2025-11-10 · 157 added, 292 removed, 299 unchanged (60% 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 $ 43.5 million as of September 30, 2025 and $ 31.7 million as of December 31, 2024

Property, plant and equipment, net

Other investments

Operating lease right-of-use assets

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

Intangible assets, net

Deferred tax assets, net

5 unchanged sentences

Liability related to pre-funded warrants

Income taxes payable

Current portion of operating lease liabilities

10 unchanged sentences

Commitments and contingencies

Shareholders' equity / (deficit)

Shareholders' equity

Ordinary shares, € 0.05 par value:

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

80,000,000 shares authorized at March 31, 2026, and December 31, 2025, and 63,033,249 and 62,336,717 shares issued and outstanding at March 31, 2026, and December 31, 2025, respectively.

Additional paid-in-capital

3 unchanged sentences

( 1,328,936 )

Total shareholders' equity / (deficit)

Total liabilities and shareholders' equity / (deficit)

Total shareholders' equity

Total liabilities and shareholders' equity

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)

License revenues

Contract manufacturing revenues

Collaboration revenues

Total revenues

1 unchanged sentence

Cost of license revenues

Cost of contract manufacturing revenues

Research and development expenses

6 unchanged sentences

Foreign currency (losses) / gains, net

Other non-operating losses, net

Other non-operating gains, net

Loss before income tax expense

Income tax (expense) / benefit

Income tax expense

Other comprehensive loss:

Foreign currency translation gains / (losses), net

Defined benefit pension gain, net of taxes

Foreign currency translation losses, net

Total comprehensive loss

3 unchanged sentences

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

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY / (DEFICIT)

FOR THE THREE MONTHS ENDED SEPTEMBER 30, 2025 AND 2024

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE THREE MONTHS ENDED MARCH 31, 2026 AND 2025

Ordinary shares

1 unchanged sentence

shareholders’

equity / (deficit)

(in thousands, except share and per share amounts)

Balance at June 30, 2024

Balance at December 31, 2024

( 1,129,965 )

1 unchanged sentence

Other comprehensive loss, net

Exercise of share options

Restricted share units distributed during the period

Share-based compensation expense

Balance at September 30, 2024

( 1,056,704 )

Balance at June 30, 2025

( 1,211,321 )

Loss for the period

Other comprehensive gain, net

Follow-on public offering

Tax benefit related to historical share issuance costs

Exercises of share options

Restricted share units distributed during the period

Restricted and performance share units distributed during the period

Share-based compensation expense

Balance at September 30, 2025

Balance at March 31, 2025

( 1,173,602 )

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

comprehensive

shareholders’

equity / (deficit)

(in thousands, except share and per share amounts)

Balance at December 31, 2025

( 1,328,936 )

Loss for the period

2 unchanged sentences

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

( 1,056,704 )

Balance at December 31, 2024

( 1,129,965 )

Loss for the period

Other comprehensive loss, net

Follow-on public offerings

Tax benefit related to historical share issuance costs

Exercises of share options

Restricted and performance share units distributed during the period

Share-based compensation expense

Balance at September 30, 2025

Balance at March 31, 2026

( 1,382,471 )

1 unchanged sentence

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS

Nine months ended September 30,

Three months ended March 31,

(in thousands)

8 unchanged sentences

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

Unrealized foreign exchange gains, net

Allowance for inventory write-downs

Unrealized foreign exchange losses / (gains), net

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 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

Cash flows from financing activities

Proceeds from follow-on public offering of ordinary shares, 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

Net cash generated from financing activities

Currency effect on cash, cash equivalents and restricted cash

Net increase in cash, cash equivalents and restricted cash

Cash, cash equivalents and restricted cash at beginning of period

Cash, cash equivalents and restricted cash at the beginning of period

Cash, cash equivalents and restricted cash at the end of period

4 unchanged sentences

Cash paid for interest

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

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

25 unchanged sentences

GAAP have been omitted.

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”) .

The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the results to be expected for the full year ending December 31, 2026, 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, 2025 filed by the Company with the SEC on March 2, 2026 (the “Annual Report”).

2.3 Use of estimates

4 unchanged sentences

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, 2025, 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, 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.

There have been no material changes in the Company’s significant accounting policies during the three months ended March 31, 2026.

2.5 Recent accounting pronouncements

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

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 $ 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

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 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 September 30, 2025 and December 31, 2024:

At September 30, 2025

There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2026, 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.

Current investment securities

The following tables summarize the Company’s investments in government debt securities as of March 31, 2026 and December 31, 2025:

At March 31, 2026

Amortized cost

3 unchanged sentences

(in thousands)

Current investments:

Current investments securities:

Government debt securities (held-to-maturity)

5 unchanged sentences

(in thousands)

Current investments:

Current investments securities:

Government debt securities (held-to-maturity)

2 unchanged sentences

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, 2025:

$ 119.3 million, December 31, 2024:

$ 15.3 million).

dollar and euro (€, or EUR) 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.

