Item 7. Management’s Discussion and Analysis
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (“MD&A”) is intended to help the reader understand our results of operations and financial condition. This MD&A is provided as a supplement to, and should be read in conjunction with, our audited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Annual Report on Form 10-K, including the disclosures under “Risk Factors”. Our consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S. GAAP”) and unless otherwise indicated are presented in U.S. dollars.
Except for the historical information contained herein, the matters discussed in this MD&A may be deemed to be forward-looking statements. Forward-looking statements are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Words such as “may,” “expect,” “anticipate,” “estimate,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements.
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, MTLE, Fabry disease, and SOD1-ALS.
Huntington’s disease program (AMT-130)
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 an AAV vector carrying a miRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
We are currently conducting U.S. and European studies. 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.
Clinical Data
In September 2025, we announced positive topline data from the pivotal Phase I/II study of AMT-130 in which 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 as of the June 30, 2025 cutoff date. 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).
Regulatory Updates
In October 2025, we held a pre-BLA meeting with the FDA 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.
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Other clinical candidates
In 2025, we completed treatment of all six patients in the first cohort AMT-260. We also started enrolling patients into the second cohort, which is expected to consist of an additional six patients.
In 2025, we completed the enrollment of three cohorts of three patients each into our Phase I/II clinical trial of AMT-191 for the treatment of Fabry disease. We announced initial data from the first cohort at a scientific conference in September 2025 and additional preliminary data at a scientific conference in February 2026.
In September 2025, we voluntarily paused enrollment in EPISOD1, our Phase I/II clinical trial of AMT-162 for the treatment of SOD1-ALS. We continue to collect and evaluate data from the five patients dosed in EPISOD1 to date.
Financing
Public offerings
In January 2025, we raised $70.1 million of net proceeds, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a price to the public of $17.00 per ordinary share. In February 2025, we raised an additional $10.4 million in net proceeds upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.
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 repayment and amendment
In September 2025, we entered into the 2025 Amended Facility with Hercules for a $175.0 million senior secured term loan facility. 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 9.45% as of December 31, 2025. The tranches mature on October 1, 2030.
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Financial Highlights
Key components of our results of operations include the following:
Years ended December 31,
2025
2024
2023
(in thousands)
Total revenues
$
16,098
$
27,119
$
15,843
Cost of license revenues
(1,686)
(1,267)
(65)
Cost of contract manufacturing revenues
—
(17,060)
(13,563)
Research and development expenses
(140,673)
(143,782)
(214,864)
Selling, general and administrative expenses
(65,456)
(52,657)
(74,591)
Net loss
(198,971)
(239,556)
(308,478)
As of December 31, 2025, we held $622.5 million in cash and cash equivalents and investment securities (December 31, 2024: $367.5 million). We had a net loss of $199.0 million in the year ended December 31, 2025 and a net loss of $239.6 million and $308.5 million during the same periods in 2024 and 2023, respectively. As of December 31, 2025, we had an accumulated deficit of $1,328.9 million (December 31, 2024: $1,130.0 million).
Refer to “ 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 SEC, we make assumptions, judgments and estimates that can have a significant impact on our net loss/income and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. On an ongoing basis, we evaluate our assumptions, estimates and judgments, including those related to what we believe to be our critical accounting policies. Refer to Note 2 “ Summary of significant accounting policies ” for a summary of our significant accounting policies.
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, judgments or estimates. We also discuss our critical accounting estimates with the Audit Committee of our Board of Directors.
We consider the following to be our critical accounting estimate in the year ended December 31, 2025:
● Contingent consideration recorded in relation to the uniQure France business combination
Contingent consideration
In July 2021, in connection with the uniQure France Acquisition, we recorded contingent consideration related to amounts contingently payable to uniQure France’s former shareholders. The amounts payable in accordance with the related share and purchase agreement are contingent upon the achievement of certain milestones associated with AMT-260. Contingent consideration was measured at fair value as of the date of the uniQure France Acquisition, with changes in fair value subsequently recognized in the Consolidated Statements of Operations and Comprehensive Loss, within Research and development expenses.
Changes in the fair value of the contingent consideration may result from changes in the assumptions regarding the probability and timing of milestone achievement, as well as changes in the discount rate used in the valuation of the liability:
● As of December 31, 2025 we used a discount rate of 11.8% compared to a range of 15.3% to 16.2% as of December 31, 2024, reflecting changes in our credit risk and market interest rates. An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability.
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● We regularly update our estimates of when milestones are expected to be achieved. The achievement of a milestone at a later than currently expected date reduces the fair value of the contingent consideration liability, whereas achievement at an earlier than currently expected date increases the fair value of the contingent consideration liability.
● During the years ended December 31, 2025 and 2024, no changes to the probabilities of achievement of future milestones were identified. During the year ended December 31, 2023, we increased the probability of achieving a certain development related milestone to 100.0% following the clearance of the IND application for AMT-260 in August 2023, and subsequently achieved the related milestone in 2024.
