What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-07-29 compared with 2025-05-09 · 274 added, 111 removed, 244 unchanged (61% of the section changed)

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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 (“SOD1-ALS”), 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, refractory mesial temporal lobe epilepsy (“MTLE”), Fabry disease, and amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”).

Business Developments

In January 2025, we received net proceeds of $70.1 million, 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 public offering price of $17.00 per ordinary share.

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

Huntington’s disease program (AMT-130)

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In addition to the RMAT and Breakthrough Therapy designations, the FDA previously granted Orphan Drug designation and Fast Track designation to AMT-130.

In May 2025, we announced key safety observations from the third cohort of the Phase I/II study evaluating the impact of prophylactic immunosuppression in 12 patients receiving AMT-130.

AMT-130 was generally well-tolerated at both doses with no treatment-related serious adverse events.

Three serious adverse events related to immunosuppression were observed and resolved with supportive care.

In the third quarter of 2025, we expect to present 3-year follow-up data from the ongoing Phase I/II studies of AMT-130 in support of a potential Biologics License Application (“BLA”) submission.

We plan to initiate a fourth cohort in the third quarter of 2025 as part of the ongoing clinical development of AMT-130.

This open-label, single-arm U.S.

study will evaluate the safety of the high-dose administration of AMT-130 in six patients with lower striatal volumes compared to those of patients enrolled in previous cohorts.

Regulatory Alignment

In March 2025, we held a Type B meeting with the FDA to discuss chemistry, manufacturing and controls (“CMC”) requirements for AMT-130, in support of a planned Biologics License Application, (“BLA”) submission.

An additional Type B meeting focused on the pivotal statistical analysis plan was held in the second quarter of 2025.

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.

In correspondence leading up to and following the Type B meeting, FDA agreed that data from the ongoing Phase I/II studies of AMT-130, compared to a natural history external control, may serve as the primary basis for a BLA, submission under FDA’s accelerated approval pathway.

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

In correspondence leading up to and following the Type B meeting, the FDA agreed that data from the ongoing Phase I/II studies of AMT-130, compared to a natural history external control, may serve as the primary basis for a BLA, submission under the FDA’s accelerated approval pathway.

The FDA also agreed that composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain (“NfL”) measured in cerebrospinal fluid (“CSF”) may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.

In June 2025, we announced the outcomes of our April 2025 Type B meeting with the FDA to discuss the proposed use of external control data and the prospectively defined statistical analysis plan (“SAP”).

The primary efficacy analysis will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-matched external control arm derived from the ENROLL-HD dataset – a large, prospective, longitudinal, natural history study of patients with Huntington’s disease.

Certain sensitivity analyses, including one using a propensity score-weighted methodology, will also be provided.

The FDA also agreed that ENROLL-HD may be acceptable as the external control dataset for the primary analysis of the trial data along with additional sensitivity analyses using the TRACK-HD/TRACK-ON and PREDICT-HD datasets.

We simultaneously announced the outcome of our March 2025 Type B meeting with the FDA to discuss the Chemistry, Manufacturing, and Controls (“CMC”) requirements in support of our planned BLA submission for AMT-130.

The FDA agreed that validation of the AMT-130 manufacturing process should be possible using experience and prior knowledge from the etranacogene dezaparvovec-drlb (“HEMGENIX®”) process, complemented with additional full-scale AMT-130 Good Manufacturing Practice (“GMP”) batches and a single process performance qualification batch.

The FDA also agreed with our proposed drug product release testing plan, including the proposed potency assay, pending completion of qualification and specification setting activities.

In July 2025, we submitted the final SAP to the FDA.

Additionally, we initiated our Process Performance Qualification (“PPQ”) campaign to support the planned BLA submission.

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

In June 2025, we announced the appointment of Kylie O’Keefe as Chief Customer and Strategy Officer, responsible for leading all commercial and medical affairs.

O’Keefe is an accomplished business leader with broad biopharmaceutical experience.

Most recently, Ms.

O’Keefe served as Chief Commercial Officer at PTC Therapeutics and previously held key roles at LEO Pharma.

Recent Developments of other Product Candidates

Temporal lobe epilepsy program (AMT-260)

We are conducting a Phase I/IIa clinical trial (GenTLE) AMT-260 for the treatment of MTLE in the United States.

We are conducting a Phase I/IIa clinical trial (GenTLE) of AMT-260 for the treatment of MTLE in the United States.

GenTLE consists of two parts.

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The second part is expected to be a randomized, controlled trial to generate proof of concept data.

The FDA-approved study protocol provides that the first three patients to be enrolled in the study are required to have MRI-confirmed unilateral, hippocampal sclerosis.

The FDA-approved study protocol provides that the first three patients to be enrolled in the study are required to have Magnetic Resonance Imaging (“MRI”)-confirmed unilateral, hippocampal sclerosis.

