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Item 7. Management’s Discussion and Analysis

2026-03-02 compared with 2025-02-27 · 246 added, 294 removed, 250 unchanged (68% of the section changed)

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The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (“MD&A”) is intended to help the reader understand our results of operations and financial condition.

This MD&A is provided as a supplement to, and should be read in conjunction with, our audited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Annual Report on Form 10-K, including the disclosures under “Risk Factors.” Our consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S.

Our consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S.

GAAP”) and unless otherwise indicated are presented in U.S.

Except for the historical information contained herein, the matters discussed in this MD&A may be deemed to be forward-looking statements.

Forward-looking statement are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied.

Forward-looking statements are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied.

We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws.

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We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results.

We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1, refractory mesial temporal lobe epilepsy (“mTLE”), and Fabry disease.

We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, MTLE, Fabry disease, and SOD1-ALS.

Huntington’s disease program (AMT-130)

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a micro ribonucleic acid (“miRNA”) specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

We are currently conducting the U.S.

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a miRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

We are currently conducting U.S.

and European studies.

We completed the enrollment of all 26 patients in the first two cohorts of our US study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023.

As of February 2025, we have enrolled 12 patients into a third cohort to further investigate both doses of AMT-130 together with perioperative immunosuppression using the current, established stereotactic administration procedure.

RMAT Designation

In June 2024, we announced that the FDA granted us RMAT designation for AMT-130.

Updated Interim Data

In July 2024, we announced updated interim clinical data, including up to 24 months of follow-up data from 29 patients enrolled in the U.S.

study and the European study as of a March 31, 2024 cut-off date.

Regulatory Alignment

In December 2024, following our initial Type B meeting, we announced that we had reached agreement with FDA on key elements of an accelerated approval pathway for AMT-130.

We completed the enrollment of all 26 patients in the first two cohorts of our U.S.

study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023.

In 2025, we completed enrollment of all 12 patients in the third cohort, and we treated six patients with the high-dose of AMT-130 in a fourth cohort to evaluate the safety and efficacy of AMT-130 in patients with lower baseline striatal volumes compared to previous cohorts in the U.S.

Phase I/II study.

Clinical Data

In September 2025, we announced positive topline data from the pivotal Phase I/II study of AMT-130 in which we analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose;

n=12 low-dose) of which 12 patients per dose group had attained 36 months of follow-up as of the June 30, 2025 cutoff date.

Outcomes for each dose group were compared to a propensity score-matched external control drawn from the Enroll-HD natural history data set (n=940 for high-dose;

n=626 for low-dose).

Regulatory Updates

In October 2025, we held a pre-BLA meeting with the FDA to discuss the application for AMT-130.

In December 2025, we announced that in the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.

In January 2026, we met with the FDA at a Type A meeting to discuss AMT-130.

In March 2026, following receipt of the final meeting minutes from the Type A meeting, we announced that the FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130.

The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.

Other clinical candidates

In November 2024, we announced that the that the first patient was dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mTLE.

In addition, the FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere.

In August 2024, we announced that the first patient was dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease.

In February 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.

In October 2024, we announced the first patient was dosed in a Phase I/IIa clinical trial of AMT-162 for the treatment of SOD1-ALS.

In January 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.

Sale of commercial manufacturing activities

In June 2024, we and Genezen entered into the Lexington Transaction, which closed in July 2024.

Genezen acquired manufacturing facility including equipment and related manufacturing operations with a carrying value of $15.2 million, inventory with a carrying value of $8.8 million and certain other assets (including allocated goodwill) with a carrying value of $2.8 million associated with the Lexington Facility on Closing.

As consideration for the Lexington Transaction, we received from Genezen (i) shares of newly issued Series C preferred stock of Genezen Holdings Inc.

valued at $12.5 million, which are convertible into common stock and will accrue an 8.0% per annum cumulative dividend, (ii) a convertible promissory note with a nominal amount of $12.5 million, bearing interest at 8.0% per annum and maturing 63 months following the date of issuance valued at $13.3 million and (iii) a right to purchase HEMGENIX® at terms considered favorable to market terms (the “Consideration”) valued at $16.7 million.

We also included a firm purchase commitment liability, valued at $8.8 million for our customer contract with CSL Behring inherently related to the right to purchase HEMGENIX® in the consideration received.

We recorded a $1.2 million gain from the divestment.

We paid a total of $8.3 million to Genezen and CSL Behring related to adjustments of working capital and to obtain consent to proceed with the divestiture.

In connection with the Closing, we, Genezen and the landlord of the Lexington Facility entered into an agreement for us to assign and Genezen to assume the existing lease agreement between us and the landlord.

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

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

At the Closing, we entered into a commercial supply agreement (“CSA”) with Genezen.

Pursuant to the CSA, Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX® on our behalf.

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

Our obligations with respect to the supply of HEMGENIX® to CSL Behring remain in effect notwithstanding the subcontracting to Genezen.

Organizational restructuring

In August 2024, we announced the Restructuring to conserve capital and streamline the organization.

As a result of the Restructuring and Lexington Transaction, we eliminated approximately 300 positions or 65% of our workforce.

We substantially completed the Restructuring at the end of 2024.

We have incurred $5.2 million of employee severance costs in the year ended December 31, 2024 and estimate that we will incur additional costs in the range of $0.5 million to $1.0 million in 2025.

Royalty financing agreement

In May 2023, we 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, we received an upfront payment of $375.0 million in exchange for the Purchaser’s rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us.

We 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 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 us.

We have 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.

As of December 31, 2024 we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date.

Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date.

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

Hercules loan repayment and amendment

Upon entering into the Royalty Financing Agreement in May 2023, we and Hercules amended our existing term loan facility.

Through the May 2023 amendment we extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”).

In connection with the Closing we once more amended the facility.

As a condition to Hercules’ consent to the Lexington Transaction we prepaid $50.0 million of the total $100.0 million principal outstanding in July 2024.

The remaining $50.0 million principal outstanding will need to be repaid at the Maturity Date.

In 2025, we completed treatment of all six patients in the first cohort AMT-260.

We also started enrolling patients into the second cohort, which is expected to consist of an additional six patients.

In 2025, we completed the enrollment of three cohorts of three patients each into our Phase I/II clinical trial of AMT-191 for the treatment of Fabry disease.

We announced initial data from the first cohort at a scientific conference in September 2025 and additional preliminary data at a scientific conference in February 2026.

In September 2025, we voluntarily paused enrollment in EPISOD1, our Phase I/II clinical trial of AMT-162 for the treatment of SOD1-ALS.

We continue to collect and evaluate data from the five patients dosed in EPISOD1 to date.

Public offerings

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

In February 2025, we raised an additional $10.4 million in net proceeds upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.

In September 2025, we received net proceeds of $323.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, 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 our ordinary shares at the public offering price per share less the $0.0001 per share exercise price of each pre-funded warrant.

Hercules loan repayment and amendment

In September 2025, we entered into the 2025 Amended Facility with Hercules for a $175.0 million senior secured term loan facility.

The 2025 Amended Facility consists of three tranches including a first tranche of $50.0 million replacing the debt outstanding as per the loan amendment date, an additional term loan tranche of $100.0 million, which can be drawn at our option, subject to the BLA approval of AMT-130 prior to June 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 a third tranche of up to $25.0 million, subject to Hercules’ approval.