The fair value of the contingent consideration liability as of December 31, 2025 was EUR 15.9 million ($18.7 million) and as of December 31, 2024 was EUR 10.5 million ($10.9 million). The increase was driven by changes to the discount rate and expected timing of future achievement of the related remaining milestones. If, as of December 31, 2025, the Company had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 51.4 million ($60.4 million). If, as of December 31, 2025 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.
Recently Adopted Accounting Pronouncements
In the year ended December 31, 2025, we adopted ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures . This update requires enhanced disaggregation and transparency of income tax disclosures, including expanded rate reconciliation and cash tax information. The adoption of ASU 2023-09 resulted in additional income tax disclosures included in this Annual Report and did not have a material impact on our consolidated financial statements.
In November 2024, the SEC adopted final rules under Release No. 33-11275, Disaggregation of Income Statement Expenses (ASU 2024-03) . The rules require registrants to provide enhanced disclosures regarding the disaggregation of certain operating expense categories. The new disclosures are required on an annual basis for fiscal years beginning after December 15, 2026 for large accelerated filers, and interim period disclosures are required beginning in the first quarterly report after the year of adoption. Early adoption is permitted. We are currently evaluating the impact of the final rules.
There are no other recently issued accounting pronouncements pending adoption that are applicable or expected to have a material impact.
Results of Operations
The following table presents a comparison of the years ended December 31, 2025, 2024 and 2023.
Years ended December 31,
2025
2024
2023
2025 vs 2024
2024 vs 2023
(in thousands)
Total revenues
$
16,098
$
27,119
$
15,843
$
(11,021)
$
11,276
Operating expenses:
Cost of license revenues
(1,686)
(1,267)
(65)
(419)
(1,202)
Cost of contract manufacturing revenues
—
(17,060)
(13,563)
17,060
(3,497)
Research and development expenses
(140,673)
(143,782)
(214,864)
3,109
71,082
Selling, general and administrative expenses
(65,456)
(52,657)
(74,591)
(12,799)
21,934
Total operating expenses
(207,815)
(214,766)
(303,083)
6,951
88,317
Other income
14,410
7,926
6,059
6,484
1,867
Other expense
(8,042)
(4,573)
(1,690)
(3,469)
(2,883)
Loss from operations
(185,349)
(184,294)
(282,871)
(1,055)
98,577
Non-operating expense, net
(7,991)
(52,833)
(23,686)
44,842
(29,147)
Loss before income tax expense
$
(193,340)
$
(237,127)
$
(306,557)
43,787
69,430
Income tax expense
(5,631)
(2,429)
(1,921)
(3,202)
(508)
Net loss
$
(198,971)
$
(239,556)
$
(308,478)
$
40,585
$
68,922
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Revenues and cost of revenues
Our revenues and associated costs for the years ended December 31, 2025, 2024 and 2023 were as follows:
Years ended December 31,
2025
2024
2023
2025 vs 2024
2024 vs 2023
(in thousands)
License revenues
$
15,934
$
10,133
$
2,758
$
5,801
$
7,375
Collaboration revenues
164
10,872
2,250
(10,708)
8,622
Contract manufacturing revenues
—
6,114
10,835
(6,114)
(4,721)
Total revenues
$
16,098
$
27,119
$
15,843
$
(11,021)
$
11,276
Cost of license revenues
(1,686)
(1,267)
(65)
(419)
(1,202)
Cost of contract manufacturing revenues
—
(17,060)
(13,563)
17,060
(3,497)
Total cost
$
(1,686)
$
(18,327)
$
(13,628)
$
16,641
$
(4,699)
CSL Behring
We sold the exclusive global rights to HEMGENIX to CSL Behring in 2021 (“License Sale”). We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of HEMGENIX have been earned. We recognized $15.9 million, $10.1 million and $2.8 million of license revenue for the years ended December 31, 2025, 2024 and 2023, respectively, related to royalty payments owed on HEMGENIX sales, when earned.
In 2008, we entered into a license agreement with St. Jude Children’s Research Hospital (“St. Jude”), which we amended in 2012. Under this license agreement, St. Jude has granted us an exclusive license, with a right to sublicense, to patent rights relating to expression of hFIX in gene therapy vectors, to make, import, distribute, use, and commercialize products containing hFIX covered by a valid patent claim in the field of gene therapy for treatment or prophylaxis of hemophilia B. The majority of the U.S. patent rights will expire in 2028 and the European patent rights (along with one U.S. patent) expired in 2025.
With respect to our collaboration with CSL Behring, we have agreed with St. Jude on an apportionment of certain amounts we receive from CSL Behring as sublicensing revenue that is equivalent to a low-single digit percentage of such amounts. The agreement will remain in effect until no further payment is due relating to any licensed product under this agreement or either we or St. Jude exercise our rights to terminate it. St. Jude may terminate the agreement in specified circumstances relating to our insolvency. We may terminate the agreement for convenience at any time subject to a specified notice period.