In November 2024 we announced that the first patient had been dosed in GenTLE.

In addition, the FDA in February 2025 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 May 2025 we presented initial safety and exploratory efficacy data from the first treated patient showing a 92% reduction in seizure frequency observed through the first five months of follow up with no serious safety events.

Fabry disease program (AMT-191)

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The multicenter, open-label clinical trial consists of two dose-escalating cohorts of up to six adult male patients each to assess safety, tolerability, and early signs of efficacy of AMT-191 in patients with Fabry disease.

In February 2025, we announced a favorable recommendation from the independent data monitoring committee (“IDMC”) following a review of safety data from the first two patients treated in the Phase I/IIa study.

The IDMC’s review did not identify any significant safety concerns and recommended proceeding with enrollment in the second cohort.

In August 2024, we announced that the first patient has been dosed.

In September 2024, we announced the FDA granted Orphan Drug Designation to AMT-191 and in October 2024, the FDA granted Fast Track Designation to AMT-191.

The IDMC’s review did not identify any significant safety concerns and recommended proceeding with enrollment in the second cohort.

We continue to enroll patients in the Phase I/IIa study and expect to present initial safety and exploratory efficacy data at the 15th ICIEM 2025 in Kyoto, Japan on Friday, September 5, 2025.

Amyotrophic Lateral Sclerosis (AMT-162)

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In January 2025, we announced that the IDMC had recommended proceeding with enrollment of the second cohort after a review of the 28-day safety data from the first cohort.

We completed enrollment in the second dose cohort and we expect to initiate enrollment in the third dose cohort in the third quarter of 2025.

We continue to enroll patients in the Phase I/II EPISOD1 study and expect to present initial data in the first half of 2026.

Financial Overview

Key components of our results of operations include the following:

Three months ended March 31,

Three months ended June 30,

Six months ended June 30,

(in thousands)

Total revenues

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Selling, general and administrative expenses

As of March 31, 2025 and December 31, 2024, we had cash and cash equivalents and investment securities of $409.0 million and $367.5 million, respectively.

We had a net loss of $43.6 million in the three months ended March 31, 2025, compared to a net loss of $65.6 million for the same period in 2024.

As of March 31, 2025 and December 31, 2024, we had accumulated deficits of $1,173.6 million and $1,130.0 million, respectively.

As of June 30, 2025 and December 31, 2024, we had cash and cash equivalents and investment securities of $377.0 million and $367.5 million, respectively.

We had a net loss of $37.7 million and $81.4 million in the three and six months ended June 30, 2025, respectively, compared to a net loss of $56.3 million and $121.9 million for the same period in 2024.

As of June 30, 2025 and December 31, 2024, we had accumulated deficits of $1,211.3 million and $1,130.0 million, respectively.

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

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A summary of our critical accounting policies as well as a discussion of our critical accounting estimates are presented in our Annual Report .

There were no material changes to our critical accounting policies during the three months ended March 31, 2025.

There were no material changes to our critical accounting policies during the six months ended June 30, 2025.

Contract manufacturing

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In July 2024, we divested our commercial manufacturing activities located in Lexington, MA (“Lexington Transaction”).

Prior to the Lexington Transaction, we recognized contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring and the cost to manufacture HEMGENIX® under such an agreement as cost of contract manufacturing.

Research and development expenses

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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.

We anticipate that our selling, general and administrative expenses will increase in the future to support activities related to the potential commercialization of AMT-130 in the United States.

Other items, net

Our other income generally consists of payments received to subsidize our research and development efforts, income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility, and for the three months ended March 31, 2025, income from a one-time sale of critical reagents.

Our other income generally consists of payments received to subsidize our research and development efforts, income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility, and for the six months ended June 30, 2025, income from a one-time sale of critical reagents.

Following the Lexington Transaction, our other expense primarily consists of costs incurred under our commercial supply agreement (“CSA”) with Genezen Holdings Inc.

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Results of Operations

Comparison of the three months ended March 31, 2025 and 2024

The following table presents a comparison of our results of operations for the three months ended March 31, 2025 and 2024:

Three months ended March 31,

Comparison of the three months ended June 30, 2025 and 2024

The following table presents a comparison of our results of operations for the three months ended June 30, 2025 and 2024:

Three months ended June 30,

(in thousands)

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Loss from operations

Non-operating expense, net

Loss before income tax benefit

Non-operating income / (expense), net

Net loss before income tax expense

Income tax expense

Our revenue for the three months ended March 31, 2025 and 2024 was as follows:

Three months ended March 31,

Our revenue for the three months ended June 30, 2025 and 2024 was as follows:

Three months ended June 30,

(in thousands)

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We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned.

