What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-07-29 compared with 2026-05-05 · 243 added, 116 removed, 229 unchanged (61% of the section changed)

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We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, refractory mesial temporal lobe epilepsy (“MTLE”), and Fabry disease.

Business Developments

In June 2026, we received net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 5.7 million ordinary shares, at a public offering price of $45.50 per ordinary share.

Hercules Loan Amendment

In July 2026, we entered into an amendment to the $175.0 million senior secured term loan facility (the “2026 Amended Facility”) with Hercules Capital, Inc.

(“Hercules”).

The 2026 Amended Facility, among other things, extends the period we can draw the $100.0 million term loan tranche from June 2027 to September 2027.

Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility otherwise remain unchanged.

Recent Product Candidate Developments

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Data from Phase I/II Clinical Studies

In September 2025, we announced positive topline data from the three-year analysis of the ongoing Phase I/II studies of AMT-130 for the treatment of Huntington’s disease.

In September 2025, we announced positive topline data from the three-year analysis of cohorts one and two of the ongoing Phase I/II studies of AMT-130 for the treatment of Huntington’s disease.

We analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose;

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AMT-130 was generally well-tolerated in the Phase I/II studies, with a manageable safety profile at both doses.

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

There have been five drug-related serious adverse events (“SAEs”) reported across all cohorts, and the most common adverse events in the treatment groups were related to the administration procedure.

Regulatory Update

From November 2024 through April 2025, we held three Type B meetings with the U.S.

Food and Drug Administration (“FDA”).

Food and Drug Administration (the “FDA”) .

As part of these interactions, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis of a Biologics License Application (“ BLA”) submission under the FDA’s accelerated approval pathway.

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

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

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

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

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

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

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

We intend to continue engaging with the FDA and have scheduled a Type B meeting with the agency in the second quarter of 2026.

In April 2026, we announced that, following feedback from a pre-submission meeting with the United Kingdom’s (“UK”) Medicines and Healthcare products Regulatory Agency (“MHRA”) regarding AMT-130, we plan to submit a UK Marketing Authorization Application for AMT-130 in the third quarter of 2026.

In March 2026, we held a successful pre-submission meeting with the United Kingdom’s (“UK”) Medicines and Healthcare products Regulatory Agency (“MHRA”) regarding AMT-130, and the regulatory submission is progressing as planned for the third quarter of 2026.

In June 2026, we held a Type B meeting with the FDA.

Official meeting minutes received in July 2026 confirmed that we and the FDA reached alignment that a BLA submission under the accelerated approval pathway for AMT-130, based on the existing clinical data, is reasonable.

In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of a randomized standard-of-care control design instead of a sham procedure.

The FDA also stated that, in accordance with the FDA’s draft public guidance for accelerated approvals, the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway, and potentially fully enrolled, at the time of accelerated approval.

Discussions with the FDA to align on the confirmatory study design and analysis are underway, and we expect to submit a BLA in the third quarter of 2026.

Temporal lobe epilepsy program (AMT-260)

We are conducting a Phase I/IIa clinical trial, GenTLE, of AMT-260 for the treatment of MTLE in the U.S.

GenTLE consists of two parts.

The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and initial efficacy of AMT-260 in patients with refractory MTLE.

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

GenTLE is a multicenter, open-label trial with two dosing cohorts of at least six patients each to assess safety, tolerability, and initial efficacy of AMT-260 in patients with refractory MTLE.

In September 2025, we completed enrollment of the first three patients in the first cohort administering AMT-260 to patients with lesions in the non-dominant hemisphere of the brain.

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We completed enrollment of six patients into the first cohort in 2025.

We also initiated enrollment of a second cohort in 2025, which is expected to include an additional six patients.

We also initiated enrollment of a second cohort in 2025.

In June 2026, we announced preliminary data on the first cohort in GenTLE.

As of the May 29, 2026 data cutoff date, three of six patients in the first, low-dose cohort (1x10 12 gc/mL) achieved meaningful reductions in disabling seizures during months four through six of follow-up, ranging from a 79% to 100% decline from baseline.

The remaining three patients in the low-dose cohort experienced variable changes in disabling seizures during months four through six of follow-up, ranging from a 33% decrease to a 36% increase compared to baseline.