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

As of March 31, 2026 and December 31, 2025, no expected credit loss was identified with respect to the investment securities.

Fair value measurement

8 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 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 :

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 Unaudited Consolidated Balance Sheets approximate their fair values due to their short-term maturities.

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

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

Restricted cash is included in Other non-current assets within the unaudited Consolidated Balance Sheets.

Investment securities are measured at amortized cost.

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, 2026 and December 31, 2025 :

Quoted prices

Classification in Consolidated

Classification in Unaudited Consolidated

Balance Sheets

5 unchanged sentences

Other non-current assets

Liability related to pre-funded warrants

Contingent consideration

3 unchanged sentences

Total liabilities

Balance at September 30, 2025

Balance at March 31, 2026

Cash and cash equivalents

9 unchanged sentences

Total liabilities

Contingent consideration

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 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:

a) Contingent consideration

The Company is required to pay up to EUR 143.1 million (or $ 164.5 million based on the foreign exchange rate on March 31, 2026) 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 March 31, 2026 was EUR 14.8 million ($ 17.0 million) (December 31, 2025:

EUR 15.9 million ($ 18.7 million)) using discount rates of approximately 11.5 % to 11.8 % (December 31, 2025:

If, as of March 31, 2026, 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 EUR 47.8 million ($ 55.0 million).

If, as of March 31, 2026, 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 (presented within non-current liabilities) between December 31, 2025 and March 31, 2026:

consideration

3 unchanged sentences

Currency translation effects

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 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.

Balance at March 31, 2026

The Company classified the total contingent consideration liability as non-current as of March 31, 2026 and December 31, 2025.

The classification of the contingent consideration within the Company’s Unaudited 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.

b) 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 (“Pre-Funded Warrant(s)”).

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;

4 unchanged sentences

Balance at December 31, 2025

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.

Unrealized change in fair value (presented within Other non-operating gains, net)

Currency translation effects

Balance at March 31, 2026

Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:

September 30,

(in thousands)

Personnel-related accruals and liabilities

(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

Personnel-related accruals and liabilities

Current portion of firm purchase commitment liability (refer to Note 13, "Commitments and contingencies" )

Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 7, " Royalty Financing Agreement ")

1 unchanged sentence

6 Long-term debt

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

(“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.

The total principal outstanding as of March 31, 2026 under the Company's amended venture debt loan facility with Hercules Capital, Inc.

(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 $ 50.3 million as of March 31, 2026, compared to $ 50.1 million as of December 31, 2025, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the loan facility in the three months ended March 31, 2026 was $ 1.1 million, compared to a foreign currency gain of $ 2.1 million during the same period in 2025.

Interest expense during the three months ended March 31, 2026 was $ 1.4 million, compared to $ 1.8 million during the same period in 2025.

During the three months ended March 31, 2026, there were no material changes to the terms, available borrowing tranches, covenants, or collateral arrangements of the 2025 Amended Facility as described in Note 11, “Long-term debt” to the consolidated financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

As of March 31, 2026, the Company was in compliance with all applicable covenants under the facility.

7 Royalty Financing Agreement

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 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.

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 the date the transaction closed on June 5, 2023.

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

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.

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 September 30,

Nine months ended September 30,

(in millions)

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 September 30, 2025:

During the three months ended March 31, 2026, there were no material changes to the terms of the royalty purchase agreement entered into in May 2023 (the “Royalty Financing Agreement”) with HemB SPV, L.P.

(the “Purchaser”), as described in Note 12, “ Royalty Financing Agreement ” within the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2025.

The following table presents the changes in the liability from royalty financing agreement for the period from December 31, 2025 to March 31, 2026:

Amount of liability

3 unchanged sentences

Interest expense for the period

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.

Balance as of March 31, 2026 (includes $ 3.6 million presented as "Accrued expenses and other current liabilities")

The Company records the debt at amortized cost using the effective interest rate method based on projected cash flows.

As of March 31, 2026, the effective interest rate is expected to be within a range of 10.5 % to 12.0 % per annum (March 31, 2025:

12.0 % to 13.5 %).

Interest expense recorded in connection with the agreement was $ 12.6 million and $ 13.3 million for the three months ended March 31, 2026 and 2025, respectively.

8 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 2025, the Company’s shareholders authorized an additional 2,400,000 shares 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.

As of March 31, 2026, a total of 2,571,395 ordinary shares remain available for issuance under the 2014 Plan.