For the three months ended March 31, 2025 and March 31, 2024, we recognized $1.6 million and $1.2 million of license revenues, respectively.

For the three months ended June 30, 2025 and June 30, 2024, we recognized $5.3 million and $1.9 million of license revenues, respectively.

Contract manufacturing revenues

We recognized contract manufacturing revenues related to the manufacturing of HEMGENIX® for CSL Behring prior to the Lexington Transaction.

Contract manufacturing revenues were realized when earned upon sales of HEMGENIX® drug product to CSL Behring.

We recognized nil contract manufacturing revenues in the three months ended June 30, 2025 , compared to $2.1 million for the same period in 2024.

Collaboration revenues

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Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.

For the three months ended March 31, 2025 and 2024 we recognized nil and $3.3 million of collaboration revenue for CSL Behring, respectively.

The decrease in collaboration revenue of $3.3 million in the three months ended March 31, 2025 compared to the same period in 2024 resulted from us not providing any development and other services to CSL Behring in relation to the CSL Behring Agreement in 2025.

For the three months ended June 30, 2025 and 2024 we recognized nil and $7.1 million of collaboration revenue from CSL Behring, respectively.

The decrease in collaboration revenue of $7.1 million in the three months ended June 30, 2025 compared to the same period in 2024 was due to the absence of applicable development and other services having been provided to CSL Behring in connection with the CSL Behring Agreement in 2025.

Cost of contract manufacturing

Costs of contract manufacturing in the three months ended June 30, 2025 were nil.

In the three months ended June 30, 2024 we incurred $7.2 million costs of contract manufacturing related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.

R&D expenses for the three months ended June 30, 2025 were $35.4 million, compared to $33.7 million for the same period in 2024.

Other research and development expenses are separately classified in the table below.

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

Three months ended June 30,

(in thousands)

Huntington's disease (AMT-130)

Temporal lobe epilepsy (AMT-260)

Fabry disease (AMT-191)

Amyotrophic lateral sclerosis (AMT-162)

Programs in preclinical development and platform related expenses

Total direct research and development expenses

Employee and contractor-related expenses

Facility expenses

Share-based compensation expense

Fair value changes related to contingent consideration

Severance costs

Other expenses

Total other research and development expenses

Total research and development expenses

Direct research and development expenses

Prior to the Lexington Transaction, we supplied GMP material utilized in our clinical programs for Huntington’s disease and Fabry disease as well as GMP material needed for our preclinical programs using our in-house manufacturing facility.

The related expenses were part of “other research and development expenses”.

Following the closing of the Lexington Transaction we rely on third party suppliers and present associated expenses for these activities within “direct research and development expenses”.

Huntington’s disease (AMT-130)

In the three months ended June 30, 2025 and June 30, 2024, we incurred costs of $10.5 million and $2.9 million respectively.

The increase of $7.6 million in external cost in 2025 compared to 2024 was primarily driven by activities related to the preparation of the BLA submission for AMT-130 in the United States.

The remainder of the external costs for the development of AMT-130 incurred during the three months ended June 30, 2025 were related to the execution of our U.S.

and European studies.

Temporal lobe epilepsy (AMT-260)

In the three months ended June 30, 2025 and June 30, 2024, we incurred costs of $2.0 million and $2.0 million respectively, for the development of AMT-260.

We incurred costs of $1.7 million and $0.2 million related to clinical trials and CMC development, respectively, compared to costs of $0.8 million and $1.1 million in the comparative period.

Fabry disease (AMT-191)

In the three months ended June 30, 2025 and June 30, 2024, we incurred costs of $1.3 million and $1.5 million, respectively, related to our development of AMT-191.

We incurred costs of $1.1 million and $0.1 million related to clinical trials and CMC development, respectively, compared to costs of $1.3 million and $0.1 million in the comparative period.

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

In the three months ended June 30, 2025 and June 30, 2024, we incurred $1.1 million and $1.7 million of expenses, respectively, to initiate a Phase I/II clinical trial which enrolled the first patient in October 2024.

We incurred $1.0 million and $0.1 million related to clinical trials and CMC development, respectively, compared to $1.0 million and $0.7 million in the comparative period.

Preclinical programs & platform development

In the three months ended June 30, 2025 and June 30 2024 , we incurred $0.4 million and $0.9 million of costs, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Other research & development expenses

● We incurred $9.5 million in personnel and contractor-related expenses in the three months ended June 30, 2025 , compared to $13.0 million for the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024 as well as a reduction in expense following the August 2024 restructuring;

● We incurred $3.9 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the three months ended June 30 , 2025 , compared to $6.0 million in the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024;

● We incurred $2.8 million in share-based compensation expenses in the three months ended June 30, 2025 , compared to $2.9 million for the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;

● We recognized a $2.3 million loss in the three months ended June 30, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $1.8 million gain for the same period in 2024;

● We incurred $0.6 million in disposable costs in the three months ended June 30 , 2025 , compared to $1.4 million for the same period in 2024;

● We incurred nil of severance costs related to the Lexington Transaction in the three months ended June 30, 2025, compared to $1.1 million for the same period in 2024;

● We incurred $1.2 million of other expenses for the three months ended June 30, 2025 , compared to $2.2 million for the same period in 2024.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended June 30, 2025 were $13.5 million, compared to $15.8 million for the same period in 2024.