As of the June 19, 2026, there have been no SAEs related to AMT-260 or the surgical procedure reported.

All reported adverse events in the low dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2).

No immunosuppression was required.

Fabry disease program (AMT-191)

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The multicenter, open-label clinical trial consists of three dose-ranging cohorts of three or more patients each to assess safety, tolerability, and efficacy of AMT-191 in patients with Fabry disease.

In February 2026, we announced updated preliminary data from the Phase I/II study of AMT-191 for Fabry disease.

Updated preliminary safety and exploratory efficacy data included the following, with a data cutoff date as of January 8, 2026:

In June 2026, we presented updated preliminary data from the Phase I/II study of AMT-191 for Fabry disease.

The data, based on a March 15, 2026 data cutoff date, included patient follow-up ranging from three months to more than 18 months and consisted of the following:

● Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 1.0- to 16.2-fold above mean normal range (1.38-8.66 nmol;

mean normal of 3.57 nmol) at the lowest dose, 14.5- to 229.6-fold at the mid dose, and 58.7- to 143.6-fold at the highest dose.

These increases were durable for the measured time period ranging from the longest follow-up period of more than a year in a treated patient (high-dose cohort) to the shortest follow-up period of four-months in a treated patient (mid-dose cohort).

● Six of 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”).

● All 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”).

● Plasma lyso-Gb3 levels were stable post-dose across all dose cohorts, regardless of ERT status through the cutoff date.

As of February 18, 2026, all 11 dosed patients were withdrawn from ERT.

AMT-191 continued to show a manageable safety profile.

No SAEs related to AMT-191 were observed at the 4x10 13 gc/kg and 2x10 13 gc/kg doses.

No additional SAEs were observed at the 6x10 13 gc/kg dose beyond the five previously reported in September 2025 in two patients.

No SAEs related to AMT-191 were observed at the 4x10 13 gc/kg (“mid-dose”) and 2x10 13 gc/kg (“low-dose”) doses.

No additional SAEs were observed at the 6x10 13 gc/kg (“high-dose”) dose beyond those previously reported in September 2025 in two patients.

Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity.

Amyotrophic Lateral Sclerosis (AMT-162)

EPISOD1 is a Phase I/II multi-center, open-label trial of AMT-162 for the treatment of amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”) in the U.S.

In September 2025, we voluntarily paused enrollment in EPISOD1 upon the recommendation of the IDMC following a 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 a SAE determined to be related to AMT-162, that was observed in one patient in the second cohort.

Following review of the preliminary efficacy and safety data generated from EPISOD1, we have decided to discontinue development of AMT-162.

We will continue to collect safety data from the five patients dosed in EPISOD1, consistent with applicable safety and regulatory requirements.

CSL Behring Collaboration

In June 2020, we entered into a commercialization and license agreement with CSL Behring LLC (the “CSL Behring Agreement”) pursuant to which CSL Behring LLC (“CSL Behring”) received exclusive global rights to HEMGENIX ® .

We and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we agreed to supply the product to CSL Behring until such time that these capabilities are transferred to CSL Behring or its designated contract manufacturing organization (the “CSL Behring CSA”).

CSL Behring in September 2022 informed us about their intent to transfer contract manufacturing to a designated third party.

In July 2024 as part of our divestment of our commercial manufacturing operations to Genezen Holdings Inc.

and its subsidiary Genezen MA, Inc.

(together “Genezen”), we entered into a commercial supply agreement to transfer the manufacturing and supply activities to Genezen such that Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX ® on our behalf (the “Genezen CSA”).

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

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

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

These elevations resolved as of the end of May 2026 following a course of immunosuppression as per the study protocol.

Financial Overview

Key components of our results of operations include the following:

Three months ended March 31,

Three months ended June 30,

Six months ended June 30,

(in thousands)

Total revenues

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Selling, general and administrative expenses

As of March 31, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $586.6 million and $622.5 million, respectively.

We had a net loss of $53.5 million in the three months ended March 31, 2026, compared to a net loss of $43.6 million for the same period in 2025.

As of March 31, 2026 and December 31, 2025, we had accumulated deficits of $1,382.5 million and $1,328.9 million, respectively.

