Item 2. Management’s Discussion and Analysis
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”). Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the United States (“U.S. GAAP”) and unless otherwise indicated are presented in United States (“ U.S.”) dollars.
Overview
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
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. and Europe. The Phase I/II AMT-130 clinical trials were supplemented by two additional protocols, each with a statistical plan that was discussed with and submitted to the United States Food and Drug Administration (the “FDA”) earlier in 2025. The additional protocols, among other things, provide for the pooling of data across the ongoing U.S. and European studies, and also prespecified the pivotal comparison of AMT-130 clinical endpoints compared to a propensity score-matched external control from the Enroll-HD natural history data set.
In September 2025, we announced positive topline data from the pivotal Phase I/II study of AMT-130. The pivotal study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) at 36 months compared to a propensity score-matched external control. The study also met a key secondary endpoint by achieving statistically significant slowing of disease progression as measured by Total Functional Capacity (“TFC”) at 36 months compared to a propensity score-matched external control.
In accordance with the prospectively defined statistical analysis plan aligned with, and submitted, to the FDA, we analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 36 months of follow up and were evaluated at that time point. Outcomes for each dose group were compared to a propensity score-matched external control drawn from the Enroll-HD natural history data set (n=940 for high-dose; n=626 for low-dose).
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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. Treated patients had a mean change in cUHDRS from baseline of -0.38 compared to a change of -1.52 for patients in the propensity score-matched external control.
● A statistically significant 60% slowing of disease progression as measured by TFC (p=0.033), which met a key secondary endpoint of the study. Treated patients had a mean change in TFC from baseline of -0.36 compared to a change of -0.88 for patients in the propensity score-matched external control.
● Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test (“SDMT”), Stroop Word Reading Test (“SWRT”) and Total Motor Score (“TMS”).
o An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of -0.44 compared to a change of -3.73 for patients in the propensity score-matched external control.
o A 113% slowing of disease progression as measured by SWRT (nominal p=0.002), with a mean change in SWRT from baseline of 0.88 compared to a change of -6.98 for patients in the propensity score-matched external control.
o A 59% slowing of disease progression as measured by TMS (nominal p=0.174), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control.
A mean reduction from baseline in cerebrospinal neurofilament light protein (“CSF NfL”) of -8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies. CSF NfL is a well-characterized, supportive biomarker of neurodegeneration. Elevation in CSF NfL has been shown to be strongly associated with greater clinical severity of Huntington’s disease.
We believe that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the high-dose group, compared to the variable trends observed in the low-dose group, reflect a dose-dependent response to AMT-130.
Various other supportive analyses of the results from the AMT-130 high-dose treatment group, including those using a propensity score-weighted external control and comparisons to the TRACK-HD and PREDICT-HD datasets, were consistent with the primary analysis.
AMT-130 was generally well-tolerated in the Phase I/II studies, with a manageable safety profile at both doses with no new drug-related serious adverse events observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure.
Data from the Phase I/II studies were presented at the 2025 Huntington’s Disease Clinical Research Congress, which took place from October 10-13, 2025, in Nashville, Tennessee.
In October 2025, we initiated and fully recruited a fourth cohort evaluating high-dose AMT-130 in six patients with lower striatal volumes compared to patients in previous cohorts. Patient dosing in the fourth cohort is expected to be completed before year-end 2025.
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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.
Financing
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.
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Recent Developments of Other Product Candidates
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. 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 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.
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.
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.
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During the third quarter of 2025, we voluntarily paused enrollment in EPISOD1 upon the recommendation of the IDMC following a September 2025 review of available preliminary data related to the safety and efficacy of AMT-162 in the context of a dose limiting toxicity, which resulted in an SAE determined to be related to AMT-162, that was observed in one patient in the second cohort. We continue to collect and evaluate data from the five patients dosed in EPISOD1 to date.