Three months ended June 30,

(in thousands)

Employee and contractor-related expenses

Professional fees

Share-based compensation expense

Depreciation and facility costs

Intellectual property fees

Information technology costs

Severance costs

Other expenses

Total selling, general and administrative expenses

● We incurred $5.4 million in personnel and contractor-related expenses in the three months ended June 30, 2025, compared to $5.7 million in the same period in 2024.

The decrease was primarily related to the reduction in personnel and contractors as a result of the divestment of our commercial manufacturing activities and the organizational restructuring in August 2024;

● We incurred $3.4 million in professional fees in the three months ended June 30, 2025 , compared to $4.1 million in the same period in 2024.

We regularly incur accounting, audit and legal fees;

● We incurred $2.3 million in share-based compensation expenses in the three months ended June 30, 2025 , compared to $3.3 million in the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;

● We incurred $0.3 million in intellectual property fees including registration and professional fees in the three months ended June 30, 2025 compared to $0.3 million in the same period in 2024;

● We incurred $1.3 million in other expenses in the three months ended June 30 , 2025, compared to $1.2 million in the same period in 2024.

Other items, net

Our other items, net, for the three months ended June 30, 2025 and June 30, 2024 were as follows:

Three months ended June 30,

(in thousands)

Research and development grants from Dutch authorities

Sublease income / (expense), net

Supply of HEMGENIX® to CSL Behring

Other income, net

Total other income, net

We recognized $1.7 million and nil in net other expenses during the three months ended June 30, 2025 and June 30 2024, respectively, 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.

Other non-operating items, net

Our other non-operating items, net, for the three months ended June 30, 2025 and June 30, 2024 were as follows:

Three months ended June 30,

(in thousands)

Interest income

Interest expense - Royalty Financing Agreement

Interest expense - Hercules debt facility

Foreign currency gains, net

Total non-operating expense, net

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

We recognized $3.5 million in interest income in the three months ended June 30, 2025, compared to $5.8 million in the same period in the prior year.

Our interest income decreased by $2.3 million, primarily due to lower balances of cash and cash equivalents and investment securities during the three months ended June 30, 2025, compared to the same period in the prior year.

We recognized interest expense of $1.8 million and $3.7 million in the three months ended June 30, 2025 , and June, 30, 2024, respectively, related to the Hercules Capital, Inc.

(“Hercules”) debt facility.

Interest expense decreased by $1.9 million in 2025, compared to 2024, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates.

We conduct transactions and hold monetary assets and liabilities denominated in foreign currencies, principally the euro and the U.S.

Monetary balances are re-measured at period-end exchange rates, and resulting foreign-currency transaction gains and losses are recorded in the Consolidated Statements of Operations and Comprehensive Loss as incurred.

We recognized a net foreign currency gain, related to our borrowings from Hercules, our royalty purchase agreement (the “Royalty Financing Agreement”) and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $18.6 million during the three months ended June 30, 2025, compared to a net loss of $1.0 million during the same period in 2024 .

Income tax expense

We recognized $0.4 million of deferred tax expense in the three months ended June 30, 2025 , and $0.9 million of deferred tax expense for the same period in 2024 .

Results of Operations

Comparison of the six months ended June 30, 2025 and 2024

The following table presents a comparison of our results of operations for the six months ended June 30, 2025 and 2024:

Six months ended June 30,

(in thousands)

Total revenues

Operating expenses:

Cost of license revenues

Cost of contract manufacturing

Research and development expenses

Selling, general and administrative expenses

Total operating expenses

Other expense

Loss from operations

Non-operating income / (expense), net

Loss before income tax expense

Income tax expense

Our revenue for the six months ended June 30, 2025 and 2024 was as follows:

Six months ended June 30,

(in thousands)

License revenues

Contract manufacturing revenues

Collaboration revenues

Total revenues

License revenues

We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned.

For the six months ended June 30, 2025 and June 30, 2024, we recognized $6.8 million and $3.1 million of license revenues, respectively.

Contract manufacturing revenues

We recognized contract manufacturing revenues related to the manufacturing of HEMGENIX® for CSL Behring prior to the Lexington Transaction.

Contract manufacturing revenues were realized when earned upon sales of HEMGENIX® drug product to CSL Behring.