As of June 30, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $810.3 million and $622.5 million, respectively.

We had a net loss of $81.1 million and $134.6 million in the three and six months ended June 30, 2026, compared to a net loss of $37.7 million and $81.4 million for the same periods in 2025.

As of June 30, 2026 and December 31, 2025, we had accumulated deficits of $1,463.5 million and $1,328.9 million, respectively.

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

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

There were no material changes to our critical accounting policies during the three months ended March 31, 2026.

There were no material changes to our critical accounting policies during the six months ended June 30, 2026.

Research and development expenses

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Our other income generally consists of payments received to subsidize our research and development efforts and income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility.

In 2024, as part of the divestment of our commercial manufacturing operations to Genezen Holdings Inc.

and its subsidiary Genezen MA, Inc.

(together “Genezen”), we entered into various service agreements, including a commercial supply a greement (the “Genezen CSA”).

In April 2026, we entered into an agreement to terminate the Genezen CSA.

Pursuant to the termination agreement, our obligation to supply HEMGENIX ® and any minimum purchase commitments under the Genezen CSA terminate once contractually specified batches have been supplied.

Our other expenses consist of costs incurred under the Genezen CSA.

These include costs related to the purchase of HEMGENIX ® from Genezen, net of income from the sales of HEMGENIX ® to CSL Behring LLC (“CSL Behring”), amortization of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA, and release of liabilities related to expected net losses associated with the remaining minimum purchase commitments under the Genezen CSA.

Additionally, other expenses also consist of expenses we incur in relation to our subleasing income.

Results of Operations

Comparison of the three months ended March 31, 2026 and 2025

The following table presents a comparison of our results of operations for the three months ended March 31, 2026 and 2025:

Three months ended March 31,

Comparison of the three months ended June 30, 2026 and 2025

The following table presents a comparison of our results of operations for the three months ended June 30, 2026 and 2025:

Three months ended June 30,

(in thousands)

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Loss from operations

Non-operating expense, net

Non-operating (expense) / income, net

Net loss before income tax expense

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

For the three months ended March 31, 2026 and 2025, we recognized $3.6 million and $1.6 million of license revenues, respectively.

R&D expenses for the three months ended March 31, 2026 were $29.2 million, compared to $36.1 million for the same period in 2025.

For the three months ended June 30, 2026 and 2025, we recognized $5.8 million and $5.3 million of license revenues, respectively.

R&D expenses for the three months ended June 30, 2026 were $34.0 million, compared to $35.4 million for the same period in 2025.

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

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

Three months ended March 31,

Three months ended June 30,

(in thousands)

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Temporal lobe epilepsy (AMT-260)

Fabry disease (AMT-191)

Amyotrophic lateral sclerosis (AMT-162)

Fabry disease (AMT-191)

Programs in preclinical development and platform related expenses

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Share-based compensation expense

Information technology costs

Fair value changes related to contingent consideration

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Huntington’s disease (AMT-130)

In the three months ended March 31, 2026 and 2025, we incurred costs of $8.0 million and $8.2 million respectively, primarily related to clinical trials and Chemistry, Manufacturing, and Controls (“CMC”) development.

We incurred costs of $4.5 million and $3.0 million related to clinical trials and CMC development, respectively, compared to costs of $5.7 million and $2.0 million in the comparative period.

In the three months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $6.3 million and $10.5 million respectively.

The decrease of $4.2 million was primarily related to lower manufacturing process validation costs, which were $0.8 million in the current year period, compared to $4.0 million in the prior year period.

Additionally, clinical trials costs decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and Marketing Authorization Application (“MAA”) submissions for AMT-130 in the United States and the United Kingdom, respectively.

Temporal lobe epilepsy (AMT-260)

In the three months ended March 31, 2026 and March 31, 2025 we incurred costs of $2.8 million and $2.1 million respectively, for the development of AMT-260.

We incurred costs of $2.6 million and $0.2 million related to clinical trials and CMC development, respectively, compared to costs of $1.6 million and $0.4 million in the comparative period.

Fabry disease (AMT-191)

In the three months ended March 31 , 2026 and March 31 , 2025, we incurred costs of $1.5 million and $2.2 million, respectively, related to our development of AMT-191, primarily in relation to our Phae I/II trial.