We treat license payments to St. Jude as contract fulfillment costs associated with license revenue we receive from CSL Behring and recognize these as costs of license revenues. We incurred $1.7 million, $1.3 million and $0.1 million of such cost in the years ended December 31, 2025, 2024 and 2023, respectively.
We recognize collaboration revenues associated with services we provide to CSL Behring. Collaboration revenue is recognized when the performance obligations are satisfied. We recognized $0.2 million, $10.9 million and $2.3 million of collaboration revenue for the years ended December 31, 2025, 2024 and 2023, respectively. The decrease in collaboration revenue in 2025 of $10.7 million compared to 2024 was primarily related to a reduction in services requested by CSL Behring following the Lexington Transaction in July 2024 . The increase in collaboration revenue in 2024 of $8.6 million compared to 2023 was primarily related to additional development and other services provided in relation to the transfer of manufacturing responsibility to CSL Behring.
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We provided contract manufacturing services to CSL Behring between April 2022 and July 2024. We transferred these activities to Genezen as part of the Lexington Transaction, which closed on July 22, 2024 (“the Closing”). Following the Closing of the Lexington Transaction, title to HEMGENIX supply directly passes from the contract manufacturer, Genezen, to CSL Behring. We do not control HEMGENIX before it is transferred to CSL Behring. We arrange for HEMGENIX to be provided by Genezen to CSL Behring. We determined that we are an agent in the sale of HEMGENIX to CSL Behring.
As a result of our being an agent, we recognize corresponding costs related to the purchase of HEMGENIX from Genezen, net of income from the sales of HEMGENIX to CSL Behring, and income related to the release of liabilities associated with expected net losses, in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss. We recognized contract manufacturing revenues related to the contract manufacture of HEMGENIX for CSL Behring when earned upon sales of HEMGENIX to CSL Behring. We recognized nil, $6.1 million and $10.8 million of contract manufacturing revenues in the years ended December 31, 2025, 2024 and 2023, respectively.
We incurred nil cost of contract manufacturing revenues related to the manufacture of HEMGENIX in the year ended December 31, 2025 and $17.1 million and $13.6 million of cost of contract manufacturing revenues, in the years ended December 31, 2024 and 2023, respectively. Costs of contract manufacturing revenues decreased in the year ended December 31, 2025 compared to the year ended December 31, 2024 as a result of the Lexington Transaction . Costs of contract manufacturing revenues increased in the year ended December 31, 2024 compared to the year ended December 31, 2023 primarily due to expensing costs that could not be recovered from selling HEMGENIX under the terms of our development and commercial supply agreement with CSL Behring. Cost of contract manufacturing revenues included a write down of inventories to net realizable value of $6.3 million and $1.6 million for the years ended December 31, 2024 and December 31, 2023, respectively.
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 the uniQure France Acquisition.
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Our research and development expenses primarily consist of costs incurred for the research and development of our product candidates, which include:
● AMT-130 (Huntington’s disease). We have incurred costs in relation to the preparation of the BLA submission for AMT-130 in the U. S., in addition to costs related to our U.S. study since February 2019, and our European study since 2021;
● AMT-260 (Temporal lobe epilepsy) . We have incurred costs related to the development of AMT-260 and costs to initiate and conduct a Phase I/IIa clinical trial. We acquired this program in July 2021;
● AMT-191 (Fabry disease) . We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease and costs to initiate and conduct a Phase I/II clinical trial;
● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ). We have incurred costs related to the acquisition in addition to costs incurred to initiate and conduct a Phase I/II clinical trial;
● AMT-061 (Hemophilia B). Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the periods ended December 31, 2025, 2024 and 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition;
● Preclinical research programs . We incurred costs related to the research of multiple preclinical gene therapy product candidates with the potential to treat certain rare and other serious medical conditions; and,
● Technology platform development and other related research . We incurred significant research and development costs related to manufacturing prior to the closing of the Lexington Transaction and other enabling technologies that are applicable across all of our programs.
Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including 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.
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Research and development expenses for the year ended December 31, 2025 were $140.7 million, compared to $143.8 million and $214.9 million for the years ended December 31, 2024 and 2023, respectively. Other research and development expenses are separately classified in the table below. These are not allocated as they are deployed across multiple projects under development.