We recognized nil contract manufacturing revenues in the three months ended March 31, 2025 , compared to $4.0 million for the same period in 2024.

We recognized nil contract manufacturing revenues in the six months ended June 30, 2025 , compared to $6.1 million for the same period in 2024.

Collaboration revenues

We provide services to CSL Behring in accordance with the CSL Behring Agreement.

Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.

For the six months ended June 30, 2025 and 2024 we recognized nil and $10.4 million of collaboration revenue for CSL Behring, respectively.

The decrease in collaboration revenue of $10.4 million in the six months ended June 30, 2025 compared to the same period in 2024 was due to the absence of applicable development and other services having been provided to CSL Behring in connection with the CSL Behring Agreement in 2025.

Cost of contract manufacturing

We incurred nil costs of contract manufacturing in the three months ended March 31, 2025.

In the three months ended March 31, 2024 we incurred $9.1 million costs of contract manufacturing related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.

R&D expenses for the three months ended March 31, 2025 were $36.1 million, compared to $40.7 million for the same period in 2024.

We incurred nil costs of contract manufacturing in the six months ended June 30, 2025.

In the six months ended June 30, 2024 we incurred $16.3 million costs of contract manufacturing related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.

R&D expenses for the six months ended June 30, 2025 were $71.5 million, compared to $74.3 million for the same period in 2024.

Other research and development expenses are separately classified in the table below.

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

Three months ended March 31,

Six months ended June 30,

(in thousands)

Huntington's disease (AMT-130)

Fabry disease (AMT-191)

Temporal lobe epilepsy (AMT-260)

Fabry disease (AMT-191)

Amyotrophic lateral sclerosis (AMT-162)

5 unchanged sentences

Fair value changes related to contingent consideration

Severance costs

Other expenses

2 unchanged sentences

Direct research and development expenses

Prior to the Lexington Transaction, we supplied Good Manufacturing Processes (“GMP”) material utilized in our clinical programs for Huntington’s disease and Fabry disease as well as GMP material needed for our preclinical programs using our in-house manufacturing facility.

The related expenses were part of “other research and development expenses”.

1 unchanged sentence

Huntington’s disease (AMT-130)

In three months ended March 31, 2025 and March 31, 2024, we incurred costs of $8.2 million and $2.4 million respectively.

In the six months ended June 30, 2025 and June 30, 2024, we incurred costs of $18.7 million and $5.3 million respectively.

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

1 unchanged sentence

The increase of $13.4 million in external cost in 2025 compared to 2024 was primarily driven by activities related to the preparation of the BLA submission for AMT-130 in the United States.

Fabry disease (AMT-191)

In the three months ended March 31, 2025 and March 31, 2024, we incurred costs of $2.2 million and $1.4 million, respectively, related to our development of AMT-19.

We incurred $2.1 million and $0.1 million related to clinical trials and CMC development, respectively, compared to $1.3 million and $0.0 million in the comparative period.

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

Temporal lobe epilepsy (AMT-260)

In three months ended March 31, 2025 and March 31, 2024, we incurred costs of $2.1 million and $2.3 million respectively, for the development of AMT-260.

In the six months ended June 30, 2025 and June 30, 2024, we incurred costs of $4.0 million and $4.3 million respectively, for the development of AMT-260.

We incurred $3.3 million and $0.7 million related to clinical trials and CMC development, respectively, compared to $1.3 million and $2.8 million in the comparative period.

Fabry disease (AMT-191)

In the six months ended June 30, 2025 and June 30, 2024, we incurred costs of $3.4 million and $2.8 million, respectively, related to our development of AMT-191.

We incurred $3.2 million and $0.2 million related to clinical trials and CMC development, respectively, compared to $2.7 million and $0.1 million in the comparative period.

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

In the three months ended March 31, 2025 and March 31, 2024, we incurred $1.8 million and $2.0 million of expenses, respectively, to initiate a Phase I/II clinical trial which enrolled the first patient in October 2024.

In the six months ended June 30, 2025 and June 30, 2024, we incurred $2.9 million and $3.6 million of expenses, respectively, to initiate a Phase I/II clinical trial which enrolled the first patient in October 2024.

We incurred $2.8 million and $0.2 million related to clinical trials and CMC development, respectively, compared to $1.9 million and $1.7 million in the comparative period.

Preclinical programs & platform development

In the three months ended March 31, 2025 and March 31, 2024 , we incurred $1.3 million and $0.4 million of costs, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

In the six months ended June 30, 2025 and June 30 2024 , we incurred $1.7 million and $1.3 million of costs, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Other research & development expenses

● We incurred $10.2 million in personnel and contractor-related expenses in the three months ended March 31, 2025 , compared to $16.9 million for the same period in 2024.

