What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-11-10 compared with 2025-07-29 · 272 added, 236 removed, 282 unchanged (64% of the section changed)

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The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”).

Our unaudited 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.

Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the United States (“U.S.

GAAP”) and unless otherwise indicated are presented in United States (“ U.S.”) dollars.

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.

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Huntington’s disease program (AMT-130)

Clinical Data

We are conducting Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease in the U.S.

The Phase I/II AMT-130 clinical trials were supplemented by two additional protocols, each with a statistical plan that was discussed with and submitted to the United States Food and Drug Administration (the “FDA”) earlier in 2025.

The additional protocols, among other things, provide for the pooling of data across the ongoing U.S.

and European studies, and also prespecified the pivotal comparison of AMT-130 clinical endpoints compared to a propensity score-matched external control from the Enroll-HD natural history data set.

In September 2025, we announced positive topline data from the pivotal Phase I/II study of AMT-130.

The pivotal study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) at 36 months compared to a propensity score-matched external control.

The study also met a key secondary endpoint by achieving statistically significant slowing of disease progression as measured by Total Functional Capacity (“TFC”) at 36 months compared to a propensity score-matched external control.

In accordance with the prospectively defined statistical analysis plan aligned with, and submitted, to the FDA, 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 and were evaluated at that time point.

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).

Topline 36-month efficacy results for patients receiving high-dose AMT-130 were as follows (data cutoff as of June 30, 2025):

● A statistically significant 75% slowing of disease progression as measured by cUHDRS (p=0.003), which met the primary endpoint of the study.

Treated patients had a mean change in cUHDRS from baseline of -0.38 compared to a change of -1.52 for patients in the propensity score-matched external control.

● A statistically significant 60% slowing of disease progression as measured by TFC (p=0.033), which met a key secondary endpoint of the study.

Treated patients had a mean change in TFC from baseline of -0.36 compared to a change of -0.88 for patients in the propensity score-matched external control.

● Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test (“SDMT”), Stroop Word Reading Test (“SWRT”) and Total Motor Score (“TMS”).

o An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of -0.44 compared to a change of -3.73 for patients in the propensity score-matched external control.

o A 113% slowing of disease progression as measured by SWRT (nominal p=0.002), with a mean change in SWRT from baseline of 0.88 compared to a change of -6.98 for patients in the propensity score-matched external control.

o A 59% slowing of disease progression as measured by TMS (nominal p=0.174), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control.

A mean reduction from baseline in cerebrospinal neurofilament light protein (“CSF NfL”) of -8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies.

CSF NfL is a well-characterized, supportive biomarker of neurodegeneration.

Elevation in CSF NfL has been shown to be strongly associated with greater clinical severity of Huntington’s disease.

We believe that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the high-dose group, compared to the variable trends observed in the low-dose group, reflect a dose-dependent response to AMT-130.

Various other supportive analyses of the results from the AMT-130 high-dose treatment group, including those using a propensity score-weighted external control and comparisons to the TRACK-HD and PREDICT-HD datasets, were consistent with the primary analysis.

AMT-130 was generally well-tolerated in the Phase I/II studies, with a manageable safety profile at both doses with no new drug-related serious adverse events observed since December 2022.

The most common adverse events in the treatment groups were related to the administration procedure.

Data from the Phase I/II studies were presented at the 2025 Huntington’s Disease Clinical Research Congress, which took place from October 10-13, 2025, in Nashville, Tennessee.

In October 2025, we initiated and fully recruited a fourth cohort evaluating high-dose AMT-130 in six patients with lower striatal volumes compared to patients in previous cohorts.

Patient dosing in the fourth cohort is expected to be completed before year-end 2025.

Regulatory Update

In October 2025, we held a pre-Biologics License Application (“BLA”) meeting with the United States Food and Drug Administration (the “FDA”) to discuss the planned BLA submission for AMT-130.

Though final meeting minutes have not yet been received, based on the discussions at the meeting, we believe that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission.

Consequently, the timing of the BLA submission for AMT-130 is now unclear.

RMAT and Breakthrough Therapy Designations

On April 17, 2025, we announced that the U.S.

