What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-05-05 compared with 2025-11-10 · 151 added, 360 removed, 194 unchanged (72% of the section changed)

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

We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, refractory mesial temporal lobe epilepsy (“MTLE”), Fabry disease, and amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”).

Business Developments

Recent Product Candidate Developments

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;

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

We are currently conducting Phase I/II clinical trials of AMT-130 in the U.S.

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

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

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

Phase I/II study .

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.

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.

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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.

● A statistically significant 75% slowing of disease progression as measured by composite Unified Huntington’s Disease Rating Scale (“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.

● A statistically significant 60% slowing of disease progression as measured by Total Function Capacity (“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.

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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.

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.

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

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).

In general, the preliminary clinical evidence should show a clear advantage over available therapy.

In June 2024, we announced that the FDA granted us Regenerative Medicine Advanced Therapy (“RMAT”) designation for AMT-130 based on the potential of AMT-130 to address the major unmet medical need among patients with Huntington’s disease.

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

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.

Recent Developments of Other Product Candidates

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

Food and Drug Administration (“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.

The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and reductions in CSF NfL may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.

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

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

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

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.

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 United States.

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 first signs for efficacy of AMT-260 in patients with refractory MTLE.

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.

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

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.

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.

Following a review by the independent data monitoring committee (“IDMC”), we expanded the first cohort into MTLE in the dominant hemisphere and initiated a second cohort.

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.

Fabry disease program (AMT-191)

We are conducting a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease.

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 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.

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.

We are conducting a Phase I/II clinical trial of AMT-191 for the treatment of Fabry disease.

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:

● Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 0.34- to 82.2-fold above mean normal range (1.38-8.66 nmol;

mean normal of 3.57 nmol) at the lowest dose, 1.6- to 312.52-fold at the mid dose, and 27.7- to 223.7-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”).

● 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.

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 SOD1-ALS being conducted in the United States consisting of three dose-escalating cohorts with up to four patients in each cohort.

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.

In October 2024, we announced the first patient had been dosed in EPISOD1.

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.

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.

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.

Financial Overview

Key components of our results of operations include the following:

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Three months ended March 31,

(in thousands)

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Cost of license revenues

Cost of contract manufacturing revenues

Research and development expenses

Selling, general and administrative expenses

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.

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.

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

Critical Accounting Policies and Estimates

In preparing our consolidated financial statements in accordance with U.S.

In preparing our unaudited consolidated financial statements in accordance with U.S.

GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the “SEC”) we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures.

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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 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 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.

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

Research and development expenses

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

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

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

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

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Selling, general and administrative expenses

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

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

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

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 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.

(together “Genezen”).

These include costs 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 .

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

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.

Results of Operations

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,

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,

(in thousands)

License revenues

Total revenues

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Cost of license revenues

Cost of contract manufacturing revenues

Research and development expenses

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Income tax expense

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

Three months ended September 30,

(in thousands)

License revenues

Collaboration revenues

Total revenues

License revenues

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

For the 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 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.

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.

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 September 30,

Three months ended March 31,

(in thousands)

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Facility expenses

Fair value changes related to contingent consideration

Share-based compensation expense

Severance costs

Fair value changes related to contingent consideration

Other expenses

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Direct research and development expenses

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 for these activities and present the associated expenses within Direct research and development expenses.

Huntington’s disease (AMT-130)

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.

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.

Temporal lobe epilepsy (AMT-260)

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 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.

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.

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

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.

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.

Preclinical programs & platform development

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.

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.

Other research & development expenses

● 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 $3.7 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.

● 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.

Selling, general and administrative expenses

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,

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,

(in thousands)

5 unchanged sentences

Information technology costs

Severance costs

Other expenses

Total selling, general and administrative expenses

● 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 September 30, 2025 and September 30, 2024 were as follows:

Three months ended September 30,

● 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.