● We incurred $19.7 million in personnel and contractor-related expenses in the six months ended June 30, 2025 , compared to $30.0 million for the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024 as well as a reduction in expense from the restructuring that occurred in August 2024;

● We incurred $4.6 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the three months ended March 31, 2025 , compared to $7.1 million in the same period in 2024.

● We incurred $8.4 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the six months ended June 30 , 2025 , compared to $13.1 million in the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024;

● We incurred $2.5 million in share-based compensation expenses in the three months ended March 31, 2025 , compared to $3.4 million for the same period in 2024.

● We incurred $5.2 million in share-based compensation expenses in the six months ended June 30, 2025 , compared to $6.3 million for the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;

● We recognized a $1.2 million loss in the three months ended March 31, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $0.2 million loss for the same period in 2024;

● We incurred $0.8 million in disposable costs in the three months ended March 31, 2025 , compared to $1.9 million for the same period in 2024;

● We incurred $1.3 million of other expenses for the three months ended March 31, 2025 , compared to $2.7 million for the same period in 2024.

● We recognized a $3.5 million loss in the six months ended June 30, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $1.6 million gain for the same period in 2024;

● We incurred $1.4 million in disposable costs in the six months ended June 30 , 2025 , compared to $3.3 million for the same period in 2024;

● We incurred nil of severance costs related to the Lexington Transaction in the six months ended June 30, 2025, compared to $1.1 million for the same period in 2024;

● We incurred $2.5 million of other expenses for the six months ended June 30, 2025 , compared to $4.8 million for the same period in 2024.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended March 31, 2025 were $10.9 million, compared to $13.9 million for the same period in 2024.

Three months ended March 31,

Selling, general and administrative expenses for the six months ended June 30, 2025 were $24.4 million, compared to $29.7 million for the same period in 2024.

Six months ended June 30,

(in thousands)

Employee and contractor-related expenses

Share-based compensation expense

Professional fees

Share-based compensation expense

Depreciation and facility costs

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Information technology costs

Severance costs

Other expenses

Total selling, general and administrative expenses

● We incurred $5.5 million in personnel and contractor-related expenses in the three months ended March 31, 2025, compared to $6.2 million in the same period in 2024.

● We incurred $1.9 million in share-based compensation expenses in the three months ended March 31, 2025 , compared to $3.4 million in the same period in 2024.

● We incurred $10.9 million in personnel and contractor-related expenses in the six months ended June 30, 2025, compared to $11.9 million in the same period in 2024.

The decrease was primarily related to the reduction in personnel and contractors as a result of the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;

● We incurred $5.3 million in professional fees in the six months ended June 30, 2025 , compared to $6.1 million in the same period in 2024.

We regularly incur accounting, audit and legal fees;

● We incurred $4.3 million in share-based compensation expenses in the six months ended June 30, 2025 , compared to $6.8 million in the same period in 2024.

The decrease was primarily related to the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;

● We incurred $1.9 million in professional fees in the three months ended March 31, 2025 , compared to $2.0 million in the same period in 2024.

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

● We incurred $0.3 million in intellectual property fees including registration and professional fees in the three months ended March 31, 2025 compared to $0.6 million in the same period in 2024.

The decrease mainly related to a decrease in professional fees;

● We incurred $0.6 million in other expenses in the three months ended March 31 , 2025, compared to $1.0 million in the same period in 2024.

● We incurred $0.6 million in intellectual property fees including registration and professional fees in the six months ended June 30, 2025 compared to $0.9 million in the same period in 2024;

● We incurred nil of severance costs related to the Lexington Transaction in the six months ended June 30, 2025, compared to $0.3 million in 2024;

● We incurred $1.9 million in other expenses in the six months ended June 30 , 2025, compared to $2.2 million in the same period in 2024.

Other items, net

Our other items, net, for the three months ended March 31, 2025 and March 31, 2024 were as follows:

Three months ended March 31,

Our other items, net, for the six months ended June 30, 2025 and June 30, 2024 were as follows:

Six months ended June 30,

(in thousands)

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Research and development grants from Dutch authorities

Sublease income

Sublease income / (expense), net

Supply of HEMGENIX® to CSL Behring

Sublease expense

Other income, net

Total other income, net

In the three months ended March 31, 2025 we recognized $6.0 million in income related to a one-time sale of critical reagents to Genezen (nil in the prior period).

We recognized $1.5 million in net other expenses during the three months ended March 31, 2025, 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 six months ended June 30, 2025 we recognized $6.0 million in income related to a one-time sale of critical reagents to Genezen (nil in the prior period).

We recognized $3.3 million and nil in net other expenses during the six months ended June 30, 2025, and June 30, 2024, respectively, 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.