Food and Drug Administration (“FDA”) granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease.

The Breakthrough Therapy designation is supported by clinical data from the ongoing Phase I/II trials of AMT-130 for the treatment of Huntington’s disease showing the potential for meaningful slowing of disease progression.

In April 2025, we announced that the FDA granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease.

The Breakthrough Therapy designation was supported by clinical data from the ongoing Phase I/II trials of AMT-130 for the treatment of Huntington’s disease showing the potential for meaningful slowing of disease progression.

Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

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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 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.

September 2025 Public offering

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 Amendment

In September 2025, we entered into a $175.0 million senior secured term loan facility (the “2025 Amended Facility”) with Hercules Capital, Inc.

(“Hercules”).

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 9.70% as of September 30, 2025.

The tranches mature on October 1, 2030.

January 2025 Public offering

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 $10.4 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

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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 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.

In September 2025, we completed enrollment of the first three patients in the first cohort, which, following a positive independent data monitoring committee (“IDMC”) review, allowed for the expansion of the first cohort into mesial temporal lobe epilepsy in the dominant hemisphere and the initiation of the second cohort.

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 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.

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.

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

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

In February 2025, we announced a favorable recommendation from the 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.

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.

In September 2025, we announced initial safety and exploratory efficacy of the first cohort of our Phase I/IIa trial of AMT-191.

We presented the preliminary data at the International Congress of Inborn Errors of Metabolism (“ICIEM”) in Kyoto, Japan in September 2025.

As of the July 24, 2025 study cutoff date, all patients in the first dose cohort showed sustained increases in α-galactosidase A (“α-Gal A”) enzyme activity, ranging from 27- to 208-fold above the mean normal range (1.38-8.66 nmol;

mean normal of 3.57 nmol).

All four patients were withdrawn from enzyme replacement therapy and maintained stable plasma lyso-Gb3 levels through the cutoff date.

As of the July 24, 2025 study cutoff date, all patients had less than three months of follow-up.

Based on data observed to date, AMT-191 showed a manageable safety profile.

At the high dose, two Serious Adverse Events (“SAEs”) unrelated to AMT-191 (stroke, diplopia), two related SAEs (chest pain, increased troponin), and one possibly related SAE (leptomeningeal enhancement) were observed in two patients.

Additionally, one patient experienced an asymptomatic Grade 3 liver enzyme elevation that resolved with corticosteroid therapy.

This event, classified as a dose-limiting toxicity per protocol, was not considered serious and did not require hospitalization.

No loss of α-Gal A expression was observed in this patient.

A second, lower dose cohort of three patients completed enrollment and a third cohort of three patients is currently enrolling.

Amyotrophic Lateral Sclerosis (AMT-162)

EPISOD1 is a Phase I/II multi-center, open-label trial of AMT-162 for the treatment of SOD1-ALS being conducted in the United States consisting of three dose-escalating cohorts with up to four patients each receiving a short course of immunosuppression prior to and after an intrathecal infusion of AMT-162.

The trial will explore the safety and tolerability of AMT-162 and will assess exploratory signs of efficacy by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein.

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

During the third quarter of 2025, we voluntarily paused enrollment in EPISOD1 upon the recommendation of the IDMC following a September 2025 review of available preliminary data related to the safety and efficacy of AMT-162 in the context of a dose limiting toxicity, which resulted in an SAE determined to be related to AMT-162, that was observed in one patient in the second cohort.

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

Financial Overview

Key components of our results of operations include the following:

Three months ended June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

(in thousands)

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Selling, general and administrative expenses

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.

As of September 30, 2025 and December 31, 2024, we had cash and cash equivalents and investment securities of $694.2 million and $367.5 million, respectively.

We had a net loss of $80.5 million and $161.9 million in the three and nine months ended September 30, 2025, respectively, compared to a net loss of $44.4 million and $166.3 million for the same period in 2024.

As of September 30, 2025 and December 31, 2024, we had accumulated deficits of $1,291.9 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 six months ended June 30, 2025.

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

Contract manufacturing

We entered into a development and commercial supply agreement with CSL Behring LLC (“CSL Behring”) in June 2020 for the commercial supply of HEMGENIX®.