All tranches have a floating interest rate of the greater of 9.45% and the prime rate plus 2.45%, reflecting a floating rate of 9.45% as of December 31, 2025.

The tranches mature on October 1, 2030.

Financial Highlights

Key components of our results of operations include the following:

Year ended December 31,

Years ended December 31,

(in thousands)

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Selling, general and administrative expenses

As of December 31, 2024, we had $367.5 million in cash and cash equivalents and investment securities (December 31, 2023:

$617.9 million cash and cash equivalents and investment securities).

We had a net loss of $239.6 million in 2024 and a net loss of $308.5 million and $126.8 million in 2023 and 2022, respectively.

As of December 31, 2025, we held $622.5 million in cash and cash equivalents and investment securities (December 31, 2024:

$367.5 million).

We had a net loss of $199.0 million in the year ended December 31, 2025 and a net loss of $239.6 million and $308.5 million during the same periods in 2024 and 2023, respectively.

As of December 31, 2025, we had an accumulated deficit of $1,328.9 million (December 31, 2024:

$1,130.0 million).

See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.

Refer to “ Results of Operations ” below for a discussion of the detailed components and analysis of the amounts above.

Critical Accounting Policies and Estimates

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We also discuss our critical accounting estimates with the Audit Committee of our Board of Directors.

We consider the following to be our critical accounting estimates in the year ended December 31, 2024:

● Contingent consideration recorded in relation to the uniQure France SAS business combination;

● Valuation of the intangible asset with favorable terms recorded as part of the Lexington Transaction;

● Date at which we expect to satisfy the commitment under the Royalty Financing Agreement.

We consider the following to be our critical accounting estimate in the year ended December 31, 2025:

● Contingent consideration recorded in relation to the uniQure France business combination

Contingent consideration

In July 2021 (“Acquisition Date”), we acquired uniQure France SAS (“Acquisition Date”) and we recorded contingent consideration related to amounts potentially payable to uniQure France SAS’s former shareholders.

The amounts payable in accordance with the share and purchase agreement (“SPA”) are contingent upon realization of certain milestones associated with AMT-260.

Contingent consideration was measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.

Changes in contingent consideration can result from changes in the assumed achievement and timing of estimated milestones and the discount rate used to estimate the fair value of the liability:

● We had used discount rates ranging from 15.3% to 15.6% to calculate the contingent consideration as of December 31, 2023.

As of December 31, 2024 we used discount rates ranging from 15.3% to 16.2% to reflect increases in market interest rates.

In July 2021, in connection with the uniQure France Acquisition, we recorded contingent consideration related to amounts contingently payable to uniQure France’s former shareholders.

The amounts payable in accordance with the related share and purchase agreement are contingent upon the achievement of certain milestones associated with AMT-260.

Contingent consideration was measured at fair value as of the date of the uniQure France Acquisition, with changes in fair value subsequently recognized in the Consolidated Statements of Operations and Comprehensive Loss, within Research and development expenses.

Changes in the fair value of the contingent consideration may result from changes in the assumptions regarding the probability and timing of milestone achievement, as well as changes in the discount rate used in the valuation of the liability:

● As of December 31, 2025 we used a discount rate of 11.8% compared to a range of 15.3% to 16.2% as of December 31, 2024, reflecting changes in our credit risk and market interest rates.

An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability.

● We need to regularly update our estimate of when a milestone is expected to be achieved.

An achievement of a milestone at a later than currently expected date reduces the fair value of the contingent consideration liability whereas an achievement at an earlier than currently expected date increases the fair value of the contingent consideration liability.

● We initially recorded the contingent consideration liability on the Acquisition Date assuming a 40% probability of advancing the TLE research program into clinical development.

We developed this estimate using data from an external study regarding the average likelihood of advancing into clinical development at a certain stage or preclinical development.

For the year ended December 31, 2022, we had increased the probability to 66.0% following the commencement of toxicology studies for the TLE program.

During the year ended December 31, 2023 we increased the probability to 100.0% following the clearance of the IND application for AMT-260 in August 2023.

We did not change the probabilities of any further milestones following the dosing of the first patient in a Phase I/II clinical trial in November 2024.

The increase in probabilities resulted in a $14.2 million expense in the year ended December 31, 2023 and a $4.4 million expense in the year ended December 31, 2022.

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

● We regularly update our estimates of when milestones are expected to be achieved.

The achievement of a milestone at a later than currently expected date reduces the fair value of the contingent consideration liability, whereas achievement at an earlier than currently expected date increases the fair value of the contingent consideration liability.

● During the years ended December 31, 2025 and 2024, no changes to the probabilities of achievement of future milestones were identified.

During the year ended December 31, 2023, we increased the probability of achieving a certain development related milestone to 100.0% following the clearance of the IND application for AMT-260 in August 2023, and subsequently achieved the related milestone in 2024.

The fair value of the contingent consideration liability as of December 31, 2025 was EUR 15.9 million ($18.7 million) and as of December 31, 2024 was EUR 10.5 million ($10.9 million).

The reduction was primarily driven by a EUR 26.8 million ($28.2 million) milestone payment we made in December 2024 related to the first patient dosed in the AMT-260 clinical trial.

If as of December 31, 2024, we 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 33.7 million ($35.0 million).

The increase was driven by changes to the discount rate and expected timing of future achievement of the related remaining milestones.

If, as of December 31, 2025, the Company had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 51.4 million ($60.4 million).

If, as of December 31, 2025 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.

Intangible asset

Part of the consideration received in the Lexington Transaction included a right, with an estimated fair market value of $16.7 million, to purchase HEMGENIX® from Genezen up to the termination of our supply agreement with CSL Behring at terms considered favorable to market terms.

The determination of the fair value required us to estimate the current and future commercial supply prices of HEMGENIX® to compare these to the contractually agreed terms at Closing.

Prices for the commercial supply of HEMGENIX® are not readily observable in the market.

Therefore, our estimate of the commercial supply price was based on what a market participant would be willing to pay in a comparable transaction, using industry benchmarks, relevant market data, current and historical commercial supply agreements, as well as historical production cost information as reference points.

If the estimated commercial supply price used in the valuation had increased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received as well as the gain recorded on the divestment would have been increased by $3.0 million.

If the estimated commercial supply price used in the valuation had been decreased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received on the divestment would have been decreased by $2.9 million.

This would have resulted in a loss on divestment of $1.7 million instead of the $1.2 million gain presented.

Date at which we expect to satisfy the commitment under the Royalty Financing Agreement

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

Under the terms of the Royalty Financing Agreement we received an upfront payment of $375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us (refer to Note 13 “Royalty Financing Agreement” for further details).

Pursuant to the agreement, the total amount of royalties to be received by the Purchaser is subject to an increasing cap equal to (i) $693.8 million up to the First Hard Cap Date if such thresholds are met or, if such cap is not met by the First Hard Cap Date (ii) $843.8 million through the Second Hard Cap Date.

We have 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.

As of December 31, 2024, we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date.

Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date.

As a result of this change in estimate we expect that our total payments of interest expense will increase from $318.8 million by $150.0 million to $468.8 million over a term that was extended from 9 years to 15.5 years.

Revenue recognition related to CSL Behring variable milestone payments

We sold the exclusive global rights HEMGENIX™ to CSL Behring in 2021.