Other non-operating items, net

Our other non-operating items, net, for the three months ended March 31, 2025 and March 31, 2024 were as follows:

Three months ended March 31,

Our other non-operating items, net, for the six months ended June 30, 2025 and June 30, 2024 were as follows:

Six months ended June 30,

(in thousands)

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

We recognized $4.1 million in interest income in the three months ended March 31, 2025, compared to $6.5 million in the same period in the prior year.

Our interest income decreased by $2.4 million due to the interest income earned on investment securities as well as cash on hand during the three months ended March 31, 2025.

We recognized interest expense of $1.8 million and $3.7 million in the three months ended March 31, 2025 , and March, 31, 2024, respectively, related to the Hercules Capital, Inc.

(“Hercules”) debt facility.

We recognized $7.7 million in interest income in the six months ended June 30, 2025, compared to $12.3 million in the same period in the prior year.

Our interest income decreased by $4.7 million, primarily due to lower balances of cash and cash equivalents and investment securities during the six months ended June 30, 2025, compared to the same period in the prior year.

We recognized interest expense of $3.6 million and $7.4 million in the six months ended June 30, 2025 , and June, 30, 2024, respectively, related to the Hercules debt facility.

Interest expense decreased by $3.8 million in 2025, compared to 2024, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates.

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.

We recognized a net foreign currency gain, 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, of $7.2 million during the three months ended March 31, 2025, compared to a net loss of $1.1 million during the same period in 2024 .

We conduct transactions and holds monetary assets and liabilities denominated in foreign currencies, principally the euro and the U.S.

We recognized a net foreign currency gain, 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, of $25.8 million during the six months ended June 30, 2025, compared to a net loss of $2.1 million during the same period in 2024 .

Income tax expense

We recognized $0.5 million of deferred tax expense in the three months ended March 31, 2025 , and $0.7 million of deferred tax expense for the same period in 2024 .

We recognized $0.9 million of deferred tax expense in the six months ended June 30, 2025 , and $1.6 million of deferred tax expense for the same period in 2024 .

Financial Position, Liquidity and Capital Resources

As of March 31, 2025, we had cash and cash equivalents, restricted cash and investment securities of $410.4 million.

As of June 30, 2025, we had cash and cash equivalents, restricted cash and investment securities of $378.5 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, the progress of regulatory interactions, approval (if any) and commercialization efforts with respect to 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.

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

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

We expect that we will require additional funding to support the commercialization of AMT-130 in the United States and if we decide to advance any of our other clinical product candidates into late-stage clinical development.

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

As of June 30, 2025, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million.

Future interest payments and financing fees associated with the loan total $12.9 million, with $8.6 million payable within 12 months.

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

As of March 31, 2025, we had fixed lease payment obligations of $22.8 million, with $4.2 million payable within 12 months.

Following the Lexington Transaction, we assigned our lease for the Lexington Facility to Genezen.

As of March 31, 2025, we continue to guarantee $19.6 million of fixed lease payments under the lease for the Lexington Facility until the end of the lease term in May 2029.

As of June 30, 2025, we had fixed lease payment obligations of $23.2 million, with $4.4 million payable within 12 months.

Following the Lexington Transaction, we assigned our lease for our prior manufacturing facility in Lexington, MA (the “Lexington Facility”) to Genezen.

As of June 30, 2025, we continue to guarantee $18.5 million of fixed lease payments under the lease for the Lexington Facility until the end of the lease term in May 2029.

Commitments related to uniQure France SAS acquisition (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.

In connection with 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 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 March 31, 2025, our remaining commitment amounts include EUR 160.0 million ($166.2 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S.

As of June 30, 2025, our remaining commitment amounts include EUR 160.0 million ($187.9 million) in potential milestone payments associated with Phase III development and the approval 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.

These payments are owed in euro and have been translated at the foreign exchange rate as of March 31, 2025, of $1.08/€1.00.

As of March 31, 2025, we expect these obligations will become payable between 2029 and 2033.

These payments are owed in euro and have been translated at the foreign exchange rate as of June 30, 2025, of $1.17/€1.00.

As of June 30, 2025, we expect these obligations will become payable between 2029 and 2033.

If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.

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Our CSA with Genezen requires us to take or pay contract manufacturing services for HEMGENIX® during a three-year period ending July 2027.

As of March 31, 2025, our remaining minimum purchase commitments to Genezen amount to $33.1 million, with $17.0 million to be paid within the next 12 months.

As of June 30, 2025, our remaining minimum purchase commitments to Genezen amount to $25.2 million, with $13.6 million to be paid within the next 12 months.

CSL Behring’s minimum purchase commitments to us over the next 12 months are $17.5 million.

Our development and other manufacturing services agreement (“DMSA”) with Genezen requires us to take or pay contract development services during a three-year period ending July 2027.

As of March 31, 2025 our remaining minimum purchase commitments amount to $11.6 million with $4.5 million to be paid within the next 12 months.