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 agreement as cost of contract manufacturing.

We entered into a development and commercial supply agreement with CSL Behring LLC (“CSL Behring”) in June 2020 for the commercial supply of etranacogene dezaparvovec-drlb (HEMGENIX®).

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

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

Research and development expenses

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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;

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● 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.

Selling, general and administrative 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 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.

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 nine months ended September 30, 2025, income from a one-time sale of critical reagents.

Following the Lexington Transaction, our expenses consist of costs incurred under our commercial supply agreement (“CSA”) with Genezen Holdings Inc.

and its affiliate Genezen MA, Inc.

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Results of Operations

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,

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

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

Three months ended September 30,

(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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Loss from operations

Non-operating income / (expense), net

Non-operating expense, net

Net loss before income tax expense

Income tax expense

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

Three months ended June 30,

Our revenues for the three months ended September 30, 2025 and 2024 were as follows:

Three months ended September 30,

(in thousands)

License revenues

Contract manufacturing revenues

Collaboration revenues

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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 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.

For the three months ended September 30, 2025 and September 30, 2024, we recognized $3.6 million and $2.1 million of license revenues, respectively.

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 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.

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

For the three months ended September, 2025 and 2024, we recognized $0.1 million and $0.2 million of collaboration revenues from CSL Behring, respectively.

Cost of contract manufacturing revenues

Cost of contract manufacturing revenues in the three months ended September 30, 2025 were nil.

In the three months ended September 30, 2024 we incurred $0.8 million in cost of contract manufacturing revenues related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.

R&D expenses for the three months ended September 30, 2025 were $34.4 million, compared to $30.6 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,

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

Three months ended September 30,

(in thousands)

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Facility expenses

Share-based compensation expense

Fair value changes related to contingent consideration

Share-based compensation expense

Severance costs

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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.

Prior to the Lexington Transaction, we supplied Good Manufacturing Practice (“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”.

Following the closing of the Lexington Transaction, we rely on third party suppliers for these activities and present the associated expenses 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.

In the three months ended September 30, 2025 and September 30, 2024, we incurred costs of $11.8 million and $4.5 million respectively.

The increase of $7.3 million includes $6.6 million of expenses incurred in relation to the preparation of the Biologics License Application submission for AMT-130 in the United States, with the remainder attributable 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.

In the three months ended September 30, 2025 and September 30, 2024, we incurred costs of $2.2 million and $1.6 million respectively, for the development of AMT-260.

We incurred costs of $2.1 million and $0.1 million related to clinical trials and Chemistry, Manufacturing, and Controls (“CMC”) development, respectively, compared to costs of $1.0 million and $0.5 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.

In the three months ended September 30, 2025 and September 30, 2024, we incurred costs of $1.7 million and $0.8 million, respectively, related to our development of AMT-191.

We incurred costs of $1.5 million and $0.3 million related to clinical trials and CMC development, respectively, compared to costs of $0.7 million and $0.1 million in the comparative period.

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

In the three months ended 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.

In the three months ended September 30, 2025 and September 30, 2024, we incurred $1.6 million and $0.9 million of expenses, respectively, to initiate a Phase I/II clinical trial in which the first patient was enrolled in October 2024.

We incurred $1.4 million and $0.2 million related to clinical trials and CMC development, respectively, compared to $0.7 million and $0.1 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.

In the three months ended September 30, 2025 and September 30 2024 , we incurred $1.4 million and $1.0 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.

● We incurred $8.6 million in personnel and contractor-related expenses in the three months ended September 30, 2025 , compared to $10.0 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.

● We incurred $3.7 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended September 30 , 2025 , compared to $3.8 million in the same period in 2024;

● We recognized a $1.8 million loss in the three months ended September 30, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to a $0.4 million gain for the same period in 2024;

● We incurred $0.7 million in share-based compensation expenses in the three months ended September 30, 2025, compared to $1.4 million for the same period in 2024;

● We incurred $0.5 million in disposables costs in the three months ended September 30 , 2025 , compared to $1.0 million for the same period in 2024;

● We incurred nil severance costs in the three months ended September 30, 2025, compared to $3.4 million related to the Lexington Transaction for the same period in 2024;

● We incurred $0.3 million of other expenses for the three months ended September 30, 2025 , compared to $2.7 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,

Selling, general and administrative expenses for the three months ended September 30, 2025 were $19.4 million, compared to $11.6 million for the same period in 2024.