Years ended December 31,
2025
2024
2023
2025 vs 2024
2024 vs 2023
(in thousands)
Huntington's disease (AMT-130)
$
42,462
$
17,989
$
12,991
$
24,473
$
4,998
Temporal lobe epilepsy (AMT-260)
9,751
12,053
13,037
(2,302)
(984)
Fabry disease (AMT-191)
6,827
4,814
2,659
2,013
2,155
Amyotrophic lateral sclerosis (AMT-162)
6,336
7,306
16,039
(970)
(8,733)
Programs in preclinical development and platform related expenses
3,611
3,927
11,005
(316)
(7,078)
Hemophilia B (AMT-061)
—
—
(1,336)
—
1,336
Total direct research and development expenses
$
68,987
$
46,089
$
54,395
$
22,898
$
(8,306)
Employee and contractor-related expenses
35,268
49,818
74,514
(14,550)
(24,696)
Facility expenses
15,173
21,627
29,490
(6,454)
(7,863)
Share-based compensation expense
7,579
9,295
16,881
(1,716)
(7,586)
Fair value changes related to contingent consideration
6,247
(1,817)
15,895
8,064
(17,712)
Disposables
2,585
5,084
13,232
(2,499)
(8,148)
Severance costs
—
3,967
2,188
(3,967)
1,779
Impairment charge
—
—
1,438
—
(1,438)
Other expenses
4,834
9,719
6,831
(4,885)
2,888
Total other research and development expenses
$
71,686
$
97,693
$
160,469
$
(26,007)
$
(62,776)
Total research and development expenses
$
140,673
$
143,782
$
214,864
$
(3,109)
$
(71,082)
Direct research and development expenses
Huntington disease (AMT-130)
In the years ended December 31, 2025, 2024 and 2023, we incurred $42.5 million, $18.0 million and $13.0 million of expenses, respectively. Our external costs for the development of AMT-130 were primarily related to the execution of our U.S. and European studies. The increase of $24.5 million in external cost in 2025 compared to 2024 includes $19.4 million of costs incurred in relation to the preparation of the BLA submission for AMT-130 in the U. S., including manufacturing process validation-related costs as well as expenses to support the process performance qualification campaign and other activities. Other costs incurred for the development of AMT-130 were a result of treating all six patients in the fourth cohort in 2025, as well as expenses related to the execution of our U.S. and European studies. The increase of $5.0 million in external cost in 2024 compared to 2023 is a result of enrolling patients into a third cohort between November 2023 and December 2024 as well as expenses we incurred associated with the RMAT application and in preparation of the Type B meeting with the FDA in November 2024.
Temporal lobe epilepsy (AMT-260)
In the years ended December 31, 2025, 2024 and 2023, we incurred $9.8 million, $12.1 million and $13.0 million of expenses, respectively. In August 2023, the FDA cleared our IND application, and we began incurring costs for the preparation of a Phase I/IIa clinical trial. In the years ended December 31, 2025, 2024 and 2023 we incurred costs of $7.5 million, $7.4 million and $3.6 million, respectively, related to our Phase I/IIa clinical trial. In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $0.7 million, $4.3 million and $4.6 million, respectively, related to CMC development. In the year ended December 31, 2025, we incurred $1.5 million in expenses related to a milestone payment.
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Fabry disease (AMT-191)
In the years ended December 31, 2025, 2024 and 2023, we incurred $6.8 million, $4.8 million and $2.7 million of expenses, respectively, related to our development of AMT-191. In November 2023, the FDA cleared the IND application, and we began incurring additional costs for Phase I/II clinical trial preparation. In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $6.0 million, $4.6 million and $1.1 million, respectively, related to our ongoing Phase I/II clinical trial. In the years ended December 31, 2025, 2024 and 2023 we incurred costs of $0.7 million, $0.2 million and $0.4 million, respectively, related to CMC development.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In January 2023, we entered into a global licensing agreement with Apic Bio for AMT-162. In the years ended December 31, 2025, 2024 and 2023, we incurred $6.3 million, $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial, in which we enrolled the first patient in October 2024. In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $6.0 million, $5.2 million and $2.6 million, respectively, related to EPISOD1. In the years ended December 31, 2025, 2024 and 2023, we incurred costs of $0.3 million, $1.9 million and $3.3 million, respectively, related to CMC development. During the year ended December 31, 2023, we incurred expenses of $10.0 million related to the upfront consideration paid to Apic Bio for the global licensing rights to AMT-162.
Preclinical programs & platform development
In the years ended December 31, 2025, 2024 and 2023, we incurred $3.6 million, $3.9 million and $11.0 million of expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Hemophilia B (AMT-061)
We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022. Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.