Financial Overview
Key components of our results of operations include the following:
Three months ended September 30,
Nine months ended September 30,
2025
2024
2025
2024
(in thousands)
(in thousands)
Total revenues
$
3,701
$
2,287
$
10,530
$
21,898
Cost of license revenues
(398)
(264)
(1,251)
(648)
Cost of contract manufacturing revenues
—
(757)
—
(17,060)
Research and development expenses
(34,366)
(30,595)
(105,889)
(104,942)
Selling, general and administrative expenses
(19,438)
(11,575)
(43,846)
(41,279)
Net loss
(80,529)
(44,378)
(161,885)
(166,295)
As of September 30, 2025 and December 31, 2024, we had cash and cash equivalents and investment securities of $694.2 million and $367.5 million, respectively. We had a net loss of $80.5 million and $161.9 million in the three and nine months ended September 30, 2025, respectively, compared to a net loss of $44.4 million and $166.3 million for the same period in 2024. As of September 30, 2025 and December 31, 2024, we had accumulated deficits of $1,291.9 million and $1,130.0 million, respectively. See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Critical Accounting Policies and Estimates
In preparing our 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. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. 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.
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Research and development expenses
We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:
● employee-related expenses, including salaries, benefits, travel and share-based compensation expense;
● costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;
● costs incurred to conduct consistency and comparability studies;
● costs incurred for the development and improvement of our manufacturing processes and methods;
● costs associated with research activities for enabling technology platforms;
● costs associated with the rendering of collaboration services;
● payments related to identifiable intangible assets without an alternative future use;
● payments to our licensors for milestones that have been achieved related to our product candidates;
● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and,
● changes in the fair value of liabilities recorded in relation to our 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. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgment due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:
● the scope, rate of progress and expense of our research and development activities;
● clinical trial protocols, speed of enrollment and resulting data;
● the effectiveness and safety of our product candidates; and,
● the timing of regulatory approvals.
A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidates.
Selling, general and administrative expenses
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.
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.
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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,
2025
2024
2025 vs 2024
(in thousands)
Total revenues
$
3,701
$
2,287
$
1,414
Operating expenses:
Cost of license revenues
(398)
(264)
(134)
Cost of contract manufacturing revenues
—
(757)
757
Research and development expenses
(34,366)
(30,595)
(3,771)
Selling, general and administrative expenses
(19,438)
(11,575)
(7,863)
Total operating expenses
(54,202)
(43,191)
(11,011)
Other income
1,510
2,591
(1,081)
Other expense
(2,044)
(1,915)
(129)
Loss from operations
(51,035)
(40,228)
(10,807)
Non-operating expense, net
(20,868)
(4,181)
(16,687)
Net loss before income tax expense
$
(71,903)
$
(44,409)
$
(27,494)
Income tax expense
(8,626)
31
(8,657)
Net loss
$
(80,529)
$
(44,378)
$
(36,151)
Revenues
Our revenues for the three months ended September 30, 2025 and 2024 were as follows:
Three months ended September 30,
2025
2024
2025 vs 2024
(in thousands)
License revenues
$
3,582
$
2,111
$
1,471
Collaboration revenues
119
176
(57)
Total revenues
$
3,701
$
2,287
$
1,414
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.
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R&D expense
R&D expenses for the three months ended September 30, 2025 were $34.4 million, compared to $30.6 million for the same period in 2024. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.
Three months ended September 30,
2025
2024
2025 vs 2024
(in thousands)
Huntington's disease (AMT-130)
$
11,801
$
4,470
$
7,331
Temporal lobe epilepsy (AMT-260)
2,245
1,606
639
Fabry disease (AMT-191)
1,709
792
917
Amyotrophic lateral sclerosis (AMT-162)
1,638
910
728
Programs in preclinical development and platform related expenses
1,419
965
454
Total direct research and development expenses
$
18,812
$
8,743
$
10,069
Employee and contractor-related expenses
8,566
9,967
(1,401)
Facility expenses
3,716
3,839
(123)
Fair value changes related to contingent consideration
1,822
(410)
2,232
Share-based compensation expense
695
1,413
(718)
Disposables
492
1,013
(521)
Severance costs
—
3,378
(3,378)
Other expenses
263
2,652
(2,389)
Total other research and development expenses
$
15,554
$
21,852
$
(6,298)
Total research and development expenses
$
34,366
$
30,595
$
3,771
Direct research and development expenses
General
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.