The consideration received included variable milestone payments which we recorded as revenue once probable.

We recorded $100.0 million of variable milestone revenue related to a first sale of HEMGENIX™ as license revenue in the year ended December 31, 2022 following the November 2022 BLA approval.

We collected the payment in July 2023.

We do not consider this to be a critical accounting estimate for the years ended December 31, 2024 and 2023 because we currently do not expect to recognize any regulatory milestone payments within the next 12 months.

Recently Adopted Accounting Pronouncements

In the year ended December 31, 2024, we adopted Accounting Standards Update (ASU) 2023-07, Segment Reporting (Topic 280):

Improvements to Reportable Segment Disclosures (“ASU 2023-07”).

This update requires us to disclose segment expenses that are significant and regularly provided to our chief operating decision maker (“CODM”).

In addition, ASU 2023-07 requires us to disclose the title and position of its CODM and how the CODM uses segment profit or loss information in assessing segment performance and deciding how to allocate resources.

The adoption of this update did not have a material impact on our financial position or results of operations but resulted in expanded disclosures in the segment reporting section of the financial statements.

We adopted ASU 2023-07 using a retrospective transition method.

In the year ended December 31, 2025, we adopted ASU 2023-09, Income Taxes (Topic 740):

Improvements to Income Tax Disclosures .

This update requires enhanced disaggregation and transparency of income tax disclosures, including expanded rate reconciliation and cash tax information.

The adoption of ASU 2023-09 resulted in additional income tax disclosures included in this Annual Report and did not have a material impact on our consolidated financial statements.

In November 2024, the SEC adopted final rules under Release No.

33-11275, Disaggregation of Income Statement Expenses (ASU 2024-03) .

The rules require registrants to provide enhanced disclosures regarding the disaggregation of certain operating expense categories.

The new disclosures are required on an annual basis for fiscal years beginning after December 15, 2026 for large accelerated filers, and interim period disclosures are required beginning in the first quarterly report after the year of adoption.

Early adoption is permitted.

We are currently evaluating the impact of the final rules.

There are no other recently issued accounting pronouncements pending adoption that are applicable or expected to have a material impact.

Results of Operations

The following table presents a comparison of the years ended December 31, 2025, 2024 and 2023.

Year ended December 31,

Years ended December 31,

(in thousands)

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Cost of license revenues

Cost of contract manufacturing

Cost of contract manufacturing revenues

Research and development expenses

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Non-operating expense, net

Loss before income tax benefit

Income tax (expense) / benefit

Loss before income tax expense

Income tax expense

Revenues and cost of revenues

Our revenues and associated costs for the years ended December 31, 2025, 2024 and 2023 were as follows:

Year ended December 31,

Years ended December 31,

(in thousands)

License revenues

Contract manufacturing revenues

Collaboration revenues

Contract manufacturing revenues

Total revenues

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We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of HEMGENIX have been earned.

We recognized $10.1 million, $2.8 million and $100.0 million of license revenue for the years ended December 31, 2024, 2023 and 2022, respectively.

We recognized $10.1 million and $2.8 million of license revenue in 2024 and 2023 related to royalty payments owed on HEMGENIX® sales, when earned.

We recognized $100.0 million of license revenue in 2022 related to a milestone payment owed on the first sale of HEMGENIX ® in the U.S.

We recognized $15.9 million, $10.1 million and $2.8 million of license revenue for the years ended December 31, 2025, 2024 and 2023, respectively, related to royalty payments owed on HEMGENIX sales, when earned.

In 2008, we entered into a license agreement with St.

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patent rights will expire in 2028 and the European patent rights (along with one U.S.

patent) will expire in 2025.

patent) expired in 2025.

With respect to our collaboration with CSL Behring, we have agreed with St.

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We recognized $0.2 million, $10.9 million and $2.3 million of collaboration revenue for the years ended December 31, 2025, 2024 and 2023, respectively.

The decrease in collaboration revenue in 2025 of $10.7 million compared to 2024 was primarily related to a reduction in services requested by CSL Behring following the Lexington Transaction in July 2024 .

The increase in collaboration revenue in 2024 of $8.6 million compared to 2023 was primarily related to additional development and other services provided in relation to the transfer of manufacturing responsibility to CSL Behring.

The decrease in collaboration revenue in 2023 of $0.7 million compared to 2022 was primarily related to a reduction in services requested by CSL Behring following the submissions of the BLA and MAA for HEMGENIX ® in 2022.

We provided contract manufacturing service to CSL Behring between April 2022 and July 2024.

We transferred these activities to Genezen as part of the Lexington Transaction.

We provided contract manufacturing services to CSL Behring between April 2022 and July 2024.

We transferred these activities to Genezen as part of the Lexington Transaction, which closed on July 22, 2024 (“the Closing”).

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

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We determined that we are an agent in the sale of HEMGENIX to CSL Behring.

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

We recognized contract manufacturing revenues related to contract manufacturing HEMGENIX ® for CSL Behring when earned upon sales of HEMGENIX ® to CSL Behring.

We recognized $6.1 million, $10.8 million and $1.7 million contract manufacturing revenues in the years ended December 31, 2024, 2023 and 2022, respectively.

We incurred $17.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the year ended December 31, 2024 and $13.6 million and $2.1 million of cost of contract manufacturing revenues, in the years ended December 31, 2023 and 2022 respectively.

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

We recognized contract manufacturing revenues related to the contract manufacture of HEMGENIX for CSL Behring when earned upon sales of HEMGENIX to CSL Behring.

We recognized nil, $6.1 million and $10.8 million of contract manufacturing revenues in the years ended December 31, 2025, 2024 and 2023, respectively.

We incurred nil cost of contract manufacturing revenues related to the manufacture of HEMGENIX in the year ended December 31, 2025 and $17.1 million and $13.6 million of cost of contract manufacturing revenues, in the years ended December 31, 2024 and 2023, respectively.

Costs of contract manufacturing revenues decreased in the year ended December 31, 2025 compared to the year ended December 31, 2024 as a result of the Lexington Transaction .

Costs of contract manufacturing revenues increased in the year ended December 31, 2024 compared to the year ended December 31, 2023 primarily due to expensing costs that could not be recovered from selling HEMGENIX under the terms of our development and commercial supply agreement with CSL Behring.

Costs of contract manufacturing revenues increased in the year ended December 31, 2023 compared to the year ended December 31, 2022 due to the increase in sales of HEMGENIX® and a write-down of inventory that had a cost basis in excess of its expected net realizable value.

Following the termination of the collaboration, research and license agreements and the amended agreements with Bristol-Myers Squibb (“BMS”) in February 2023, we did not recognize any further revenue for services rendered.

We recognized nil collaboration revenue for the years ended December 31, 2024 and 2023 and $1.8 million for the year ended December 31, 2022.

Res e arch and development expenses

Cost of contract manufacturing revenues included a write down of inventories to net realizable value of $6.3 million and $1.6 million for the years ended December 31, 2024 and December 31, 2023, respectively.

Research and development expenses

We expense research and development (“R&D”) expenses as incurred.

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● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies;

● changes in the fair value of liabilities recorded in relation to our acquisition of uniQure France SAS.

● changes in the fair value of liabilities recorded in relation to the uniQure France Acquisition.

Our research and development expenses primarily consist of costs incurred for the research and development of our product candidates, which include:

● AMT-130 (Huntington’s disease).