The table below summarizes our consolidated cash flow data for the three months ended March 31, 2025 and 2024:

Three months ended March 31,

As of June 30, 2025 our remaining minimum purchase commitments amount to $9.8 million with $3.8 million to be paid within the next 12 months.

The table below summarizes our consolidated cash flow data for the six months ended June 30, 2025 and 2024:

Six months ended June 30,

(in thousands)

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We continue to incur losses in the current period.

We recorded a net loss of $43.6 million in the three months ended March 31, 2025, respectively, compared to a net loss of $65.6 million during the same period in 2024.

As of March 31, 2025, we had an accumulated deficit of $1,173.6 million.

We recorded a net loss of $37.7 million and $81.4 million in the three and six months ended June 30, 2025, respectively, compared to a net loss of $56.3 million and $121.9 million during the same period in 2024.

As of June 30, 2025, we had an accumulated deficit of $1,211.3 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 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®.

Between July 2021 and July 2023, we 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 Agreement) on net sales of HEMGENIX®.

In January 2025, we raised net proceeds of $70.1 million, 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 $10.6 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 Lexington Transaction in July 2024 we amended the debt facility again and repaid $50.0 million of the principal balance outstanding.

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

In May 2023, we amended our debt facility with Hercules (the “2023 Amended Facility”).

The 2023 Amended Facility extends the Maturity Date (as defined in the 2023 Amended Facility) and interest-only period from December 2025 to January 2027.

In connection with the Lexington Transaction in July 2024 we amended our debt facility again (the “2024 Amended Facility”) 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.

1 unchanged sentence

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.

We are subject to certain covenants under the 2024 Amended 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.

The 2024 Amended Facility permits us to issue up to $500.0 million of convertible debt.

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.

3 unchanged sentences

Net cash used in operating activities

Net cash used in operating activities was $44.1 million for the three months ended March 31, 2025 and consisted of net loss of $43.6 million adjusted for non-cash items, including depreciation and amortization expense of $4.5 million, amortization of the discount on investment securities of $2.1 million, share-based compensation expense of $4.4 million, $8.4 million of interest expense net of interest paid related to May 2023 Royalty Financing Agreement, a change in deferred taxes of $0.5 million, changes in the fair value of contingent consideration of $1.2 million, and unrealized foreign exchange gains of $7.1 million.

Net cash used in operating activities was $84.0 million for the six months ended June 30, 2025 and consisted of net loss of $81.4 million adjusted for non-cash items, including depreciation and amortization expense of $8.2 million, amortization of the discount on investment securities of $4.2 million, share-based compensation expense of $9.5 million, $20.6 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $0.9 million, changes in the fair value of contingent consideration of $3.5 million, and unrealized foreign exchange gains of $25.2 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $12.3 million.

1 unchanged sentence

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $5.6 million.

Net cash used in operating activities was $60.6 million for the three months ended March 31, 2024 and consisted of net loss of $65.6 million adjusted for non-cash items, including depreciation and amortization expense of $2.6 million, amortization of the discount on investment securities of $3.7 million, share-based compensation expense of $7.2 million, $10.9 million of interest expense net of interest paid related to May 2023 Royalty Financing Agreement, a change in deferred taxes of $0.7 million, changes in the fair value of contingent consideration of $0.2 million and unrealized foreign exchange losses of $1.4 million.

Net cash used in operating activities was $93.3 million for the six months ended June 30, 2024 and consisted of net loss of $121.9 million adjusted for non-cash items, including depreciation and amortization expense of $5.3 million, amortization of the discount on investment securities of $6.7 million, share-based compensation expense of $14.1 million, $22.2 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $1.6 million, changes in the fair value of contingent consideration of $1.6 million, a provision for inventory write-downs of $5.8 million and unrealized foreign exchange losses of $2.9 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $16.7 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $7.2 million.

There was a decrease in inventory balances of $2.2 million.

There was an increase in inventory balances of $2.6 million.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $6.8 million, primarily related to a decrease of $5.2 million from personnel-related accruals.

Net cash generated from investing activities

In the three months ended March 31, 2025, we generated $20.1 million in our investing activities compared to generating $64.0 million for the same period in 2024.

Three months ended March 31,

In the six months ended June 30, 2025, we generated $93.2 million in our investing activities compared to generating $141.9 million for the same period in 2024.

Six months ended June 30,

(in thousands)

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Net cash generated from financing activities

In the three months ended March 31, 2025, we generated $80.6 million in financing activities compared to generating nil for the same period in 2024.

Three months ended March 31,

In the six months ended June 30, 2025, we generated $80.7 million in financing activities compared to generating $0.1 million for the same period in 2024.

Six months ended June 30,

(in thousands)

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

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

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

● 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;

10 unchanged sentences

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

What changed 10-Q

Item 2. Management’s Discussion and Analysis