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.
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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. We incurred costs of $1.5 million and $0.3 million related to clinical trials and CMC development, respectively, compared to costs of $0.7 million and $0.1 million in the comparative period.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the three months ended September 30, 2025 and September 30, 2024, we incurred $1.6 million and $0.9 million of expenses, respectively, to initiate a Phase I/II clinical trial in which the first patient was enrolled in October 2024. We incurred $1.4 million and $0.2 million related to clinical trials and CMC development, respectively, compared to $0.7 million and $0.1 million in the comparative period.
Preclinical programs & platform development
In the three months ended September 30, 2025 and September 30 2024 , we incurred $1.4 million and $1.0 million of costs, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Other research & development expenses
● We incurred $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; and,
● 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.
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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,
2025
2024
2025 vs 2024
(in thousands)
Employee and contractor-related expenses
$
7,055
$
5,467
$
1,588
Professional fees
6,362
1,453
4,909
Share-based compensation expense
2,828
2,025
803
Intellectual property fees
582
505
77
Depreciation and facility costs
466
467
(1)
Information technology costs
310
201
109
Severance costs
—
740
(740)
Other expenses
1,835
717
1,118
Total selling, general and administrative expenses
$
19,438
$
11,575
$
7,863
● 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,
2025
2024
2025 vs 2024
(in thousands)
Research and development grants from Dutch authorities
$
1,012
$
1,071
$
(59)
Gain on sale of commercial manufacturing activities
—
1,171
(1,171)
Sublease income / (expense), net
31
(115)
146
Supply of HEMGENIX® to CSL Behring
(1,586)
(1,467)
(119)
Other income, net
9
16
(7)
Total other items, net
$
(534)
$
676
$
(1,210)
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.
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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,
2025
2024
2025 vs 2024
(in thousands)
Interest income
$
3,503
$
4,866
$
(1,363)
Interest expense - Royalty Financing Agreement
(13,806)
(12,832)
(974)
Interest expense - Hercules debt facility
(1,794)
(3,779)
1,985
Interest expense - other
(1,121)
—
(1,121)
Foreign currency (losses) / gains, net
(428)
7,564
(7,992)
Changes in fair value of liability related to pre-funded warrants
(5,722)
—
(5,722)
Issuance costs - Pre-funded warrants
(1,500)
—
(1,500)
Total non-operating items, net
$
(20,868)
$
(4,181)
$
(16,687)
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. dollar. 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.
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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,
2025
2024
2025 vs 2024
(in thousands)
Total revenues
$
10,530
$
21,898
$
(11,368)
Operating expenses:
Cost of license revenues
(1,251)
(648)
(603)
Cost of contract manufacturing revenues
—
(17,060)
17,060
Research and development expenses
(105,889)
(104,942)
(947)
Selling, general and administrative expenses
(43,846)
(41,279)
(2,567)
Total operating expenses
(150,986)
(163,929)
12,943
Other income
12,413
5,950
6,463
Other expense
(6,188)
(2,385)
(3,803)
Loss from operations
(134,231)
(138,466)
4,235
Non-operating expense, net
(18,107)
(26,256)
8,149
Loss before income tax expense
$
(152,338)
$
(164,722)
12,384
Income tax expense
(9,547)
(1,573)
(7,974)
Net loss
$
(161,885)
$
(166,295)
$
4,410
Revenues
Our revenues for the nine months ended September 30, 2025 and 2024 were as follows:
Nine months ended September 30,
2025
2024
2025 vs 2024
(in thousands)
License revenues
$
10,411
$
5,182
$
5,229
Contract manufacturing revenues
—
6,114
(6,114)
Collaboration revenues
119
10,602
(10,483)
Total revenues
$
10,530
$
21,898
$
(11,368)
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.