We have been incurring costs related to our U.S.

study since February 2019.

Since 2021, we have also incurred costs related to our European study;

We have incurred costs in relation to the preparation of the BLA submission for AMT-130 in the U.

S., in addition to costs related to our U.S.

study since February 2019, and our European study since 2021;

● AMT-260 (Temporal lobe epilepsy) .

We have incurred costs related to the development of AMT-260.

We have incurred costs related to the development of AMT-260 and costs to initiate and conduct a Phase I/IIa clinical trial.

We acquired this program in July 2021;

● AMT-191 (Fabry disease) .

We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease;

● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ) We have incurred costs related to the acquisition in addition to costs incurred to initiate and conduct a Phase I/II clinical trial;

We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease and costs to initiate and conduct a Phase I/II clinical trial;

● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ).

We have incurred costs related to the acquisition in addition to costs incurred to initiate and conduct a Phase I/II clinical trial;

● AMT-061 (Hemophilia B).

We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the periods ended December 31, 2025, 2024 and 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition;

2 unchanged sentences

● Technology platform development and other related research .

We incurred significant research and development costs related to manufacturing prior to the Closing of the Lexington Transaction and other enabling technologies that are applicable across all our programs.

We incurred significant research and development costs related to manufacturing prior to the closing of the Lexington Transaction and other enabling technologies that are applicable across all of our programs.

Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including regulatory submissions, and enrollment of patients in clinical trials.

The successful development of our product candidates is highly uncertain.

Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgment due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

● the scope, rate of progress and expense of our research and development activities;

2 unchanged sentences

● the timing of regulatory approvals.

A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidate.

Research and development expenses for the year ended December 31, 2025 were $140.7 million, compared to $143.8 million and $214.9 million for the years ended December 31, 2024 and 2023, respectively.

1 unchanged sentence

These are not allocated as they are deployed across multiple projects under development.

Year ended December 31,

Years ended December 31,

(in thousands)

1 unchanged sentence

Temporal lobe epilepsy (AMT-260)

Amyotrophic lateral sclerosis (AMT-162)

Fabry disease (AMT-191)

Amyotrophic lateral sclerosis (AMT-162)

Programs in preclinical development and platform related expenses

4 unchanged sentences

Share-based compensation expense

Fair value changes related to contingent consideration

Severance costs

Impairment charge

Fair value changes related to contingent consideration

Other expenses

3 unchanged sentences

Huntington disease (AMT-130)

In the years ended December 31, 2024, 2023 and 2022, we incurred $18.0 million, $13.0 million and $19.8 million expenses respectively.

In the years ended December 31, 2025, 2024 and 2023, we incurred $42.5 million, $18.0 million and $13.0 million of expenses, respectively.

Our external costs for the development of AMT-130 were primarily related to the execution of our U.S.

and European studies.

The increase of $24.5 million in external cost in 2025 compared to 2024 includes $19.4 million of costs incurred in relation to the preparation of the BLA submission for AMT-130 in the U.

S., including manufacturing process validation-related costs as well as expenses to support the process performance qualification campaign and other activities.

Other costs incurred for the development of AMT-130 were a result of treating all six patients in the fourth cohort in 2025, as well as expenses related to the execution of our U.S.

and European studies.

The increase of $5.0 million in external cost in 2024 compared to 2023 is a result of enrolling patients into a third cohort between November 2023 and December 2024 as well as expenses we incurred associated with the RMAT application and in preparation of the Type B meeting with the FDA in November 2024.

The decrease of $6.8 million in external cost in 2023 compared to 2022 is a result of the completion of U.S.

patient enrollment in March 2022.

Temporal lobe epilepsy (AMT-260)

In the years ended December 31, 2024, 2023 and 2022 we incurred $12.1 million, $13.0 million and $16.2 million expenses respectively.

In August 2023, the FDA cleared our IND application, and we started incurring costs for the preparation of a Phase I clinical trial.

In the years ended December 31, 2025, 2024 and 2023, we incurred $9.8 million, $12.1 million and $13.0 million of expenses, respectively.

In August 2023, the FDA cleared our IND application, and we began incurring costs for the preparation of a Phase I/IIa clinical trial.

In the years ended December 31, 2025, 2024 and 2023 we incurred costs of $7.5 million, $7.4 million and $3.6 million, respectively, related to our Phase I/IIa clinical trial.

In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $0.7 million, $4.3 million and $4.6 million, respectively, related to CMC development.

In the year ended December 31, 2025, we incurred $1.5 million in expenses related to a milestone payment.

Fabry disease (AMT-191)

In the years ended December 31, 2025, 2024 and 2023, we incurred $6.8 million, $4.8 million and $2.7 million of expenses, respectively, related to our development of AMT-191.

In November 2023, the FDA cleared the IND application, and we began incurring additional costs for Phase I/II clinical trial preparation.

In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $6.0 million, $4.6 million and $1.1 million, respectively, related to our ongoing Phase I/II clinical trial.

In the years ended December 31, 2025, 2024 and 2023 we incurred costs of $0.7 million, $0.2 million and $0.4 million, respectively, related to CMC development.

Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

In January 2023, we entered into a global licensing agreement with Apic Bio for AMT-162.

In the years ended December 31, 2024 and 2023, we incurred $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial in which enrolled the first patient in October 2024.

In the years ended December 31, 2025, 2024 and 2023, we incurred $6.3 million, $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial, in which we enrolled the first patient in October 2024.

In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $6.0 million, $5.2 million and $2.6 million, respectively, related to EPISOD1.

In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $0.3 million, $1.9 million and $3.3 million, respectively, related to CMC development.

During the year ended December 31, 2023, we incurred expenses of $10.0 million related to the upfront consideration paid to Apic Bio for the global licensing rights to AMT-162.

Fabry disease (AMT-191)

In the years ended December 31, 2024, 2023 and 2022 we incurred $4.8 million, $2.7 million and $2.9 million expenses, respectively, related to our development of AMT-191.

In November 2023, the FDA cleared the IND application, and we started incurring additional costs for Phase I/II clinical trial preparation.

Costs incurred in the year ended December 31, 2022 primarily related to our preclinical activities.

Preclinical programs & platform development

In the years ended December 31, 2024, 2023 and 2022 we incurred $3.9 million, $11.0 million and $7.2 million expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

In the years ended December 31, 2025, 2024 and 2023, we incurred $3.6 million, $3.9 million and $11.0 million of expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Hemophilia B (AMT-061)

We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 and 2022 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

Other research & development expenses

● We incurred $35.3 million in employee and contractor expenses in the year ended December 31, 2025 compared to $49.8 million in 2024 and $74.5 million in 2023.

Our cost decreased in 2024 by $24.7 million compared to 2023 primarily as a result of the July 2024 Lexington Transaction as well as the October 2023 and August 2024 reorganizations.

The increase in 2023 of $9.6 million, compared to 2022, primarily related to the hiring of new personnel and contractors in 2022 through mid-2023 to support our clinical-stage programs and manufacturing operations;

Our costs decreased in 2025 by $14.6 million compared to 2024 and decreased in 2024 by $24.7 million compared to 2023, primarily related to the divestment of our commercial manufacturing activities in July 2024 as well as reorganization activity in October 2023 and August 2024;

● We incurred $15.2 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the year ended December 31, 2025 compared to $21.6 million in 2024 and $29.5 million in 2023.