Other research & development expenses
● We incurred $35.3 million in employee and contractor expenses in the year ended December 31, 2025 compared to $49.8 million in 2024 and $74.5 million in 2023. Our costs decreased in 2025 by $14.6 million compared to 2024 and decreased in 2024 by $24.7 million compared to 2023, primarily related to the divestment of our commercial manufacturing activities in July 2024 as well as reorganization activity in October 2023 and August 2024;
● We incurred $15.2 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the year ended December 31, 2025 compared to $21.6 million in 2024 and $29.5 million in 2023. The decrease in 2025 compared to 2024 of $6.5 million and the decrease in 2024 compared to 2023 of $7.9 million, is primarily related to the divestment of our commercial manufacturing activities in July 2024;
● We incurred $7.6 million in share-based compensation expenses in the year ended December 31, 2025 compared to $9.3 million in 2024 and $16.9 million in 2023. The decrease in 2025 of $1.7 million, compared to 2024, was primarily related to the divestment of our commercial manufacturing activities in July 2024 and a reduction in expense from the restructuring that occurred in August 2024. The decrease in 2024 of $7.6 million, compared to 2023, is primarily related to the divestment of our commercial manufacturing activities in July 2024, the reorganizations in October 2023 and August 2024, and a decrease in the fair value of granted long-term incentive awards;
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● We recognized a $6.2 million loss in the year ended December 31, 2025 related to an increase in the fair value of contingent consideration associated with the uniQure France Acquisition, compared to a gain of $1.8 million in 2024 and a loss of $15.9 million in 2023. The loss recognized in 2023 was primarily due to an increase in the probability of achievement of future milestones, upon dosing the first patient in the Phase I/IIa clinical trial of AMT-260, after receiving IND acceptance in August 2023;
● We incurred $2.6 million of disposables costs in the year ended December 31, 2025 compared to $5.1 million in 2024 and $13.2 million in 2023. The decrease of $2.5 million in 2025 primarily related to the divestment of our commercial manufacturing activities in July 2024, as well as the reorganization in August 2024. The decrease of $8.1 million in 2024 primarily related to the divestment of our commercial manufacturing activities in July 2024, as well as reorganization activities in October 2023 and August 2024;
● We incurred nil severance costs in the year ended December 31, 2025. We incurred $4.0 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization, compared to $2.2 million of severance cost in 2023 related to the October 2023 reorganization;
● We incurred nil costs in the years ended December 31, 2025 and 2024, and $1.4 million of costs in the year ended December 31, 2023, related to the impairment of the right-of-use asset at the Lexington Facility and related leasehold improvements; and,
● We incurred $4.8 million in other expenses in the year ended December 31, 2025 compared to $9.7 million in 2024 and $6.8 million in 2023. The decrease in costs in 2025 of $4.9 million, compared to 2024, is primarily due to a decrease in information technology expenses and consultant-related expenses. The increase in costs in 2024 of $2.9 million, compared to 2023, is primarily a result of an increase in information technology expenses, partially offset by decreases in contractual license expenses and consultant-related expenses.
Selling, general 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, expenses to maintain our patent and license portfolio, and fees related to business development. As from 2025, we also incurred material expenses associated with activities to prepare for a potential launch of AMT-130 in the U.S., and present expenses related to medical affairs, new product planning and patient advocacy within selling, general and administrative expenses.
Selling, general and administrative expenses for the year ended December 31, 2025 were $65.5 million, compared to $52.7 million and $74.6 million for the years ended December 31, 2024 and 2023, respectively.
Years ended December 31,
2025
2024
2023
2025 vs 2024
2024 vs 2023
(in thousands)
Employee and contractor-related expenses
$
24,941
$
21,343
$
24,399
$
3,598
$
(3,056)
Professional fees
16,638
7,261
11,673
9,377
(4,412)
Share-based compensation expense
10,100
11,928
17,386
(1,828)
(5,458)
Intellectual property fees
1,944
1,744
3,819
200
(2,075)
Depreciation and facility costs
1,910
2,110
2,051
(200)
59
Information technology costs
1,129
1,101
6,526
28
(5,425)
Severance costs
—
1,223
361
(1,223)
862
Financial advisory fees
—
—
3,800
—
(3,800)
Other expenses
8,794
5,947
4,576
2,847
1,371
Total selling, general and administrative expenses
$
65,456
$
52,657
$
74,591
$
12,799
$
(21,934)
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● We incurred $24.9 million in employee and contractor-related expenses in the year ended December 31, 2025 compared to $21.3 million in 2024 and $24.4 million in 2023. The $3.6 million increase in 2025, compared to 2024, was primarily due to higher personnel-related expenses incurred in preparation for a potential commercial launch of AMT-130 in the U.S. The decrease in 2024 of $3.1 million, compared to 2023, was primarily related to the reduction in personnel and contractors following the divestment of our commercial manufacturing activities in July 2024, and the reorganization activities in October 2023 and August 2024;
● We incurred $16.6 million in professional fees in the year ended December 31, 2025 compared to $7.3 million in 2024 and $11.7 million in 2023. We regularly incur accounting, audit and legal fees associated with operating as a public company. The increase to professional fees in 2025 of $9.4 million included $6.5 million of expenses incurred in relation to the preparation of a potential commercial launch of AMT-130 in the U.S. In 2024, we incurred additional professional fees related to the divestment of our commercial manufacturing activities. In 2023, we incurred additional professional fees related to our global licensing agreement with Apic Bio, the Royalty Purchase Agreement and other corporate initiatives;
● We incurred $10.1 million of share-based compensation expenses in the year ended December 31, 2025 compared to $11.9 million in 2024 and $17.4 million in 2023. The decrease in 2025 of $1.8 million was primarily related to the divestment of our commercial manufacturing activities in July 2024 and a reduction in expense from the restructuring that occurred in August 2024. The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the divestment of our commercial manufacturing activities in July 2024, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of granted long-term incentive awards, partially offset by an increase in granted long-term incentive awards and costs associated with performance share units, which were granted in December 2021, and for which achievement of performance criteria was deemed probable in 2024;
● We incurred $1.9 million in intellectual property fees including registration and professional fees in the year ended December 31, 2025 compared to $1.7 million in 2024 and $3.8 million in 2023. The decrease of $2.1 million in 2024, compared to 2023, is primarily due to a decrease in professional fees;
● We incurred nil severance costs in the year ended December 31, 2025. We incurred $1.2 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization and $0.4 million of severance costs in 2023 related to an October 2023 reorganization; and,
● We incurred nil financial advisory fees pertaining to our licensing transaction with CSL Behring in the years ended December 31, 2025 and 2024 and $3.8 million in the year ended December 31, 2023.