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $4.2 million and $2.0 million, respectively.

The current year period included $3.7 million of clinical expenses and $0.3 million of CMC expenses, respectively, compared to $1.7 million and $0.2 million, respectively, in the prior year period.

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

In the three months ended March 31, 2026 and March 31, 2025, we incurred $0.9 million and $1.8 million of expenses, respectively, primarily in relation to our Phase I/II clinical trial.

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $2.5 million and $1.1 million, respectively.

These costs were primarily related to our Phase I/II clinical trial.

Fabry disease (AMT-191)

In the three months ended June 30, 2026 and June 30, 2025 , we incurred costs related to the development of AMT-191 of $ 2.2 million and $1.3 million, respectively.

These costs were primarily related to our Phase I/II trial.

Preclinical programs & platform development

In the three months ended March 31 , 2026 and March 31, 2025 , we incurred $1.1 million and $1.3 million of costs, respectively, related to activities associated with product candidates for various other research programs and technology innovation projects.

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $1.9 million and $0.4 million, respectively.

Other research & development expenses

● We incurred $9.1 million in personnel and contractor-related expenses in the three months ended March 31 , 2026 , compared to $10.2 million for the same period in 2025.

The decrease was due to a decrease in personnel-related expenses, as well as a decrease in severance costs and reorganization expenses related to the divestment of our commercial manufacturing activities in July 2024, incurred in the first quarter of 2025 ;

● We incurred $3.6 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended March 31, 2026 compared to $4.6 million in the same period in 2025.

The decrease of $1.0 million was primarily driven by a revision to the estimated useful lives of certain assets, resulting in higher depreciation recognized during the three months ended March 31, 2025, compared with the current period;

● We recognized a $1.3 million gain in the three months ended March 31 , 2026, related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to a $1.2 million loss for the same period in 2025.

The decrease was primarily related to changes to the discount rate and expected timing of future achievement of the related remaining milestones ;

● We incurred $2.0 million in share-based compensation expenses in the three months ended March 31, 2026, compared to $2.5 million for the same period in 2025;

● We incurred $1.5 million of other expenses for the three months ended March 31 , 2026 , compared to $2.1 million for the same period in 2025.

● We incurred $ 9.1 million in personnel and contractor-related expenses in the three months ended June 30 , 2026 , compared to $9.5 million for the same period in 2025 ;

● We incurred $ 2.4 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended June 30, 2026 compared to $3.9 million in the same period in 2025;

● We incurred $1.9 million in share-based compensation expenses in the three months ended June 30, 2026, compared to $2.8 million for the same period in 2025.

The decrease of $0.9 million was primarily driven by lower costs related to performance stock units during the current year period;

● We incurred a $1.3 million loss related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the three months ended June 30, 2026, compared to a $2.3 million loss for the same period in 2025.

The $1.0 million favorable change reflects that the prior period included an adjustment to the expected timing of achieving future milestones that did not recur in the current year period.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended March 31, 2026 were $20.1 million, compared to $10.9 million for the same period in 2025.

Three months ended March 31,

Selling, general and administrative expenses for the three months ended June 30, 2026 were $17.4 million, compared to $13.5 million for the same period in 2025.

Three months ended June 30,

(in thousands)

Employee and contractor-related expenses

Professional fees

Share-based compensation expense

Professional fees

Intellectual property fees

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Total selling, general and administrative expenses

● We incurred $9.9 million in personnel and contractor-related expenses in the three months ended March 31 , 2026, compared to $5.5 million in the same period in 2025.

The increase in 2026 was primarily the result of a higher number of employees recruited in the second half of 2025 in support of a potential commercial launch of AMT-130 ;

● We incurred $3.7 million in professional fees in the three months ended March 31, 2026 compared to $1.9 million in the same period in 2025.

The increase was related to fees incurred in relation to legal and commercial services ;

● We incurred $3.1 million in share-based compensation expenses in the three months ended March 31 , 2026 , compared to $1.9 million in the same period in 2025.

The increase was primarily the result of a higher number of grants outstanding in the three months ended March 31 , 2026, in comparison with the prior period ;

● We incurred $1.7 million in other expenses in the three months ended March 31, 2026, compared to $0.6 million in the same period in 2025.