The decrease in 2024 compared to 2023 of $7.9 million is primarily a result of the July 2024 Lexington Transaction.

Our costs increased by $5.9 million in 2023 compared to 2022 as a result of incurring additional operating and depreciation expenses in both our Lexington and Amsterdam facilities;

The decrease in 2025 compared to 2024 of $6.5 million and the decrease in 2024 compared to 2023 of $7.9 million, is primarily related to the divestment of our commercial manufacturing activities in July 2024;

● We incurred $7.6 million in share-based compensation expenses in the year ended December 31, 2025 compared to $9.3 million in 2024 and $16.9 million in 2023.

The decrease in 2024 of $7.6 million, compared to 2023, is primarily a result of the July 2024 Lexington Transaction, the reorganizations in October 2023 and August 2024 and a decrease in the fair value of the long-term incentive awards granted.

The decrease in 2023 of $1.5 million, compared to 2022, is primarily due to the October 2023 reorganization and a decrease in the fair value of long-term incentive awards granted.

This was partially offset by cost related to performance share units granted in 2021, for which achievement of related performance conditions was deemed probable in 2023 and 2024;

● We incurred $5.1 million in disposables costs in the year ended December 31, 2024 compared to $13.2 million in 2023 and $17.8 million in 2022.

The decrease in 2024 primarily related to the July 2024 Lexington Transaction as well as the reorganizations in October 2023 and August 2024.

The decrease in 2023 compared to 2022 was related to the October 2023 reorganization;

● We incurred $4.0 million of severance costs in the year ended December 31, 2024 related to the July 2024 Lexington Transaction and the August 2024 reorganization, compared to $2.1 million of severance cost in 2023 related to the October 2023 reorganization.

No such costs were incurred in 2022;

● We incurred nil and $1.4 million of costs in the years ended December 31, 2024 and 2023, respectively, related to the impairment of the Lexington, MA facility right-of-use asset and related leasehold improvements, compared to nil in 2022;

● We recognized a $1.8 million gain in the year ended December 31, 2024 related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to losses of $15.9 million in 2023 and $7.1 million in 2022.

The increase in the 2023 loss was primarily due to an increase in the probability of making future milestone payments due upon dosing of the first patient in Phase I/II clinical trial of AMT-260 after receiving IND acceptance in August 2023;

The decrease in 2025 of $1.7 million, compared to 2024, was primarily related to the divestment of our commercial manufacturing activities in July 2024 and a reduction in expense from the restructuring that occurred in August 2024.

The decrease in 2024 of $7.6 million, compared to 2023, is primarily related to the divestment of our commercial manufacturing activities in July 2024, the reorganizations in October 2023 and August 2024, and a decrease in the fair value of granted long-term incentive awards;

● We recognized a $6.2 million loss in the year ended December 31, 2025 related to an increase in the fair value of contingent consideration associated with the uniQure France Acquisition, compared to a gain of $1.8 million in 2024 and a loss of $15.9 million in 2023.

The loss recognized in 2023 was primarily due to an increase in the probability of achievement of future milestones, upon dosing the first patient in the Phase I/IIa clinical trial of AMT-260, after receiving IND acceptance in August 2023;

● We incurred $2.6 million of disposables costs in the year ended December 31, 2025 compared to $5.1 million in 2024 and $13.2 million in 2023.

The decrease of $2.5 million in 2025 primarily related to the divestment of our commercial manufacturing activities in July 2024, as well as the reorganization in August 2024.

The decrease of $8.1 million in 2024 primarily related to the divestment of our commercial manufacturing activities in July 2024, as well as reorganization activities in October 2023 and August 2024;

● We incurred nil severance costs in the year ended December 31, 2025.

We incurred $4.0 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization, compared to $2.2 million of severance cost in 2023 related to the October 2023 reorganization;

● We incurred nil costs in the years ended December 31, 2025 and 2024, and $1.4 million of costs in the year ended December 31, 2023, related to the impairment of the right-of-use asset at the Lexington Facility and related leasehold improvements;

● We incurred $4.8 million in other expenses in the year ended December 31, 2025 compared to $9.7 million in 2024 and $6.8 million in 2023.

The decrease in costs in 2025 of $4.9 million, compared to 2024, is primarily due to a decrease in information technology expenses and consultant-related expenses.

The increase in costs in 2024 of $2.9 million, compared to 2023, is primarily a result of an increase in information technology expenses, partially offset by decreases in contractual license expenses and consultant-related expenses.

The decrease in costs in 2023 of $10.4 million, compared to 2022, is due to decreases in contractual license expenses and consultant-related expenses.

Selling, general and administrative expenses

Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses.

We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.

Our selling costs included employee expenses as well as advisory fees related to obtaining the commercialization and license agreement (“CSL Behring Agreement”) between us and CSL Behring LLC (“CSL Behring”).

Our selling, general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses.

We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations, expenses to maintain our patent and license portfolio, and fees related to business development.

As from 2025, we also incurred material expenses associated with activities to prepare for a potential launch of AMT-130 in the U.S., and present expenses related to medical affairs, new product planning and patient advocacy within selling, general and administrative expenses.

Selling, general and administrative expenses for the year ended December 31, 2025 were $65.5 million, compared to $52.7 million and $74.6 million for the years ended December 31, 2024 and 2023, respectively.

Year ended December 31,

Years ended December 31,

(in thousands)

Employee and contractor-related expenses

Share-based compensation expense

Professional fees

Depreciation and facility costs

Share-based compensation expense

Intellectual property fees

Severance costs

Depreciation and facility costs

Information technology costs

Severance costs

Financial advisory fees

1 unchanged sentence

Total selling, general and administrative expenses

● We incurred $21.3 million in personnel and contractor expenses in the year ended December 31, 2024 compared to $24.4 million in 2023 and $21.4 million in 2022.

The decrease in 2024 of $3.1 million, compared to 2023, was primarily related to the reduction in personnel and contractors as a result of the Lexington Transaction and the October 2023 and August 2024 reorganizations.

The increase in 2023 of $3.3 million, compared to 2022, was primarily related to the hiring of personnel and contractors to support our business operations;

● We incurred $11.9 million of share-based compensation expenses in the year ended December 31, 2024 compared to $17.4 million in 2023 and $15.5 million in 2022.

The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the Lexington Transaction, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of long-term incentive awards granted, partially offset by an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2024.

The increase in 2023 of $1.9 million, compared to 2022, was primarily related to an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2023.

These increases were partially offset by a decrease in expense related to a reduction in the fair value of long-term incentive awards granted;

● We incurred $24.9 million in employee and contractor-related expenses in the year ended December 31, 2025 compared to $21.3 million in 2024 and $24.4 million in 2023.

The $3.6 million increase in 2025, compared to 2024, was primarily due to higher personnel-related expenses incurred in preparation for a potential commercial launch of AMT-130 in the U.S.

The decrease in 2024 of $3.1 million, compared to 2023, was primarily related to the reduction in personnel and contractors following the divestment of our commercial manufacturing activities in July 2024, and the reorganization activities in October 2023 and August 2024;

● We incurred $16.6 million in professional fees in the year ended December 31, 2025 compared to $7.3 million in 2024 and $11.7 million in 2023.

We regularly incur accounting, audit and legal fees associated with operating as a public company.