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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 expense
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,
2025
2024
2025 vs 2024
(in thousands)
Huntington's disease (AMT-130)
$
30,548
$
9,790
$
20,758
Temporal lobe epilepsy (AMT-260)
6,275
5,858
417
Fabry disease (AMT-191)
5,151
3,617
1,534
Amyotrophic lateral sclerosis (AMT-162)
4,567
4,539
28
Programs in preclinical development and platform related expenses
3,104
2,312
792
Total direct research and development expenses
$
49,645
$
26,116
$
23,529
Employee and contractor-related expenses
28,248
39,926
(11,678)
Facility expenses
12,119
16,910
(4,791)
Share-based compensation expense
5,916
7,694
(1,778)
Fair value changes related to contingent consideration
5,293
(2,032)
7,325
Disposables
1,878
4,354
(2,476)
Severance costs
—
4,475
(4,475)
Other expenses
2,790
7,499
(4,709)
Total other research and development expenses
$
56,244
$
78,826
$
(22,582)
Total research and development expenses
$
105,889
$
104,942
$
947
Direct research and development expenses
General
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.
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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; and,
● 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.
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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,
2025
2024
2025 vs 2024
(in thousands)
Employee and contractor-related expenses
$
17,937
$
17,355
$
582
Professional fees
11,693
5,692
6,001
Share-based compensation expense
7,100
8,780
(1,680)
Depreciation and facility costs
1,424
1,552
(128)
Intellectual property fees
1,146
1,397
(251)
Information technology costs
784
713
71
Severance costs
—
1,040
(1,040)
Other expenses
3,762
4,750
(988)
Total selling, general and administrative expenses
$
43,846
$
41,279
$
2,567
● 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; and,
● 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,
2025
2024
2025 vs 2024
(in thousands)
Sale of critical reagents to Genezen
$
6,000
$
—
$
6,000
Research and development grants from Dutch authorities
4,123
3,957
166
Gain on sale of commercial manufacturing activities
—
1,171
(1,171)
Sublease income / (expense), net
402
(260)
662
Supply of HEMGENIX® to CSL Behring
(4,842)
(1,467)
(3,375)
Other income, net
542
164
378
Total other items, net
$
6,225
$
3,565
$
2,660
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.
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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,
2025
2024
2025 vs 2024
(in thousands)
Interest income
$
11,154
$
17,179
$
(6,025)
Interest expense - Royalty Financing Agreement
(40,885)
(37,671)
(3,214)
Interest expense - Hercules debt facility
(5,415)
(11,194)
5,779
Interest expense - other
(1,121)
—
(1,121)
Foreign currency gains, net
25,382
5,430
19,952
Changes in fair value of liability related to pre-funded warrants
(5,722)
—
(5,722)
Issuance costs - Pre-funded warrants
(1,500)
—
(1,500)
Total non-operating items, net
$
(18,107)
$
(26,256)
$
8,149
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 conduct transactions and hold monetary assets and liabilities denominated in foreign currencies, principally the euro and the U.S. dollar. 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 gain, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $25.4 million during the nine months ended September 30, 2025, compared to a net gain of $5.4 million during the same period in 2024 .
We recognized a $5.7 million loss in the nine months ended September 30, 2025, related to an increase in the fair value of the liability related to pre-funded warrants, compared to nil for the same period in 2024.
We recognized $1.5 million of expenses in the nine months ended September 30, 2025 related to the issuance of the Pre-Funded Warrants, compared to nil for the same period in 2024.
Income tax expense
We recognized $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.
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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. We have based our estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, and as such, we may require additional funding.
If the BLA for AMT-130 is approved prior to June 15, 2027, provided that confirmatory trials to the extent and in the manner required to support full approval (if applicable) remain ongoing or are being planned, we could draw down up to $100.0 million under our senior secured term loan facility with Hercules. Additional funding could also include a combination of public equity offerings, collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible. If adequate funds are not available to us on acceptable terms when we need them, we may be unable to pursue further development of our clinical product candidates.
Our current material cash requirements include the following contractual and other obligations:
Debt
As of September 30, 2025, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million. The loan has an interest-only period until October 1, 2028 and we are contractually required to repay the $50.0 million in equal installments between October 1, 2028 and October 1, 2030. The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to June 15, 2027. The loan will be interest-only until October 1, 2030 if certain commercial milestones are met prior to December 31, 2028. Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $26.1 million, with $7.4 million payable within the next 12 months.