Three months ended September 30,

(in thousands)

2 unchanged sentences

Share-based compensation expense

Depreciation and facility costs

Intellectual property fees

Depreciation and facility costs

Information technology costs

2 unchanged sentences

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.

● We incurred $7.1 million in personnel and contractor-related expenses in the three months ended September 30, 2025, compared to $5.5 million in the same period in 2024.

The increase was primarily the result of an increase in personnel and contractor-related expenses to support the preparation of a potential commercialization of AMT-130;

● We incurred $6.4 million in professional fees in the three months ended September 30, 2025 , compared to $1.5 million in the same period in 2024.

The increase was primarily related to professional fees incurred in relation to the preparation of a potential commercialization of AMT-130 ;

● We incurred $2.8 million in share-based compensation expenses in the three months ended September 30, 2025 , compared to $2.0 million in the same period in 2024.

The increase was primarily a result of an increase in awards granted in the three months ended September 30, 2025, in comparison to the prior period ;

● We incurred nil severance costs in the three months ended September 30, 2025, compared to $0.7 million related to the Lexington Transaction for the same period in 2024;

● We incurred $1.8 million in other expenses in the three months ended September 30 , 2025, compared to $0.7 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,

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

Three months ended September 30,

(in thousands)

Research and development grants from Dutch authorities

Gain on sale of commercial manufacturing activities

Sublease income / (expense), net

1 unchanged sentence

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.

Total other items, net

We recognized $1.6 million and $1.5 million in other expenses during the three months ended September 30, 2025 and September 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,

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

Three months ended September 30,

(in thousands)

2 unchanged sentences

Interest expense - Hercules debt facility

Foreign currency gains, net

Total non-operating expense, net

Interest expense - other

Foreign currency (losses) / gains, 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 $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 recognized $3.5 million in interest income in the three months ended September 30, 2025, compared to $4.9 million in the same period in the prior year.

Our interest income decreased by $1.4 million, primarily due to lower balances of cash and cash equivalents and investment securities during the three months ended September 30, 2025, compared to the same period in the prior year.

We recognized interest expense of $13.8 million and $12.8 million in the three months ended September 30, 2025, and September, 30, 2024, respectively, related to royalty purchase agreement (the “Royalty Financing Agreement”).

We recognized interest expense of $1.8 million and $3.8 million in the three months ended September 30, 2025, and September, 30, 2024, respectively, related to the Hercules debt facility, primarily due to the $50.0 million repayment of the Hercules debt in July 2024, as well as a decrease in market interest rates.

We recognized other interest expense of $1.1 million and nil in the three months ended September 30, 2025, and September 30, 2024, respectively.

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 .

Monetary balances are remeasured 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 loss, 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 $0.4 million during the three months ended September 30, 2025, compared to a net gain of $7.6 million during the same period in 2024.

We recognized a $5.7 million loss in the three months ended September 30, 2025, related to an increase in the fair value of the liability related to pre-funded warrants, compared to nil for the same period in 2024.

We recognized $1.5 million expenses in the three months ended September 30, 2025, related to the issuance of the Pre-Funded Warrants, compared to nil for 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 .

We recognized $1.1 million of deferred tax expense in the three months ended September 30, 2025, and $0.1 million of deferred tax benefit for the same period in 2024.

We recognized $7.5 million of current income tax expense in the three months ended September 30, 2025, and $0.1 million of current income 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,

Comparison of the nine months ended September 30, 2025 and 2024

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

Nine months ended September 30,

(in thousands)

2 unchanged sentences

Cost of license revenues

Cost of contract manufacturing

Cost of contract manufacturing revenues

Research and development expenses

3 unchanged sentences

Loss from operations

Non-operating income / (expense), net

Non-operating 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,

Our revenues for the nine months ended September 30, 2025 and 2024 were as follows:

Nine months ended September 30,

(in thousands)

5 unchanged sentences

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.