In 2024, we incurred additional professional fees related to the Lexington Transaction.

The increase to professional fees in 2025 of $9.4 million included $6.5 million of expenses incurred in relation to the preparation of a potential commercial launch of AMT-130 in the U.S.

In 2024, we incurred additional professional fees related to the divestment of our commercial manufacturing activities.

In 2023, we incurred additional professional fees related to our global licensing agreement with Apic Bio, the Royalty Purchase Agreement and other corporate initiatives;

● We incurred $10.1 million of share-based compensation expenses in the year ended December 31, 2025 compared to $11.9 million in 2024 and $17.4 million in 2023.

The decrease in 2025 of $1.8 million was primarily related to the divestment of our commercial manufacturing activities in July 2024 and a reduction in expense from the restructuring that occurred in August 2024.

The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the divestment of our commercial manufacturing activities in July 2024, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of granted long-term incentive awards, partially offset by an increase in granted long-term incentive awards and costs associated with performance share units, which were granted in December 2021, and for which achievement of performance criteria was deemed probable in 2024;

● We incurred $1.9 million in intellectual property fees including registration and professional fees in the year ended December 31, 2025 compared to $1.7 million in 2024 and $3.8 million in 2023.

The decrease of $2.1 million in 2024, compared to 2023, is primarily due to a decrease in professional fees;

The increase of $2.3 million in 2023, compared to 2022, is mainly related to an increase in professional fees;

● We incurred $1.2 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization and $0.4 million of severance costs in 2023 related to the October 2023 reorganization.

No such costs were incurred in 2022;

● We incurred nil, $3.8 million and $1.0 million in financial advisory fees pertaining to our licensing transaction with CSL Behring in the years ended December 31, 2024, 2023 and 2022, respectively.

● We incurred nil severance costs in the year ended December 31, 2025.

We incurred $1.2 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization and $0.4 million of severance costs in 2023 related to an October 2023 reorganization;

● We incurred nil financial advisory fees pertaining to our licensing transaction with CSL Behring in the years ended December 31, 2025 and 2024 and $3.8 million in the year ended December 31, 2023.

● We incurred $8.8 million in other operating expenses in the year ended December 31, 2025 compared to $5.9 million in 2024 and $4.6 million in 2023.

The increase in costs in 2025 of $2.8 million, compared 2024, was primarily due to an increase in other tax expenses recorded in 2025, partially offset by a decrease in other expenses relating to the Lexington Transaction in 2024.

Other items, net

In year ended December 31, 2024, we recognized a $1.2 million net gain related to the Lexington Transaction, with no such amounts recognized in 2023 and 2022.

We recognized $3.2 million in net other expenses during the year ended December 31, 2024 (nil in 2023 and 2022) related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.

In the year ended December 31, 2025, we recognized $6.0 million in income related to a one-time sale of critical reagents to Genezen, compared to nil in the same periods in 2024 and 2023.

We recognized nil income related to our equity stake in VectorY B.V.

in the year ended December 31, 2024.

in the years ended December 31, 2025 and 2024.

In 2023, we recognized $0.8 million in other expense related to a reduction in the fair market value of our equity stake in VectorY B.V.

following an October 2023 financing round.

We recognized other income of $0.3 million related to the equity stake in 2022.

In 2024, we recognized $5.6 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands and France compared to $5.3 million in 2023 and $6.1 million in 2022.

In the year ended December 31, 2025, we recognized $5.7 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands and France compared to $5.6 million in 2024 and $5.3 million in 2023.

In the year ended December 31, 2024, we recognized a $1.2 million net gain related to the Lexington Transaction, with no such amounts applicable in either 2025 or 2023.

Other income for the years ended December 31, 2025, 2024, and 2023 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility.

We present expenses related to such income as other expense.

Other non-operating items, net

We recognized $6.3 million and $3.2 million in net other expenses during the year ended December 31, 2025 and 2024 and nil in 2023, related to the purchase of HEMGENIX from Genezen, net of income from the sales of HEMGENIX to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.

Non-operating items, net

Our non-operating items, net, for the years ended December 31, 2025, 2024, and 2023 were as follows:

Year ended December 31,

Years ended December 31,

(in thousands)

2 unchanged sentences

Interest expense - Hercules debt facility

Foreign currency (losses) / gains, net

Other non-operating gains

Total non-operating (expense) / income, net

Interest expense - other

Foreign currency gains / (losses), net

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

Issuance costs - Pre-funded warrants

Total non-operating items, net

We recognize interest income associated with our cash and cash equivalents and investment securities.

We recognized interest income of $17.0 million in 2025, $21.4 million in 2024 and $19.6 million in 2023.

Our interest income increased in 2024 by $1.9 million compared to 2023 primarily due to the interest income earned on investment securities and cash on hand.

Interest income increased by $19.0 million in 2023, compared to 2022, as a result of receiving $370.1 million of net proceeds from the May 2023 royalty financing agreement and a $100.0 million milestone payment from CSL Behring in July 2023.

We recognized non-cash interest expenses related to the May 2023 royalty financing agreement of $50.9 million in 2024, $26.9 million in 2023 and nil in 2022.

We recognized interest expense of $12.9 million in 2024, $14.6 million in 2023 and $11.7 million in 2022 related to the Hercules debt facility.

Our interest income decreased in 2025 by $4.4 million compared to 2024, and increased in 2024 by $1.9 million compared to 2023, primarily due to market interest rate developments and fluctuating balances of investment securities and cash on hand year over year.

In May 2023, uniQure biopharma entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P.

to sell certain current and future royalties due to uniQure biopharma from CSL Behring under the CSL Behring Agreement by and between uniQure biopharma and CSL Behring from the net sales of HEMGENIX.

We recognized non-cash interest expenses related to the Royalty Financing Agreement of $54.1 million in 2025, $50.9 million in 2024 and $26.9 million in 2023, respectively.

We recognized interest expenses of $6.9 million in 2025, $12.9 million in 2024 and $14.6 million in 2023 related to the Hercules debt facility.

Interest expenses decreased by $6.0 million in 2025 compared to 2024, primarily due to the $50.0 million repayment of the Hercules debt in July 2024, as well as a decrease in market interest rates.

Interest expense decreased by $1.7 million in 2024, compared to 2023, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates.

Interest expense increased by $2.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt.

We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S.

We recognize foreign exchange results related to changes in these foreign currencies.

In 2024, we recognized a net foreign currency loss of $10.5 million related to our borrowings from Hercules, the May 2023 royalty financing agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group.

We recognized a net loss of $1.7 million in 2023 and a net gain of $23.2 million in 2022.

In 2024 and 2023, we recognized no Other non-operating gains.

In 2022, we recognized a $2.8 million net gain related to a decrease in the fair value market value of a derivative financial liability

We recognized $2.3 million of deferred tax expense in 2024 compared to $1.9 million of deferred tax expense in 2023 and $1.5 million of deferred tax income in 2022.

In 2024 and 2023 deferred tax expense recorded in the U.S., related to the consumption of net operating losses.

From 2022 up to mid-2023 we recognized deferred tax income in France as a result of the buildup of net operating losses by the French entity.

The deferred tax income in France in 2024 is almost fully offset with an increase in valuation allowance.

In 2025, we recognized a net foreign currency gain of $26.1 million related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group.