● We incurred $8.8 million in other operating expenses in the year ended December 31, 2025 compared to $5.9 million in 2024 and $4.6 million in 2023. The increase in costs in 2025 of $2.8 million, compared 2024, was primarily due to an increase in other tax expenses recorded in 2025, partially offset by a decrease in other expenses relating to the Lexington Transaction in 2024.
Other items, net
In the year ended December 31, 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 periods in 2024 and 2023.
We recognized nil income related to our equity stake in VectorY B.V. in the years ended December 31, 2025 and 2024. In 2023, we recognized $0.8 million in other expense related to a reduction in the fair market value of our equity stake in VectorY B.V. following an October 2023 financing round.
In the year ended December 31, 2025, we recognized $5.7 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands and France compared to $5.6 million in 2024 and $5.3 million in 2023.
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In the year ended December 31, 2024, we recognized a $1.2 million net gain related to the Lexington Transaction, with no such amounts applicable in either 2025 or 2023.
Other income for the years ended December 31, 2025, 2024, and 2023 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility. We present expenses related to such income as other expense.
We recognized $6.3 million and $3.2 million in net other expenses during the year ended December 31, 2025 and 2024 and nil in 2023, 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.
Non-operating items, net
Our non-operating items, net, for the years ended December 31, 2025, 2024, and 2023 were as follows:
Years ended December 31,
2025
2024
2023
2025 vs 2024
2024 vs 2023
(in thousands)
Interest income
$
16,967
$
21,415
$
19,562
$
(4,448)
$
1,853
Interest expense - Royalty Financing Agreement
(54,099)
(50,865)
(26,933)
(3,234)
(23,932)
Interest expense - Hercules debt facility
(6,924)
(12,876)
(14,624)
5,952
1,748
Interest expense - other
(971)
—
—
(971)
—
Foreign currency gains / (losses), net
26,131
(10,507)
(1,691)
36,638
(8,816)
Changes in fair value of liability related to pre-funded warrants
12,405
—
—
12,405
—
Issuance costs - Pre-funded warrants
(1,500)
—
—
(1,500)
—
Total non-operating items, net
$
(7,991)
$
(52,833)
$
(23,686)
$
44,842
$
(29,147)
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized interest income of $17.0 million in 2025, $21.4 million in 2024 and $19.6 million in 2023. Our interest income decreased in 2025 by $4.4 million compared to 2024, and increased in 2024 by $1.9 million compared to 2023, primarily due to market interest rate developments and fluctuating balances of investment securities and cash on hand year over year.
In May 2023, uniQure biopharma entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. to sell certain current and future royalties due to uniQure biopharma from CSL Behring under the CSL Behring Agreement by and between uniQure biopharma and CSL Behring from the net sales of HEMGENIX. We recognized non-cash interest expenses related to the Royalty Financing Agreement of $54.1 million in 2025, $50.9 million in 2024 and $26.9 million in 2023, respectively.
We recognized interest expenses of $6.9 million in 2025, $12.9 million in 2024 and $14.6 million in 2023 related to the Hercules debt facility. Interest expenses decreased by $6.0 million in 2025 compared to 2024, primarily due to the $50.0 million repayment of the Hercules debt in July 2024, as well as a decrease in market interest rates. Interest expense decreased by $1.7 million in 2024, compared to 2023, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates.
We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S. dollars. We recognize foreign exchange results related to changes in these foreign currencies. In 2025, we recognized a net foreign currency gain of $26.1 million 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. We recognized a net foreign currency loss of $10.5 million in 2024 and $1.7 million in 2023.