For the nine months ended September 30, 2025 and September 30, 2024, we recognized $10.4 million and $5.2 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 six months ended June 30, 2025 , compared to $6.1 million for the same period in 2024.

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

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

We recognized nil contract manufacturing revenues in the nine months ended September 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 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.

Collaboration revenues related to these contracted services are recognized when the associated performance obligations are satisfied.

For the nine months ended September 30, 2025 and September 30, 2024, we recognized $0.1 million and $10.6 million of collaboration revenues for CSL Behring, respectively.

The decrease in collaboration revenues of $10.5 million in the nine months ended September 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 revenues

We incurred nil cost of contract manufacturing revenues in the nine months ended September 30, 2025.

In the nine months ended September 30, 2024 we incurred $17.1 million cost of contract manufacturing revenues related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.

R&D expenses for the nine months ended September 30, 2025 were $105.9 million, compared to $104.9 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.

Six months ended June 30,

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

Nine months ended September 30,

(in thousands)

16 unchanged sentences

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”.

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

Huntington’s disease (AMT-130)

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.

In the nine months ended September 30, 2025 and September 30, 2024, we incurred costs of $30.6 million and $9.8 million respectively.

The increase of $20.8 million includes $14.2 million of costs incurred in relation to the preparation of the BLA submission for AMT-130 in the United States, including manufacturing process validation-related costs as well as expenses to support the initiation of the Process Performance Qualification campaign and other activities.

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

and European studies.

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.

Temporal lobe epilepsy (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.

In the nine months ended September 30, 2025 and September 30, 2024, we incurred costs of $6.3 million and $5.9 million respectively, for the development of AMT-260.

We incurred $5.4 million and $0.7 million related to clinical trials and CMC development, respectively, compared to $2.3 million and $3.3 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.

In the nine months ended September 30, 2025 and September 30, 2024, we incurred costs of $5.2 million and $3.6 million, respectively, related to our development of AMT-191.

We incurred $4.6 million and $0.4 million related to clinical trials and CMC development, respectively, compared to $3.4 million and $0.2 million in the comparative period.

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

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.

In the nine months ended September 30, 2025 and September 30, 2024, we incurred $4.6 million and $4.5 million of expenses, respectively, to initiate a Phase I/II clinical trial in which the first patient was enrolled in October 2024.

We incurred $4.2 million and $0.3 million related to clinical trials and CMC development, respectively, compared to $2.6 million and $1.8 million in the comparative period.

Preclinical programs & platform development

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.

In the nine months ended September 30, 2025 and September 30 2024 , we incurred $3.1 million and $2.3 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 $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.

● We incurred $28.2 million in personnel and contractor-related expenses in the nine months ended September 30, 2025 , compared to $39.9 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 $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.

● We incurred $12.1 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the nine months ended September 30 , 2025 , compared to $16.9 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 $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.

● We incurred $5.9 million in share-based compensation expenses in the nine months ended September 30, 2025, compared to $7.7 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 $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.

● We recognized a $5.3 million loss in the nine months ended September 30, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to a $2.0 million gain in the same period in 2024;

● We incurred $1.9 million in disposables costs in the nine months ended September 30 , 2025 , compared to $4.4 million for the same period in 2024;

● We incurred nil severance costs in the nine months ended September 30, 2025, compared to $4.5 million related to the Lexington Transaction for the same period in 2024;

● We incurred $2.8 million of other expenses for the nine months ended September 30, 2025 , compared to $7.5 million for the same period in 2024.

Selling, general and administrative expenses

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,

Selling, general and administrative expenses for the nine months ended September 30, 2025 were $43.9 million, compared to $41.3 million for the same period in 2024.

Nine months ended September 30,

(in thousands)

8 unchanged sentences

Total selling, general and administrative expenses

● 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.

● We incurred $17.9 million in personnel and contractor-related expenses in the nine months ended September 30, 2025, compared to $17.4 million in the same period in 2024;

● We incurred $11.7 million in professional fees in the nine months ended September 30, 2025 , compared to $5.7 million in the same period in 2024.