We recognized a net foreign currency loss of $10.5 million in 2024 and $1.7 million in 2023.

We recognized a $10.9 million gain in 2025, net of issuance costs, related to a decrease in the fair value of the liability related to pre-funded warrants, compared to nil for the same periods in 2024 and 2023.

Income tax expense

We recognized $5.6 million of income tax expense in 2025 compared to $2.4 million of income tax expense in 2024 and $1.9 million of income tax expense in 2023.

The $3.2 million increase of income tax expense in 2025 compared to 2024 is primarily attributable to the recognition of $3.9 million of current income tax expense related to the agreement reached with the Dutch tax authorities in 2025, to treat the $375.0 million upfront payment received in 2023 under the Royalty Financing Agreement as taxable income in that year.

The income tax recorded in 2024 and 2023 primarily related to the consumption of net operating losses in the U.S.

Until mid-2023, we also recognized deferred tax income in France related to the buildup of net operating losses by our French entity.

Financial Position, Liquidity and Capital Resources

As of December 31, 2025, we had cash and cash equivalents, restricted cash and investment securities of $624.1 million.

Until such time, if ever, as we can generate substantial cash flows from successfully commercializing our proprietary product candidates, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution, and licensing arrangements.

Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe that our cash and cash equivalents and investment securities will fund our operations through the second half of 2027.

We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

The amount and timing of our actual expenditures may vary significantly depending on numerous factors, including the timing of a potential BLA submission, approval and commercialization of AMT-130.

We expect that we will require additional funding if we decide to advance any of our other clinical product candidates into late-stage clinical development.

We believe our cash and cash equivalents, restricted cash, and investment securities will be sufficient to fund our projected operating expenses into the second half of 2029.

Such operating expenses include expenses to fund the ongoing clinical trials of AMT-130, AMT-162, AMT-191 and AMT-260, as well as the potential investment of a material portion of the proceeds from our September 2025 public follow-on offering primarily into advancing certain of our clinical candidates into late state development.

The amount and timing of our actual expenditures may vary significantly depending on when we commence, as well as the design of, any Phase III trials for AMT-130, AMT-191, and AMT-260 that we may conduct.

We have based our estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, and as such, we may require additional funding.

If the BLA for AMT-130 is approved 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, we could draw down up to $100.0 million under our senior secured term loan facility with Hercules.

Additional funding could also include a combination of public equity offerings, collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible.

If adequate funds are not available to us on acceptable terms when we need them, we may be unable to pursue further development of our clinical product candidates.

Our material cash requirements include the following contractual and other obligations:

As of December 31, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc.

(“Hercules”) for an aggregate principal amount of $50.0 million.

In July 2024, in connection with the Closing of the Lexington Transaction, we repaid $50.0 million of the $100.0 million of principal outstanding as well as $3.1 million in end-of-term fees.

Future interest payments and financing fees associated with the loan total $16.0 million, with $8.6 million payable within 12 months.

We are contractually required to repay the $50.0 million principal outstanding in full in January 2027.

As of December 31, 2025, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million.

The loan has an interest-only period until October 1, 2028 and we are contractually required to repay the $50.0 million in equal installments between October 1, 2028 and October 1, 2030.

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 loan will be interest-only until October 1, 2030 if certain commercial milestones are met prior to December 31, 2028.

Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $21.9 million, with $4.8 million payable within the next 12 months.

We entered into lease arrangements for facilities, including corporate, laboratory and office space.

As of December 31, 2024, we had fixed lease payment obligations of $23.0 million, with $4.1 million payable within 12 months.

Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen.

This reduced our fixed lease payment obligations by $20.7 million.

We continue to guarantee such payments until the end of the lease term in May 2029.

Commitments related to acquisition of uniQure France SAS (nominal amounts)

In relation to our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractual milestones.

The commitments include payments related to post-acquisition services that we agreed to as part of the transaction.

In September 2023, we made a payment of EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS following the FDA’s clearance of the IND application for AMT-260.

In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS following the dosing of the first patient in Phase I/II clinical trial for AMT-260.

As of December 31, 2024, our remaining commitment amounts include a EUR 160.0 million ($166.2 million) in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the U.S.

and European Union.

The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestone will be achieved at all, is generally uncertain.

As of December 31, 2025, we had fixed lease payment obligations of $21.7 million, with $4.6 million payable within the next 12 months.

Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen, which reduced our fixed lease payment obligations by $20.7 million.

As of December 31, 2025, we remained obligated under a guarantee of lease payments of $16.3 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.

Commitments related to the uniQure France Acquisition (nominal amounts)

In connection with the uniQure France Acquisition, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractually defined milestones.

The commitments include payments related to post-acquisition services that we agreed as part of the transaction.

In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France following the dosing of the first patient in Phase I/IIa clinical trial for AMT-260.

As of December 31, 2025, our remaining commitment amounts include EUR 160.0 million ($188.0 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S.

The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestones will be achieved at all, is generally uncertain.

These payments are owed in euro and have been translated at the foreign exchange rate as of December 31, 2025, of $1.18/€1.00.

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As of December 31, 2025, our remaining minimum purchase commitments to Genezen amount to $16.2 million, with $10.3 million to be paid within the next 12 months.

CSL Behring’s minimum purchase commitments to us over the next 12 months are $13.6 million.

CSL Behring’s minimum purchase commitments to us over the next 12 months are $16.4 million, of which $16.2 million relates to such minimum purchase commitments to Genezen.

Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 2027.

As of December 31, 2025 our remaining minimum purchase commitments amount to $7.3 million with $4.3 million to be paid within the next 12 months.

The table below summarizes our consolidated cash flow data for the years ended December 31:

The table below summarizes our consolidated cash flow data for the years ended December 31, 2025, 2024, and 2023

Year ended December 31,

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Net cash used in operating activities

Net cash generated from / (used in) investing activities

Net cash (used in) / generated from financing activities

Net cash (used in) / generated from investing activities

Net cash generated from / (used in) financing activities

Foreign exchange impact

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

We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V.

in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement.

We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V.

in 1998, with the exception of our generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement.

We continued to incur losses in the current period.

We recorded a net loss of $239.6 million for the year ended December 31, 2024, and net loss of $308.5 million in 2023, and a net loss of $126.8 million in 2022.

We recorded a net loss of $199.0 million for the year ended December 31, 2025, and a net loss of $239.6 million and $308.5 million in the years ended December 31, 2024 and 2023, respectively.

As of December 31, 2025, we had an accumulated deficit of $1,328.9 million.

Sources of liquidity

From our first institutional venture capital financing in 2006 through the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023.

Between July 2021 and July 2023, we have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL and other milestones collected from CSL, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX®.

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

On May 12, 2023 we and Hercules amended our debt facility.

The amendment extends the Maturity Date and interest-only period from December 2025 to January 2027.

In connection with the Closing of the Lexington Transaction in July 2024 we amended the debt facility again and repaid $50.0 million of the principal balance outstanding.

We are required to repay the remaining principal balance of $50.0 million 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.

We owe 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.

We are subject to certain covenants under the debt facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.

In addition, our pledge of assets as collateral to secure our obligations under the 2024 Amended Facility may limit our ability to obtain debt financing.

The debt facility permits us to issue up to $500.0 million of convertible debt.

From our first institutional venture capital financing in 2006 through to the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners, as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023 in connection with the Royalty Financing Transaction.