We recognized a $10.9 million gain in 2025, net of issuance costs, related to a decrease in the fair value of the liability related to pre-funded warrants, compared to nil for the same periods in 2024 and 2023.
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Income tax expense
We recognized $5.6 million of income tax expense in 2025 compared to $2.4 million of income tax expense in 2024 and $1.9 million of income tax expense in 2023.
The $3.2 million increase of income tax expense in 2025 compared to 2024 is primarily attributable to the recognition of $3.9 million of current income tax expense related to the agreement reached with the Dutch tax authorities in 2025, to treat the $375.0 million upfront payment received in 2023 under the Royalty Financing Agreement as taxable income in that year. The income tax recorded in 2024 and 2023 primarily related to the consumption of net operating losses in the U.S. Until mid-2023, we also recognized deferred tax income in France related to the buildup of net operating losses by our French entity.
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Financial Position, Liquidity and Capital Resources
As of December 31, 2025, we had cash and cash equivalents, restricted cash and investment securities of $624.1 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-162, 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.
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 material cash requirements include the following contractual and other obligations:
Debt
As of December 31, 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 $21.9 million, with $4.8 million payable within the next 12 months.
Leases
We entered into lease arrangements for facilities, including corporate, laboratory and office space. As of December 31, 2025, we had fixed lease payment obligations of $21.7 million, with $4.6 million payable within the next 12 months. Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen, which reduced our fixed lease payment obligations by $20.7 million. As of December 31, 2025, we remained obligated under a guarantee of lease payments of $16.3 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.
Commitments related to the uniQure France Acquisition (nominal amounts)
In connection with the uniQure France Acquisition, 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 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 following the dosing of the first patient in Phase I/IIa clinical trial for AMT-260. As of December 31, 2025, our remaining commitment amounts include EUR 160.0 million ($188.0 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and EU. 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 December 31, 2025, of $1.18/€1.00. As of December 31, 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 License Sale to CSL Behring. We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.
Commitments related to the CSA and 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 December 31, 2025, our remaining minimum purchase commitments to Genezen amount to $16.2 million, with $10.3 million to be paid within the next 12 months. CSL Behring’s minimum purchase commitments to us over the next 12 months are $16.4 million, of which $16.2 million relates to such minimum purchase commitments to Genezen.
Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 2027. As of December 31, 2025 our remaining minimum purchase commitments amount to $7.3 million with $4.3 million to be paid within the next 12 months.
The table below summarizes our consolidated cash flow data for the years ended December 31, 2025, 2024, and 2023
Year ended December 31,
2025
2024
2023
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period
$
160,329
$
244,544
$
231,173
Net cash used in operating activities
(177,963)
(182,728)
(145,929)
Net cash (used in) / generated from investing activities
(321,619)
162,968
(205,686)
Net cash generated from / (used in) financing activities
415,398
(59,486)
362,721
Foreign exchange impact
5,657
(4,969)
2,265
Cash, cash equivalents and restricted cash at the end of period
$
81,802
$
160,329
$
244,544
We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V. in 1998, with the exception of our generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement. We continued to incur losses in the current period. We recorded a net loss of $199.0 million for the year ended December 31, 2025, and a net loss of $239.6 million and $308.5 million in the years ended December 31, 2024 and 2023, respectively. As of December 31, 2025, we had an accumulated deficit of $1,328.9 million.
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Sources of liquidity
From our first institutional venture capital financing in 2006 through to the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners, as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023 in connection with the Royalty Financing Transaction. Between July 2021 and July 2023, we have 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 Transaction) on net sales of HEMGENIX.
In 2025, we received aggregate net proceeds of $404.2 million, after deducting underwriting discounts and commissions and other offering expenses, through the completion of follow-on public offerings. In September 2025, we received aggregate net proceeds of $323.7 million in connection with 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 January and February 2025, we received aggregate net proceeds of $80.5 million, in connection with a follow-on public offering of 5.1 million ordinary shares at a public offering price of $17.00 per ordinary share.
We have amended the venture debt loan facility entered into with Hercules in June 2013 from time to time, most recently in September 2025. 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, 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, subject to approval by Hercules.
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.
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 December 31, 2025, no sales have been made under the ATM offering program.
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.