The increase was primarily related to professional fees incurred to support the preparation of a potential commercialization of AMT-130 ;

● We incurred $7.1 million in share-based compensation expenses in the nine months ended September 30, 2025 , compared to $8.8 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 $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.

● We incurred nil severance costs in the nine months ended September 30, 2025, compared to $1.0 million related to the Lexington Transaction in 2024;

● We incurred $3.8 million in other expenses in the nine months ended September 30 , 2025, compared to $4.8 million in the same period in 2024.

Other items, net

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

Six months ended June 30,

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

Nine months ended September 30,

(in thousands)

1 unchanged sentence

Research and development grants from Dutch authorities

Gain on sale of commercial manufacturing activities

Sublease income / (expense), net

1 unchanged sentence

Other income, net

Total other income, net

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.

Total other items, net

In the nine months ended September 30, 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 period in 2024.

We recognized $4.8 million and $1.5 million in other expenses during the nine months ended September 30, 2025, and September 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 six months ended June 30, 2025 and June 30, 2024 were as follows:

Six months ended June 30,

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

Nine months ended September 30,

(in thousands)

2 unchanged sentences

Interest expense - Hercules debt facility

Foreign currency gains / (losses), net

Total non-operating expense, net

Interest expense - other

Foreign currency gains, 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 $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 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 .

We recognized $11.2 million in interest income in the nine months ended September 30, 2025, compared to $17.2 million in the same period in the prior year.

Our interest income decreased by $6.0 million, primarily due to lower balances of cash and cash equivalents and investment securities during the nine months ended September 30, 2025, compared to the same period in the prior year.

We recognized interest expense of $40.9 million and $37.7 million in the nine months ended September 30, 2025 , and September, 30, 2024, respectively, related to the Royalty Financing Agreement.

We recognized interest expense of $5.4 million and $11.2 million in the nine months ended September 30, 2025 , and September, 30, 2024, respectively, related to the Hercules debt facility, primarily due to the $50.0 million repayment of the Hercules debt in July 2024, as well as a decrease in market interest rates.

We recognized other interest expense of $1.1 million and nil in the nine months ended September 30, 2025, and September 30, 2024, respectively.

We conduct transactions and hold 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 $25.4 million during the nine months ended September 30, 2025, compared to a net gain of $5.4 million during the same period in 2024 .

We recognized a $5.7 million loss in the nine months ended September 30, 2025, related to an increase in the fair value of the liability related to pre-funded warrants, compared to nil for the same period in 2024.

We recognized $1.5 million of expenses in the nine months ended September 30, 2025 related to the issuance of the Pre-Funded Warrants, compared to nil for the same period in 2024.

Income tax expense

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 .

We recognized $2.0 million of deferred tax expense in the nine months ended September 30, 2025, and $1.4 million of deferred tax expense for the same period in 2024.

We recognized $7.6 million of current income tax expense in the nine months ended September 30, 2025, and $0.2 million of current income tax expense for the same period in 2024.

Financial Position, Liquidity and Capital Resources

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 into 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 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.

As of September 30, 2025, we had cash and cash equivalents, restricted cash, and investment securities of $695.7 million.

We believe our cash and cash equivalents, restricted cash, and investment securities will be sufficient to fund our projected operating expenses into 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 potential additional expenses that might be required to obtain marketing authorizations for AMT-130.

The amount and timing of our actual expenditures may vary significantly.

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 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.

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

As of September 30, 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 $26.1 million, with $7.4 million payable within the next 12 months.

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

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

As of September 30, 2025, we had fixed lease payment obligations of $22.1 million, with $4.5 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.

As of September 30, 2025, we continue to guarantee $17.4 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 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.

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 contractually defined 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 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.

As of September 30, 2025, our remaining commitment amounts include EUR 160.0 million ($187.8 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 June 30, 2025, of $1.17/€1.00.

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

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 September 30, 2025, of $1.17/€1.00.

As of September 30, 2025, we expect these obligations will become payable between 2028 and 2033.

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

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We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our sale of the exclusive global rights of HEMGENIX® to CSL Behring.

We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.