Between July 2021 and July 2023, we have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX to CSL Behring and other milestones collected from CSL Behring, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX.

In 2025, we received aggregate net proceeds of $404.2 million, after deducting underwriting discounts and commissions and other offering expenses, through the completion of follow-on public offerings.

In September 2025, we received aggregate net proceeds of $323.7 million in connection with 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 our ordinary shares at the public offering price per share less the $0.0001 per share exercise price for each pre-funded warrant.

In January and February 2025, we received aggregate net proceeds of $80.5 million, in connection with a follow-on public offering of 5.1 million ordinary shares at a public offering price of $17.00 per ordinary share.

We have amended the venture debt loan facility entered into with Hercules in June 2013 from time to time, most recently in September 2025.

Pursuant to the 2025 Amended Facility, we may draw down (i) an additional $100.0 million in two increments of at least $25.0 million, following the approval of a 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, subject to approval by Hercules.

We are subject to certain covenants under the senior secured term loan facility with Hercules and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.

In addition, our pledge of assets as collateral to secure our obligations under the senior secured term loan facility with Hercules may limit our ability to obtain debt financing.

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

As of December 31, 2025, no sales have been made under the ATM offering program.

To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all.

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Royalty financing agreement interest expense, net of interest paid

Deferred tax expense / (income)

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

Unrealized foreign exchange losses / (gains), net

Deferred tax expense

Change in fair value of contingent consideration

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

Unrealized foreign exchange (gains) / losses, net

Other items, net

Changes in operating assets and liabilities:

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

Contract asset related to CSL Behring milestone payments

Accounts payable

Accrued expenses, other liabilities, and operating leases

Contingent consideration milestone payment

Contingent consideration milestone payments

Contract asset related to CSL Behring milestone payment

Net cash used in operating activities

Net cash used in operating activities was $182.7 million for the year ended December 31, 2024, and consisted of a net loss of $239.6 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $12.6 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $22.3 million, $44.2 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $2.4 million, $1.8 million change in the fair value of contingent consideration and unrealized foreign exchange losses of $15.4 million.

Net cash used in operating activities was $178.0 million for the year ended December 31, 2025 and consisted of a net loss of $199.0 million, adjusted for non-cash items including depreciation and amortization expense of $14.8 million, amortization of the discount on investment securities of $10.5 million, share-based compensation expense of $17.7 million, $38.8 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $1.6 million, changes in the fair value of contingent consideration of $6.2 million, changes in the fair value of the liability related to pre-funded warrants of $12.4 million and unrealized foreign exchange gains of $23.0 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $6.8 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $8.6 million and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $1.8 million.

Net cash used in operating activities was $182.7 million for the year ended December 31, 2024, and consisted of a net loss of $239.6 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $12.6 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $22.3 million, $44.2 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $2.4 million, $1.8 million change in the fair value of contingent consideration and unrealized foreign exchange losses of $15.4 million.

Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.2 million.

There was an decrease in inventory balances of $2.4 million.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to a increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals.

Net cash used in operating activities also includes a payment for a contingent consideration milestone of $19.6 million.

Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.

There was a decrease in inventory balances of $2.4 million.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to an increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals.

Net cash used in operating activities also includes a payment for a contingent consideration payment of $19.6 million.

Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.

Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $80.5 million.

4 unchanged sentences

Net cash used in operating activities also includes a payment for a contingent consideration milestone of $1.9 million.

Net cash used in operating activities was $145.1 million for the year ended December 31, 2022, and consisted of a net loss of $126.8 million adjusted for non-cash items, including depreciation and amortization expense of $8.5 million, share-based compensation expense of $34.2 million, changes in the fair value of contingent consideration and the derivative financial liability of $4.3 million, unrealized foreign exchange gains of $22.1 million and a change in deferred taxes of $1.5 million.

Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $43.4 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $4.1 million.

There was a net increase in contract assets related to CSL Behring milestone payments of $45.0 million.

The net increase related to $100.0 million recognized as a contract asset in the current period and collection of $55.0 million of the contract asset related to the CSL milestones of $55.0 million in March 2022 and April 2022.

There was an increase in inventories of $6.9 million related to the production of HEMGENIX ® under the CSL Behring Agreement.

These changes also relate to a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $12.6 million, primarily related to an increase in accounts payable.

Net cash generated from / (used in) investing activities

In 2024, we generated $163.0 million from our investing activities compared to using $205.7 million in 2023 and $182.7 million in 2022.

Net cash (used in) / generated from investing activities

In 2025, we used $321.6 million in our investing activities, compared to $163.0 million generated from investing activities in 2024 and $205.7 million used in investing activities in 2023.

Year ended December 31,

(in thousands)

Cash flows from investing activities

Proceeds from maturity of debt securities

Investment in debt securities

Divestment of commercial manufacturing facility

Capital expenditures - European sites

Capital expenditures - Lexington site

Acquisition of uniQure France SAS, net of cash acquired

Net cash generated from / (used in) investing activities

Net cash (used in) / generated from financing activities

Capital expenditures - Lexington sites

Divestment of commercial manufacturing facility

Net cash (used in) / generated from investing activities

Net cash generated from / (used in) financing activities

In 2025, we generated $415.4 million from our financing activities compared to $59.5 million used in financing activities in 2024 and $362.7 million generated from financing activities in 2023.

Year ended December 31,

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Cash flows from financing activities

Proceeds from Royalty Financing Agreement, net of debt issuance costs

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

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

Contingent consideration milestone payment

Net cash (used in) / generated from financing activities

In December 2024, following the dosing of the first patient in Phase I/II clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS based on contractually defined milestones.

Contingent consideration milestone payments

Proceeds from royalty financing agreement, net of issuance costs

Net cash generated from / (used in) financing activities

In December 2024, following the dosing of the first patient in Phase I/IIa clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France based on contractually defined milestones.

EUR 26.8 million ($28.2 million) of this payment related to a contingent consideration of which EUR 8.2 million ($8.6 million) was classified as cash flows from financing activities and EUR 18.7 million ($19.6 million) was classified as a net cash flow used in operating activities.

In September 2023, following the FDA’s clearance of the IND application for AMT-260, we made a payment of EUR 10.0 million ($10.7 million) to the former shareholders of uniQure France SAS based on contractually defined milestones.

EUR 8.9 million ($9.6 million) of this payment related to a contingent consideration of which EUR 7.2 million ($7.6 million) was classified as cash flows from financing activities and EUR 1.8 million ($1.9 million) was classified as a net cash flow used in operating activities.

1 unchanged sentence

Our future capital requirements will depend on many factors, including but not limited to:

● activities related to submission of a BLA for AMT-130 in Huntington’s disease;

● activities to prepare for the commercialization of AMT-130 in Huntington’s disease in the United States;

● investments to launch AMT-130 in Huntington’s disease in the United States including the amounts of revenue we generate following the launch;

● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones;

● Investments to develop AMT-130, AMT-191 and AMT-260 beyond completing the ongoing trials;

● activities to prepare for the potential commercialization of AMT-130 for Huntington’s disease, if we can align on the requirements for marketing authorization with the respective regulatory authorities;

● earnout payments we might owe the former shareholders of uniQure France, which are subject to the achievement of specific development and regulatory milestones;

● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;

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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-K

Item 7. Management’s Discussion and Analysis