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Net Cash used in operating activities
Year ended December 31,
2025
2024
2023
(in thousands)
Cash flows from operating activities
Net loss
$
(198,971)
$
(239,556)
$
(308,478)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation, amortization and impairment
14,759
12,641
11,900
Amortization of discount on investment securities
(10,463)
(10,901)
(10,917)
Share-based compensation expense
17,679
22,258
35,093
Royalty financing agreement interest expense, net of interest paid
38,841
44,203
25,616
Deferred tax expense
1,588
2,429
1,921
Change in fair value of contingent consideration
6,247
(1,817)
15,895
Changes in fair value of liability related to pre-funded warrants
(12,405)
—
—
Unrealized foreign exchange (gains) / losses, net
(23,019)
15,415
(2,206)
Other items, net
(5,429)
(3,848)
4,721
Accounts receivable, prepaid expenses, and other current assets and receivables
(8,597)
(2,243)
(1,323)
Accounts payable
(2,667)
1,520
(4,169)
Accrued expenses, other liabilities, and operating leases
4,474
(5,642)
(5,328)
Contingent consideration milestone payments
—
(19,608)
(1,914)
Inventories
—
2,421
(6,740)
Contract asset related to CSL Behring milestone payment
—
—
100,000
Net cash used in operating activities
$
(177,963)
$
(182,728)
$
(145,929)
Net cash used in operating activities was $178.0 million for the year ended December 31, 2025 and consisted of a net loss of $199.0 million, adjusted for non-cash items including depreciation and amortization expense of $14.8 million, amortization of the discount on investment securities of $10.5 million, share-based compensation expense of $17.7 million, $38.8 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $1.6 million, changes in the fair value of contingent consideration of $6.2 million, changes in the fair value of the liability related to pre-funded warrants of $12.4 million and unrealized foreign exchange gains of $23.0 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $6.8 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $8.6 million and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $1.8 million.
Net cash used in operating activities was $182.7 million for the year ended December 31, 2024, and consisted of a net loss of $239.6 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $12.6 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $22.3 million, $44.2 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $2.4 million, $1.8 million change in the fair value of contingent consideration and unrealized foreign exchange losses of $15.4 million. Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.2 million. There was a decrease in inventory balances of $2.4 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to an increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals. Net cash used in operating activities also includes a payment for a contingent consideration payment of $19.6 million.
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Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million. Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $80.5 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $1.3 million. There was a net decrease in contract assets of $100.0 million related to the collection of the $100.0 million milestone due from CSL Behring in July 2023 . There was an increase in inventory balances of $6.7 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $9.5 million, primarily related to a decrease of $4.2 million in accounts payable and a decrease of $5.3 million related to various accruals. Net cash used in operating activities also includes a payment for a contingent consideration milestone of $1.9 million.
Net cash (used in) / generated from investing activities
In 2025, we used $321.6 million in our investing activities, compared to $163.0 million generated from investing activities in 2024 and $205.7 million used in investing activities in 2023.
Year ended December 31,
2025
2024
2023
(in thousands)
Cash flows from investing activities
Proceeds from maturity of debt securities
$
337,195
$
534,498
$
167,907
Investment in debt securities
(658,375)
(359,841)
(366,439)
Capital expenditures - European sites
(406)
(1,783)
(3,389)
Capital expenditures - Lexington sites
(33)
(1,585)
(3,765)
Divestment of commercial manufacturing facility
—
(8,321)
—
Net cash (used in) / generated from investing activities
$
(321,619)
$
162,968
$
(205,686)
Net cash generated from / (used in) financing activities
In 2025, we generated $415.4 million from our financing activities compared to $59.5 million used in financing activities in 2024 and $362.7 million generated from financing activities in 2023.
Year ended December 31,
2025
2024
2023
(in thousands)
Cash flows from financing activities
Proceeds from follow-on public offerings of ordinary shares, 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 options and purchase plans
11,162
2,123
308
Repayment of long-term debt
—
(53,050)
—
Contingent consideration milestone payments
—
(8,559)
(7,649)
Proceeds from royalty financing agreement, net of issuance costs
—
—
370,062
Net cash generated from / (used in) financing activities
$
415,398
$
(59,486)
$
362,721
In December 2024, following the dosing of the first patient in Phase I/IIa clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France based on contractually defined milestones. EUR 26.8 million ($28.2 million) of this payment related to a contingent consideration of which EUR 8.2 million ($8.6 million) was classified as cash flows from financing activities and EUR 18.7 million ($19.6 million) was classified as a net cash flow used in operating activities.
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In September 2023, following the FDA’s clearance of the IND application for AMT-260, we made a payment of EUR 10.0 million ($10.7 million) to the former shareholders of uniQure France based on contractually defined milestones. EUR 8.9 million ($9.6 million) of this payment related to a contingent consideration of which EUR 7.2 million ($7.6 million) was classified as cash flows from financing activities and EUR 1.8 million ($1.9 million) was classified as a net cash flow used in operating activities.
Funding requirements
Our future capital requirements will depend on many factors, including but not limited to:
● Investments to develop AMT-130, AMT-191 and AMT-260 beyond completing the ongoing trials;
● activities to prepare for the potential commercialization of AMT-130 for Huntington’s disease, if we can align on the requirements for marketing authorization with the respective regulatory authorities;
● earnout payments we might owe the former shareholders of uniQure France, 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;
● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements;
● 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.
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