We also owe payments to a financial advisor related to certain payments we may collect under the CSL Behring Agreement.

Commitments related to the CSA and the DMSA with Genezen

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 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.

As of September 30, 2025, our remaining minimum purchase commitments to Genezen amount to $25.2 million, with $16.5 million to be paid within the next 12 months.

CSL Behring’s minimum purchase commitments to us over the next 12 months are $17.2 million, of which $12.6 million relates to such minimum purchase commitments to Genezen.

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 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,

As of September 30, 2025 our remaining minimum purchase commitments amount to $9.8 million with $5.2 million to be paid within the next 12 months.

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

Nine months ended September 30,

(in thousands)

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Net cash generated from investing activities

Net cash generated from financing 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 Therapeutics Holding N.V.

We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics 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 continue to incur losses in the current period.

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.

We recorded a net loss of $80.5 million and $161.9 million in the three and nine months ended September 30, 2025, respectively, compared to a net loss of $44.4 million and $166.3 million during the same period in 2024.

As of September 30, 2025, we had an accumulated deficit of $1,291.9 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.

From our first institutional venture capital financing in 2006 through to the current period, we have funded our operations primarily through private and public placements of equity securities, debt securities, pre-funded warrants, and payments from our collaboration partners, as well as $370.1 million through the sale of 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 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 September 2025, we received aggregate net proceeds of $323.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, following the completion of 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 September 2025, we amended the 2024 Amended Facility with Hercules.

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 (the “Tranche 2 Advances”), 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 (the “Tranche 3 Advance”), subject to approval by Hercules.

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

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

In January 2025, we received aggregate 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 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.

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

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.

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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Net cash used in operating activities

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 was $100.4 million for the nine months ended September 30, 2025 and consisted of a net loss of $161.9 million, adjusted for non-cash items including depreciation and amortization expense of $11.8 million, amortization of the discount on investment securities of $6.1 million, share-based compensation expense of $13.0 million, $29.2 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $2.0 million, changes in the fair value of contingent consideration of $5.3 million, changes in the fair value of the liability related to pre-funded warrants of $5.7 million and unrealized foreign exchange gains of $24.5 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $28.1 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $6.8 million.

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 $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.

There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $2.9 million.

There was a net increase in accounts payable, accrued expenses, other liabilities, income taxes payable and operating leases of $25.2 million.

Net cash used in operating activities was $129.9 million for the nine months ended September 30, 2024 and consisted of a net loss of $166.3 million adjusted for non-cash items, including depreciation and amortization expense of $8.3 million, amortization of the discount on investment securities of $8.5 million, share-based compensation expense of $17.5 million, $33.1 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 $2.0 million, recognition of an allowance for inventory write-downs of $6.3 million and unrealized foreign exchange gains of $5.7 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $11.7 million.

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There was an increase in inventory balances of $3.9 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.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $2.0 million.

Net cash generated from investing activities

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 the nine months ended September 30, 2025, we generated $119.8 million from our investing activities compared to $189.6 million generated during the same period in 2024.

Nine months ended September 30,

(in thousands)

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Capital expenditures - European sites

Capital expenditures - Lexington site

Capital expenditures - Lexington sites

Divestment of commercial manufacturing facility

Net cash generated from investing activities

Net cash generated from financing activities

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 the nine months ended September 30, 2025, net cash generated from financing activities was $412.9 million, compared to net cash used in financing activities of $53.0 million in the same period in 2024.

Nine months ended September 30,

(in thousands)

Cash flows from financing activities

Proceeds from public offering of shares in connection with follow on, net of issuance costs

Proceeds from public offerings of shares from follow-ons, 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 option and purchase plans

Net cash generated from financing activities

Long-term debt repayment

Net cash generated from / (used in) financing activities

Funding requirements

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 potential commercialization of AMT-130 for Huntington’s disease in the United States, the European Union, the United Kingdom or other territories if we can align on the requirements for marketing authorization with the respective regulatory authorities;

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

● earnout payments we might owe the former shareholders of uniQure France SAS, 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;

● the scope, timing, results, and costs of our current and planned clinical trials;

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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-Q

Item 2. Management’s Discussion and Analysis