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

5 unchanged sentences

2014 Plans and ESPP

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

Three months ended September 30,

Nine months ended September 30,

(in thousands)

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

Three months ended March 31,

(in thousands)

Cost of contract manufacturing revenues

Research and development expenses

1 unchanged sentence

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

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Three months ended March 31,

(in thousands)

3 unchanged sentences

Performance share units

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

As of March 31, 2026, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

Weighted average

6 unchanged sentences

Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years .

The first 25 % of each grant vests after one year from the initial grant date and the remainder vests in equal quarterly installments over years two, three and four.

The first 25 % of each grant vests one year after the initial grant date and the remainder vests in equal quarterly installments over years two, three and four.

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

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

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

Weighted average

ordinary shares

exercise price

The following table summarizes option activity under the 2014 Plans for the three months ended March 31, 2026:

Number of ordinary shares

average exercise price

Outstanding at December 31, 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 at March 31, 2026

Thereof, fully vested, and exercisable on March 31, 2026

Thereof, outstanding and expected to vest after March 31, 2026

Outstanding and expected to vest after December 31, 2025

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 September 30,

Nine months ended September 30,

Three months ended March 31,

Expected volatility

3 unchanged sentences

Expected dividend yield

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

The following table summarizes the RSU activity for the three months ended March 31, 2026:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2025

Non-vested at September 30, 2025

Non-vested at March 31, 2026

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

RSUs generally vest over one to three years .

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

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

RSUs are measured at grant date based on the market price of the Company’s ordinary shares and, except for certain grants made to non-executive directors, vest over a three-year period.

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

The Company granted PSUs to certain employees in 2024 and 2026, that, with respect to the 2024 PSUs, will be earned upon the Board of Directors’ assessment of the level of achievement of agreed upon performance targets, and, with respect to the 2026 PSUs, will be earned upon management’s assessment of the level of achievement of agreed upon performance targets, subject, in each case, to the grantee’s continued employment.

The fair value of PSUs granted in 2024 and 2026 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:

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

As of March 31, 2026, the PSUs granted in 2026 were assessed as not probable of achieving the requisite performance conditions in accordance with ASC 718, Compensation-Stock Compensation ;

therefore, no stock-based compensation expense has been recognized.

The following table summarizes the PSU activity for the three months ended March 31, 2026:

Weighted average

2 unchanged sentences

Non-vested at December 31, 2025

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.

Non-vested at March 31, 2026

During the three months ended March 31, 2026, 13,939 ordinary shares were issued under the ESPP compared to nil ordinary shares issued during the same period in 2025.

As of March 31, 2026, 72,773 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 ordinary shares as of March 31, 2025.

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 September 30,

Nine months ended September 30,

(in thousands)

Three months ended March 31,

(in thousands)

2 unchanged sentences

Professional fees

Information technology system costs

Facility expenses

4 unchanged sentences

Share-based compensation expense

Fair value loss - contingent consideration

Fair value loss - liability related to pre-funded warrants

Foreign currency gains, net

Fair value gain / (loss) - contingent consideration

Fair value gain - liability related to pre-funded warrants

Foreign currency (losses) / gains, net

Interest income

Interest expense - Royalty Financing Agreement

Interest expense - Hercules debt facility

Interest expense - Hercules loan 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.

CSL Behring collaboration

In June 2020, uniQure biopharma B.V.

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.

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:

September 30,

(in thousands)

Accounts receivable

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)

Sale of critical reagents to Genezen

Research and development grants from Dutch authorities

Sublease income

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)

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

Other investments and concentration of credit risk

The following table summarizes the Company’s other investments as of March 31, 2026 and December 31, 2025:

(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

Carrying amount of investments valued under measurement alternative

Convertible note receivable

The Company did not recognize any upward adjustments, downward adjustments, or impairments relating to investments carried under the measurement alternative during the three months ended March 31, 2026, and 2025.

In 2024, as part of the Company’s divestment of its commercial manufacturing operations to Genezen Holdings Inc.

and its subsidiary Genezen MA, Inc.

(together “Genezen”), the Company entered into a commercial supply agreement (the “Genezen CSA”), as well as a development and other manufacturing agreement, with Genezen.

The Company recorded a balance of prepaid expenses of $ 12.3 million and $ 13.9 million as of March 31, 2026 and December 31, 2025, respectively, for services that the Company expects to receive from Genezen.

In addition, as of March 31, 2026, the Company holds a convertible note receivable of $ 14.9 million which is due in September 2029 and for which Genezen is the counterparty (December 31, 2025:

$ 14.6 million).

These balances represent a concentration of credit risk, and the Company’s maximum exposure to loss is the carrying value of these assets.

No expected credit losses were recognized during the three months ended March 31, 2026 or 2025.