● We incurred $8.9 million in personnel and contractor-related expenses in the three months ended June 30 , 2026, compared to $5.4 million in the same period in 2025.

The $3.5 million increase was primarily driven by of a higher headcount following employee recruitment in the second half of 2025 to support the potential commercial launches of AMT-130 ;

● We incurred $3.1 million in share-based compensation expenses in the three months ended June 30, 2026, compared to $2.3 million in the same period in 2025.

The $0.8 million increase was primarily driven by the higher fair value of awards outstanding during the current year period, in comparison with the prior period ;

● We incurred $1.6 million in professional fees in the three months ended June 30, 2026 compared to $3.4 million in the same period in 2025.

The $1.8 million decrease was primarily a result of lower costs in connection with the potential commercial launches of AMT-130 ;

● We incurred $1.9 million in other expenses in the three months ended June 30, 2026, compared to $1.3 million in the same period in 2025.

Other income and expense

Three months ended March 31,

Three months ended June 30,

(in thousands)

Research and development grants from Dutch authorities

Sublease income / (expense), net

Sale of critical reagents to Genezen

Sublease income, net

Supply of HEMGENIX ® to CSL Behring

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Total other items, net

In April 2026, we entered into an agreement to terminate the Genezen CSA.

In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset within Other expense.

Other non-operating items, net

Our other non-operating items, net, for the three months ended March 31 , 2026 and March 31 , 2025 were as follows:

Three months ended March 31,

Our other non-operating items, net, for the three months ended June 30 , 2026 and June 30 , 2025 were as follows:

Three months ended June 30,

(in thousands)

1 unchanged sentence

Interest expense - Royalty Financing Agreement

Interest expense - Hercules debt facility

Interest expense - Hercules loan facility

Foreign currency (losses) / gains, net

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

We recognized $5.2 million in interest income in the three months ended March 31, 2026, compared to $4.1 million in the same period in the prior year.

Our interest income increased by $1.1 million, primarily due to increases in amounts of our investment securities during the three months ended March 31, 2026, compared to the same period in the prior year.

We recognized $5.1 million in interest income in the three months ended June 30, 2026, compared to $3.5 million in the same period in 2025.

The $1.5 million increase was primarily due to higher average balances of our investment securities held during the current year period, compared to the prior year period.

In May 2023, uniQure biopharma B.V.

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from CSL Behring from the net sales of HEMGENIX ® pursuant to the CSL Behring Agreement.

We recognized non-cash interest expenses related to the Royalty Financing Agreement of $12.6 million and $13.3 million in the three months ended March 31, 2026 and 2025, respectively.

We recognized interest expense of $1.4 million and $1.8 million in the three months ended March 31, 2026, and March 31, 2025, respectively, related to the Hercules loan facility.

Our interest expense decreased by $0.4 million, primarily due to improved terms following the September 2025 amendment as well as a decrease in market interest rates.

We recognized non-cash interest expenses related to the Royalty Financing Agreement of $13.0 million and $13.8 million in the three months ended June 30, 2026 and 2025, respectively.

We recognized interest expense related to the Hercules loan facility of $1.4 million and $1.8 million in the three months ended June 30, 2026 and June 30, 2025, respectively.

The $0.4 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.

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

Monetary balances are remeasured at period-end exchange rates, and resulting foreign currency transaction gains and losses are recorded in the Unaudited 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 $2.3 million during the three months ended March 31, 2026, compared to a net gain of $7.2 million during the same period in 2025.

We issued pre-funded warrants as part of our September 2025 follow-on public offering.

We recognize changes related to a liability recorded in relation to this issuance at fair value.

We recognized a $3.8 million gain in the three months ended March 31, 2026 related to a decrease in the fair value of the liability related to pre-funded warrants, compared to nil for the same period in 2025.

We recognized a net foreign currency loss of $1.7 million during the three months ended June 30, 2026, compared to a net gain of $18.6 million during the same period in 2025.

The foreign currency movements relate to our Hercules loan facility, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.

In connection with our September 2025 follow-on public offering, we issued pre-funded warrants that are classified as a liability and measured at fair value each reporting period.

