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The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”). Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the 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”), and Fabry disease.
Business Developments
Financing
In June 2026, we received net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 5.7 million ordinary shares, at a public offering price of $45.50 per ordinary share.
Hercules Loan Amendment
In July 2026, we entered into an amendment to the $175.0 million senior secured term loan facility (the “2026 Amended Facility”) with Hercules Capital, Inc. (“Hercules”). The 2026 Amended Facility, among other things, extends the period we can draw the $100.0 million term loan tranche from June 2027 to September 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility otherwise remain unchanged.
Recent Product Candidate Developments
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 a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
We are currently conducting Phase I/II clinical trials of AMT-130 in the U.S. and Europe. We completed the enrollment of all 26 patients in the first two cohorts of our U.S. study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023. In 2025, we completed enrollment of all 12 patients in the third cohort, and we treated six patients with the high-dose of AMT-130 in a fourth cohort to evaluate the safety and efficacy of AMT-130 in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.
Data from Phase I/II Clinical Studies
In September 2025, we announced positive topline data from the three-year analysis of cohorts one and two of the ongoing Phase I/II studies of AMT-130 for the treatment of Huntington’s disease. We analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 36 months of follow-up and were evaluated at that time point. Outcomes for each dose group were compared to a propensity score-matched external control drawn from the Enroll-HD natural history data set (n=940 for high-dose; n=626 for low-dose).
Topline 36-month efficacy results for patients receiving high-dose AMT-130 were as follows (data cutoff as of June 30, 2025):
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● A statistically significant 75% slowing of disease progression as measured by composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) (p=0.003), which met the primary endpoint of the study. Treated patients had a mean change in cUHDRS from baseline of -0.38 compared to a change of -1.52 for patients in the propensity score-matched external control.
● A statistically significant 60% slowing of disease progression as measured by Total Function Capacity (“TFC”) (p=0.033), which met a key secondary endpoint of the study. Treated patients had a mean change in TFC from baseline of -0.36 compared to a change of -0.88 for patients in the propensity score-matched external control.
● 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. There have been five drug-related serious adverse events (“SAEs”) reported across all cohorts, and the most common adverse events in the treatment groups were related to the administration procedure.
Regulatory Update
From November 2024 through April 2025, we held three Type B meetings with the U.S. Food and Drug Administration (the “FDA”). As part of these interactions, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis of a Biologics License Application (“BLA”) submission under the FDA’s accelerated approval pathway. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and reductions in CSF NfL may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.
In October 2025, we met with the FDA at a pre-BLA meeting to discuss the application for AMT-130. In December 2025, we announced that in the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 were currently unlikely to provide the primary evidence to support a BLA submission.
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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 could not agree that data from the Phase I/II studies, compared to an external control, were sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.
In March 2026, we held a successful pre-submission meeting with the United Kingdom’s (“UK”) Medicines and Healthcare products Regulatory Agency (“MHRA”) regarding AMT-130, and the regulatory submission is progressing as planned for the third quarter of 2026.
In June 2026, we held a Type B meeting with the FDA. Official meeting minutes received in July 2026 confirmed that we and the FDA reached alignment that a BLA submission under the accelerated approval pathway for AMT-130, based on the existing clinical data, is reasonable. In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of a randomized standard-of-care control design instead of a sham procedure. The FDA also stated that, in accordance with the FDA’s draft public guidance for accelerated approvals, the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway, and potentially fully enrolled, at the time of accelerated approval. Discussions with the FDA to align on the confirmatory study design and analysis are underway, and we expect to submit a BLA in the third quarter of 2026.
Temporal lobe epilepsy program (AMT-260)
We are conducting a Phase I/IIa clinical trial, GenTLE, of AMT-260 for the treatment of MTLE in the U.S. GenTLE is a multicenter, open-label trial with two dosing cohorts of at least six patients each to assess safety, tolerability, and initial efficacy of AMT-260 in patients with refractory MTLE.
In September 2025, we completed enrollment of the first three patients in the first cohort administering AMT-260 to patients with lesions in the non-dominant hemisphere of the brain. Following a review by the independent data monitoring committee (“IDMC”), we expanded the first cohort into MTLE in the dominant hemisphere and initiated a second cohort. We completed enrollment of six patients into the first cohort in 2025. We also initiated enrollment of a second cohort in 2025.
In June 2026, we announced preliminary data on the first cohort in GenTLE. As of the May 29, 2026 data cutoff date, three of six patients in the first, low-dose cohort (1x1012 gc/mL) achieved meaningful reductions in disabling seizures during months four through six of follow-up, ranging from a 79% to 100% decline from baseline. The remaining three patients in the low-dose cohort experienced variable changes in disabling seizures during months four through six of follow-up, ranging from a 33% decrease to a 36% increase compared to baseline. As of the June 19, 2026, there have been no SAEs related to AMT-260 or the surgical procedure reported. All reported adverse events in the low dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2). No immunosuppression was required.