Other income and expense

Three months ended March 31,

(in thousands)

Research and development grants from Dutch authorities

Gain on sale of commercial manufacturing activities

Sublease income / (expense), net

Supply of HEMGENIX® to CSL Behring

Other income, net

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 September 30, 2025 and September 30, 2024 were as follows:

Three months ended September 30,

(in thousands)

Interest income

Interest expense - Royalty Financing Agreement

Interest expense - Hercules debt facility

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 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 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 $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 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)

Total revenues

Operating expenses:

Cost of license revenues

Cost of contract manufacturing revenues

Research and development expenses

Selling, general and administrative expenses

Total operating expenses

Other expense

Loss from operations

Non-operating expense, net

Loss before income tax expense

Income tax expense

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

Nine months ended September 30,

(in thousands)

License revenues

Contract manufacturing revenues

Collaboration revenues

Total revenues

License revenues

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

For the 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 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 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 to specific projects, as they are deployed across multiple projects under development.

Nine months ended September 30,

(in thousands)

Huntington's disease (AMT-130)

Temporal lobe epilepsy (AMT-260)

Fabry disease (AMT-191)

Amyotrophic lateral sclerosis (AMT-162)

Programs in preclinical development and platform related expenses

Total direct research and development expenses

Employee and contractor-related expenses

Facility expenses

Share-based compensation expense

Fair value changes related to contingent consideration

Severance costs

Other expenses

Total other research and development expenses

Total research and development expenses

Direct research and development expenses

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

The related expenses were part of Other 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 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.

Temporal lobe epilepsy (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 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 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 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 $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 $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 $5.9 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 $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 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)

Employee and contractor-related expenses

Professional fees

Share-based compensation expense

Depreciation and facility costs

Intellectual property fees

Information technology costs

Severance costs

Other expenses

Total selling, general and administrative expenses

● We incurred $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 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 nine months ended September 30, 2025 and September 30, 2024 were as follows:

Nine months ended September 30,

(in thousands)

Sale of critical reagents to Genezen

Research and development grants from Dutch authorities

Gain on sale of commercial manufacturing activities

Sublease income / (expense), net

Supply of HEMGENIX ® to CSL Behring

1 unchanged sentence

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 nine months ended September 30, 2025 and September 30, 2024 were as follows:

Nine months ended September 30,

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,

(in thousands)

2 unchanged sentences

Interest expense - Hercules debt facility

Interest expense - other

Foreign currency gains, net

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 $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 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.

In May 2023, uniQure biopharma B.V.

entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P.

to sell certain current and future royalties due to uniQure biopharma B.V.

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 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.

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.

Income tax expense

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.

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.

Financial Position, Liquidity and Capital Resources

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.

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.

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.

3 unchanged sentences

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

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

As of March 31, 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.

3 unchanged sentences

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

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 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.

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.

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.

Commitments related to uniQure France SAS acquisition (nominal amounts)

1 unchanged sentence

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 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.

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.

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 September 30, 2025, of $1.17/€1.00.

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

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.

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

1 unchanged sentence

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 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 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 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,

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,

(in thousands)

2 unchanged sentences

Net cash generated from investing activities

Net cash generated from / (used in) financing activities

Net cash generated from financing activities

Foreign exchange impact

2 unchanged sentences

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 $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.

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.

Sources of liquidity

1 unchanged sentence

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 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.

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 $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.

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 also included favorable changes in operating assets and liabilities of $7.1 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 $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 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.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $6.3 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $3.0 million.

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 $3.3 million.

Net cash generated from investing activities

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 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 thousands)

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Investment in debt securities

Capital expenditures - European sites

Capital expenditures - Lexington sites

Divestment of commercial manufacturing facility

Capital expenditures

Net cash generated from investing activities

Net cash generated from financing activities

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 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 thousands)

Cash flows from financing activities

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

Long-term debt repayment

Net cash generated from / (used in) financing activities

Proceeds from follow-on public offering of ordinary shares, net of issuance costs

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

Net cash generated from 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 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;

● 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;

● 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