We recognized a $16.0 million loss in the three months ended June 30, 2026, reflecting an increase in the fair value of this liability, compared to nil for the same period in 2025.

Income tax expense

We recognized $0.5 million of deferred tax expense in the three months ended March 31, 2026, and $0.5 million of deferred tax expense for the same period in 2025.

We recognized $1.8 million of deferred tax expense in the three months ended June 30, 2026, and $0.4 million of deferred tax expense for the same period in 2025.

Results of Operations

Comparison of the six months ended June 30, 2026 and 2025

The following table presents a comparison of our results of operations for the six months ended June 30, 2026 and 2025:

Six months ended June 30,

(in thousands)

Total revenues

Operating expenses:

Cost of license revenues

Research and development expenses

Selling, general and administrative expenses

Total operating expenses

Other expense

Loss from operations

Non-operating (expense) / income, net

Loss before income tax expense

Income tax expense

License revenues

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

For the six months ended June 30, 2026 and 2025, we recognized $9.4 million and $6.8 million of license revenues, respectively.

R&D expenses for the six months ended June 30, 2026 were $63.1 million, compared to $71.5 million for the same period in 2025.

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

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

Six months ended June 30,

(in thousands)

Huntington's disease (AMT-130)

Temporal lobe epilepsy (AMT-260)

Fabry disease (AMT-191)

Amyotrophic lateral sclerosis (AMT-162)

Programs in preclinical development and platform related expenses

Total direct research and development expenses

Employee and contractor-related expenses

Facility expenses

Share-based compensation expense

Information technology costs

Fair value changes related to contingent consideration

Other expenses

Total other research and development expenses

Total research and development expenses

Direct research and development expenses

Huntington’s disease (AMT-130)

In the six months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $14.3 million and $18.7 million respectively.

The decrease of $4.5 million was primarily driven by lower costs related to clinical trials of $9.0 million in the current period, compared to $11.2 million in the prior year period.

Additionally, manufacturing process validation costs were decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and MAA submissions for AMT-130 in the United States and the United Kingdom, respectively.

Temporal lobe epilepsy (AMT-260)

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $7.0 million and $4.0 million, respectively.

The current period included costs of $6.3 million related to clinical trials and $0.5 million related to CMC development, compared to $3.3 million and $0.7 million, respectively, in the prior year period.

Fabry disease (AMT-191)

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $3.7 million and $3.4 million, respectively.

These costs primarily related to the Phase I/II trial.

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

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $3.4 million and $2.9 million, respectively.

These costs primarily related to the Phase I/II trial.

Preclinical programs & platform development

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $3.0 million and $1.7 million, respectively.

Other research & development expenses

● W e incurred $18.2 million in personnel and contractor-related expenses in the six months ended June 30, 2026, compared to $19.7 million for the same period in 2025.

The decrease of $1.5 million was primarily due to lower personnel-related expenses in the current year period;

● We incurred $ 6.0 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the six months ended June 30, 2026 compared to $8.4 million in the same period in 2025.

The decrease of $ 2.4 million was primarily driven by a revision to the estimated useful lives of certain assets, resulting in higher depreciation recognized during the prior year period;

● We incurred $ 3.9 million in share-based compensation expenses in the six months ended June 30, 2026, compared to $5.2 million for the same period in 2025.

The decrease of $1.3 million was primarily driven by lower costs related to performance stock units during the current year period;

● We incurred a $0.1 million gain related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the six months ended June 30, 2026, compared to a $3.5 million loss for the same period in 2025.

The $3.6 million favorable change was primarily driven by movements in the discount rate and the timing of future milestone achievements;

● We incurred $1.7 million of other expenses for the six months ended June 30, 2026, compared to $2.6 million for the same period in 2025.

Selling, general and administrative expenses

Selling, general and administrative expenses for the six months ended June 30, 2026 were $37.4 million, compared to $24.4 million for the same period in 2025.

Six months ended June 30,

(in thousands)

Employee and contractor-related expenses

Share-based compensation expense

Professional fees

Intellectual property fees

Information technology costs

Depreciation and facility costs

Other expenses

Total selling, general and administrative expenses

● We incurred $18.7 million in personnel and contractor-related expenses in the six months ended June 30, 2026, compared to $10.9 million in the same period in 2025.