As of March 31, 2026 and December 31, 2025, accounts receivable from CSL Behring LLC (“CSL Behring”) totaled $ 3.6 million and $ 5.9 million, respectively, in each case related to royalty revenue.

The Company recorded a deferred tax expense of $ 0.5 million related to its U.S.

operations during the three months ended March 31, 2026 ($ 0.5 million for the three months ended March 31, 2025).

The effective income tax rate of 0.9 % during the three months ended March 31, 2026 (three months ended March 31, 2025:

1.1 %) is 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.

12 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.

Diluted loss per ordinary 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 months ended March 31, 2026 and March 31, 2025, 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 months ended March 31, 2026 and 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, 2026 and March 31, 2025, respectively.

The potentially dilutive ordinary shares are summarized below:

Three months ended September 30,

Nine months ended September 30,

Anti-dilutive ordinary share equivalents

3 unchanged sentences

Total anti-dilutive ordinary share equivalents

13 Commitments and contingencies

In the course of its business, the Company enters as a licensee into contracts with other parties regarding the development and marketing of its pipeline products.

Among other payment obligations, the Company is obligated to pay royalties to the licensors based on future sales levels and milestone payments whenever specified development, regulatory and commercial milestones are met.

As both future sales levels and the timing and achievement of milestones are uncertain, the financial effect of these agreements cannot be reliably estimated.

The Company also has obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring.

The achievement and timing of these milestones is not fixed or determinable.

As of March 31, 2026, the remaining minimum purchase commitments in connection with the Genezen CSA amount to $ 16.2 million, with $ 13.3 million to be paid within the next 12 months.

As of March 31, 2026, CSL Behring’s minimum purchase commitments to the Company over the next 12 months, in connection with the development and commercial supply agreement between the Company and CSL Behring (the “CSL Behring CSA”), amount to $ 16.4 million, of which $ 16.2 million relates to such minimum purchase commitments to Genezen.

In April 2026, the Company entered into agreements with Genezen and CSL Behring to terminate the Genezen CSA and the CSL Behring CSA.

Refer to Note 14, “ Subsequent events” .

Legal Proceedings

On February 10, 2026, a class action complaint captioned Christopher Scocco v.

uniQure N.V., et al.

1:26-cv-01124, was filed against the Company, certain of its executive officers and another party in the United States District Court for the Southern District of New York.

The complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended, and Rule 10b-5 promulgated thereunder, on behalf of a putative class of investors who purchased or otherwise acquired the Company’s ordinary shares between September 24, 2025 and October 31, 2025.

Plaintiff seeks to recover damages allegedly caused by purported false and misleading statements and omissions with respect to the Company’s Phase I/II study of AMT-130 and the timing of the potential BLA filing for AMT-130.

The Company intends to vigorously defend against the claims in this action.

At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies.

At this time, the Company is unable to predict the outcome of the class action litigation or reasonably estimate a range of possible losses.

14 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.

Termination agreements with CSL Behring and Genezen

Since the Company’s July 2024 divestiture of its commercial manufacturing activities to Genezen, the Company had been sourcing etranacogene dezaparvovec-drlb (“HEMGENIX ® ”) under the Genezen CSA.

The Company entered into the Genezen CSA to enable it to meet its obligations to supply HEMGENIX ® in accordance with the June 2020 commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”) (refer to Note 10, “ Other Investments and concentration of credit risk ,” and Note 13, “ Commitments and contingencies ”) until such time that the HEMGENIX ® manufacturing technology had been transferred to CSL Behring or a contract manufacturing organization designated by CSL Behring.

The Company, since July 2024, had served as an agent in the sale of HEMGENIX ® to CSL Behring as title to HEMGENIX ® drug product supply had been directly passing from Genezen to CSL Behring.

In April 2026, the Company entered into agreements with CSL Behring and Genezen that provide for i) the termination of its obligation to supply HEMGENIX ® and any minimum purchase commitments under the Genezen CSA, once the contractually specified batches have been supplied to CSL Behring, which the Company expects to occur in mid-2026, and ii) the designation of Genezen as CSL Behring’s contract manufacturing organization.

In addition, the CSL Behring Agreement was amended to terminate certain manufacturing-related terms associated with both the CSL Behring CSA and the Genezen CSA, as well as the Company’s development support that CSL Behring could request from time to time with respect to HEMGENIX ® .

All other terms of the CSL Behring Agreement remain in full force and effect.

The Company expects that by the time the contractually specified batches have been supplied to CSL Behring, it will i) have fully amortized the carrying amount of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA of $ 7.3 million as of March 31, 2026, and ii) have fully released the firm purchase commitment liability with a March 31, 2026 carrying amount of $ 5.8 million.

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