Leases
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.
Commitments related to uniQure France SAS acquisition (nominal amounts)
In connection with our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractually defined milestones. The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS following the dosing of the first patient in Phase I/II clinical trial for AMT-260. As of September 30, 2025, our remaining commitment amounts include EUR 160.0 million ($187.8 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and European Union. The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestones will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of September 30, 2025, of $1.17/€1.00. As of September 30, 2025, we expect these obligations will become payable between 2028 and 2033. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.
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Commitments related to licensors and financial advisors
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,
2025
2024
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period
$
160,329
$
244,544
Net cash used in operating activities
(100,394)
(129,884)
Net cash generated from investing activities
119,751
189,592
Net cash generated from / (used in) financing activities
412,949
(52,969)
Foreign exchange impact
5,914
1,796
Cash, cash equivalents and restricted cash at the end of period
$
598,549
$
253,079
We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement. We continue to incur losses in the current period. We recorded a net loss of $80.5 million and $161.9 million in the three and nine months ended September 30, 2025, respectively, compared to a net loss of $44.4 million and $166.3 million during the same period in 2024. As of September 30, 2025, we had an accumulated deficit of $1,291.9 million.
Sources of liquidity
From our first institutional venture capital financing in 2006 through to the current period, we have funded our operations primarily through private and public placements of equity securities, debt securities, pre-funded warrants, and payments from our collaboration partners, as well as $370.1 million through the sale of a portion of royalties due from our collaboration partner CSL Behring in 2023. Between July 2021 and July 2023, we collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL Behring and other milestones collected from CSL Behring, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Agreement) on net sales of HEMGENIX®.
In 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.
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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. In addition, our pledge of assets as collateral to secure our obligations under the senior secured term loan facility with Hercules may limit our ability to obtain debt financing.
To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all. If financing is not available when needed, including through debt or equity financings, or is available only on unfavorable terms, we may be unable to meet our cash needs. If we raise additional funds through collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.
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. There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $2.9 million. There was a net increase in accounts payable, accrued expenses, other liabilities, income taxes payable and operating leases of $25.2 million.
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Net cash used in operating activities was $129.9 million for the nine months ended September 30, 2024 and consisted of a net loss of $166.3 million adjusted for non-cash items, including depreciation and amortization expense of $8.3 million, amortization of the discount on investment securities of $8.5 million, share-based compensation expense of $17.5 million, $33.1 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $1.6 million, changes in the fair value of contingent consideration of $2.0 million, recognition of an allowance for inventory write-downs of $6.3 million and unrealized foreign exchange gains of $5.7 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $11.7 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $5.8 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 $2.0 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,
2025
2024
(in thousands)
Cash flows from investing activities
Proceeds from maturity of debt securities
$
337,195
$
480,806
Investment in debt securities
(217,124)
(279,586)
Capital expenditures - European sites
(274)
(1,743)
Capital expenditures - Lexington sites
(46)
(1,585)
Divestment of commercial manufacturing facility
—
(8,300)
Net cash generated from investing activities
$
119,751
$
189,592
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,
2025
2024
(in thousands)
Cash flows from financing activities
Proceeds from public offerings of shares from follow-ons, net of issuance costs
$
380,737
$
—
Proceeds from issuance of pre-funded warrants, net of issuance costs
23,499
—
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans
8,713
81
Long-term debt repayment
—
(53,050)
Net cash generated from / (used in) financing activities
$
412,949
$
(52,969)
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;
● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones;
● the scope, timing, results, and costs of our current and planned clinical trials;
● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements;
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● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;
● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;
● the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates;
● the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies;
● the cost, timing and outcome of regulatory reviews associated with our product candidates;
● our ability to enter into collaboration arrangements in the future; and,
● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims.
Text extracted from the filing as submitted to EDGAR. Formatting, tables and exhibits are simplified for reading; the original document is authoritative for anything you rely on.