The $7.8 million increase was primarily driven by higher headcount following employee recruitment in the second half of 2025 to support the potential commercial launches of AMT-130;

● We incurred $5.2 million in professional fees in the six months ended June 30, 2026 compared to $5.3 million in the same period in 2025 ;

● We incurred $6.2 million in share-based compensation expenses in the six months ended June 30, 2026, compared to $4.3 million in the same period in 2025.

The $1.9 million increase was primarily driven by the higher fair value of awards outstanding during the current year period, in comparison with the prior period;

● We incurred $1.9 million in intellectual property fees in the six months ended June 30, 2026 compared to $0.6 million in the same period in 2025.

The $1.3 million increase was primarily driven by higher registration and professional fees ;

● We incurred $ 3.6 million in other expenses in the six months ended June 30, 2026, compared to $1.9 million in the same period in 2025.

Other income and expense

Six months ended June 30,

(in thousands)

Research and development grants from Dutch authorities

Sublease income, net

Sale of critical reagents to Genezen

Supply of HEMGENIX ® to CSL Behring

Other income, net

Total other items, net

In April 2026, we entered into an agreement to terminate the Genezen CSA.

In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset in Other expense.

Other non-operating items, net

Our other non-operating items, net, for the six months ended June 30, 2026 and June 30, 2025 were as follows:

Six months ended June 30,

(in thousands)

Interest income

Interest expense - Royalty Financing Agreement

Interest expense - Hercules loan facility

Foreign currency (losses) / gains, net

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

Total Other non-operating items, net

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

We recognized $10.3 million in interest income in the six months ended June 30, 2026, compared to $7.7 million in the same period in 2025.

The $2.6 million increase was primarily due to higher average balances of investment securities held during the current year period, compared to the prior year period.

We recognized interest expense related to the Hercules loan facility of $2.8 million in the six months ended June 30, 2026, compared to $3.6 million in the same period in 2025.

The $0.8 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.

We recognized a net foreign currency loss of $4.0 million in the six months ended June 30, 2026, compared to a net gain of $25.8 million in the same period in 2025.

The foreign currency movements related to our borrowings from Hercules, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.

Income tax expense

We recognized $2.3 million of deferred tax expense in the six months ended June 30, 2026, compared to $0.9 million in the same period in 2025.

Financial Position, Liquidity and Capital Resources

As of March 31, 2026, we had cash and cash equivalents, restricted cash and investment securities of $588.1 million.

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

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

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

As of June 30, 2026, we had cash and cash equivalents, restricted cash and investment securities of $811.9 million.

We believe these resources will be sufficient to fund our projected operating expenses into 2030, including costs associated with the commercial launches and confirmatory study of AMT-130, ongoing clinical trials of AMT-130, AMT-191 and AMT-260, and potential investments to advance certain pipeline candidates into late stage development.

The amount and timing of our actual expenditures may vary significantly depending on the commencement and design of any new clinical studies that we may conduct.

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

If the BLA for AMT-130 is approved prior to June 15, 2027, provided that confirmatory trials to the extent and in the manner required to support full approval (if applicable) remain ongoing or are being planned, we could draw down up to $100.0 million under our senior secured term loan facility with Hercules.

This includes up to $100.0 million in additional availability under our Hercules senior secured term loan facility, which becomes accessible upon BLA approval for AMT-130 prior to September 30, 2027, subject to confirmatory trial requirements remaining on track.

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.

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

As of March 31, 2026, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million.

As of June 30, 2026, 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.

The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to October 1, 2028.

The interest-only period will be further extended to October 1, 2030 if certain commercial milestones are met prior to March 31, 2029.

Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $19.5 million, with $5.4 million payable within the next 12 months.

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

As of March 31, 2026, we had fixed lease payment obligations of $20.9 million, with $4.6 million payable within the next 12 months.

As of June 30, 2026, we had fixed lease payment obligations of $22.9 million, with $5.2 million payable within the next 12 months.

Following the closing of the Lexington Transaction, we assigned our lease for our prior manufacturing facility in Lexington, MA to Genezen.

As of March 31, 2026, we remain obligated under a guarantee of lease payments of $15.2 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.

As of June 30, 2026, we remain obligated under a guarantee of lease payments of $14.0 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.

Commitments related to uniQure France SAS acquisition (nominal amounts)

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

As of March 31, 2026, our remaining commitment amounts include EUR 160.0 million ($184.0 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S.

As of June 30, 2026, our remaining commitment amounts include EUR 160.0 million ($182.3 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 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 March 31, 2026 of $1.15/€1.00.

As of March 31, 2026, we expect these obligations will become payable between 2030 and 2034.

These payments are owed in euro and have been translated at the foreign exchange rate as of June 30, 2026 of $1.14/€1.00.

As of June 30, 2026, we expect these obligations will become payable between 2030 and 2034.

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

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

The table below summarizes our consolidated cash flow data for the three months ended March 31, 2026 and 2025:

Three months ended March 31,

The table below summarizes our consolidated cash flow data for the six months ended June 30, 2026 and 2025:

Six months ended June 30,

(in thousands)

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We continued to incur losses in the current period.

We recorded a net loss of $53.5 million in the three months ended March 31, 2026, compared to a net loss of $43.6 million during the same period in 2025.

As of March 31, 2026, we had an accumulated deficit of $1,382.5 million.

We recorded a net loss of $81.1 million and $134.6 million in the three and six months ended June 30, 2026, compared to a net loss of $37.7 million and $81.4 million during the same period in 2025.

As of June 30, 2026, we had an accumulated deficit of $1,463.5 million.

Sources of liquidity

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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 June 2026, we received aggregate net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, following the completion of a follow-on public offering of 5.7 million ordinary shares at a public offering price of $45.50 per ordinary share.

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.

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Net cash used in operating activities

Net cash used in operating activities was $38.2 million for the three months ended March 31, 2026 and consisted of a net loss of $53.5 million, adjusted for non-cash items including depreciation and amortization expense of $3.0 million, amortization of the discount on investment securities of $4.3 million, share-based compensation expense of $5.1 million, $7.2 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $0.5 million, changes in the fair value of contingent consideration of $1.3 million, changes in the fair value of the liability related to pre-funded warrants of $3.8 million and unrealized foreign exchange losses of $2.2 million.

Net cash used in operating activities was $66.8 million for the six months ended June 30, 2026 and consisted of a net loss of $134.6 million, adjusted for non-cash items including depreciation and amortization expense of $7.9 million, amortization of the discount on investment securities of $7.9 million, share-based compensation expense of $10.1 million, $17.4 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $2.3 million, changes in the fair value of contingent consideration of $0.1 million, changes in the fair value of the liability related to pre-funded warrants of $12.2 million and unrealized foreign exchange losses of $8.3 million.

Net cash used in operating activities also included favorable changes in operating assets and liabilities of $20.0 million.

There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $3.8 million.

There was a net increase in accounts payable, accrued expenses, other liabilities, income taxes payable and operating leases of $3.3 million.

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

There was a net decrease in accounts receivable, accrued income, prepaid expenses, and other current assets and receivables of $8.8 million.

There was a net increase in accounts payable, accrued expenses, and operating leases of $11.1 million.

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

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

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Net cash generated from investing activities

In the three months ended March 31, 2026, we generated $98.6 million from our investing activities compared to $20.1 million generated during the same period in 2025.

Three months ended March 31,

In the six months ended June 30, 2026, we generated $151.8 million from our investing activities compared to $93.2 million generated during the same period in 2025.

Six months ended June 30,

(in thousands)

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Net cash generated from financing activities

In the three months ended March 31, 2026, net cash generated from financing activities was $0.6 million, compared to net cash generated from financing activities of $80.7 million in the same period in 2025.

Three months ended March 31,

In the six months ended June 30, 2026, net cash generated from financing activities was $249.7 million, compared to net cash generated from financing activities of $80.7 million in the same period in 2025.

Six months ended June 30,

(in thousands)

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

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

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

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

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

● investments required to support the MAAs of AMT-130 or generate confirmatory evidence in conjunction with obtaining accelerated approval;

● investments in the late stage development of AMT-191 and AMT-260;

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

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

9 unchanged sentences

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

What changed 10-Q

Item 2. Management’s Discussion and Analysis