Fabry disease program (AMT-191)
We are conducting a Phase I/II clinical trial of AMT-191 for the treatment of Fabry disease. The multicenter, open-label clinical trial consists of three dose-ranging cohorts of three or more patients each to assess safety, tolerability, and efficacy of AMT-191 in patients with Fabry disease.
In June 2026, we presented updated preliminary data from the Phase I/II study of AMT-191 for Fabry disease. The data, based on a March 15, 2026 data cutoff date, included patient follow-up ranging from three months to more than 18 months and consisted of the following:
● Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 1.0- to 16.2-fold above mean normal range (1.38-8.66 nmol; mean normal of 3.57 nmol) at the lowest dose, 14.5- to 229.6-fold at the mid dose, and 58.7- to 143.6-fold at the highest dose.
● All 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”).
● Plasma lyso-Gb3 levels were stable post-dose across all dose cohorts, regardless of ERT status through the cutoff date.
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AMT-191 continued to show a manageable safety profile. No SAEs related to AMT-191 were observed at the 4x1013 gc/kg (“mid-dose”) and 2x1013 gc/kg (“low-dose”) doses. No additional SAEs were observed at the 6x1013 gc/kg (“high-dose”) dose beyond those previously reported in September 2025 in two patients.
Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity. These elevations resolved as of the end of May 2026 following a course of immunosuppression as per the study protocol.
Financial Overview
Key components of our results of operations include the following:
Three months ended June 30, Six months ended June 30,
2026 2025 2026 2025
(in thousands) (in thousands)
Total revenues $ 5,841 $ 5,262 $ 9,403 $ 6,829
Cost of license revenues (350) (656) (569) (853)
Research and development expenses (33,964) (35,383) (63,140) (71,523)
Selling, general and administrative expenses (17,367) (13,500) (37,435) (24,408)
Net loss (81,059) (37,719) (134,594) (81,356)
As of June 30, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $810.3 million and $622.5 million, respectively. We had a net loss of $81.1 million and $134.6 million in the three and six months ended June 30, 2026, compared to a net loss of $37.7 million and $81.4 million for the same periods in 2025. As of June 30, 2026 and December 31, 2025, we had accumulated deficits of $1,463.5 million and $1,328.9 million, respectively. See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Critical Accounting Policies and Estimates
In preparing our unaudited consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the “SEC”) we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. 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 six months ended June 30, 2026.
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;
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● 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 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 selling, general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors’ and officers’ liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.
Other items, net
Our other income generally consists of payments received to subsidize our research and development efforts and income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility.
In 2024, as part of the divestment of our commercial manufacturing operations to Genezen Holdings Inc. and its subsidiary Genezen MA, Inc. (together “Genezen”), we entered into various service agreements, including a commercial supply agreement (the “Genezen CSA”). In April 2026, we entered into an agreement to terminate the Genezen CSA. Pursuant to the termination agreement, our obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA terminate once contractually specified batches have been supplied.
Our other expenses consist of costs incurred under the Genezen CSA. These include costs related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring LLC (“CSL Behring”), amortization of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA, and release of liabilities related to expected net losses associated with the remaining minimum purchase commitments under the Genezen CSA.
Additionally, other expenses also consist of expenses we incur in relation to our subleasing income.
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Results of Operations
Comparison of the three months ended June 30, 2026 and 2025
The following table presents a comparison of our results of operations for the three months ended June 30, 2026 and 2025:
Three months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
License revenues $ 5,841 $ 5,262 $ 579
Total revenues 5,841 5,262 579
Operating expenses:
Cost of license revenues (350) (656) 306
Research and development expenses (33,964) (35,383) 1,419
Selling, general and administrative expenses (17,367) (13,500) (3,867)
Total operating expenses (51,681) (49,539) (2,142)
Other income 1,598 2,597 (999)
Other expense (7,961) (2,185) (5,776)
Loss from operations (52,203) (43,865) (8,338)
Non-operating (expense) / income, net (27,015) 6,571 (33,586)
Net loss before income tax expense $ (79,218) $ (37,294) $ (41,924)
Income tax expense (1,841) (425) (1,416)
Net loss $ (81,059) $ (37,719) $ (43,340)
License revenues
We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended June 30, 2026 and 2025, we recognized $5.8 million and $5.3 million of license revenues, respectively.
R&D expense
R&D expenses for the three months ended June 30, 2026 were $34.0 million, compared to $35.4 million for the same period in 2025. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.
Three months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Huntington's disease (AMT-130) $ 6,280 $ 10,517 $ (4,237)
Temporal lobe epilepsy (AMT-260) 4,168 1,955 2,213
Amyotrophic lateral sclerosis (AMT-162) 2,503 1,098 1,405
Fabry disease (AMT-191) 2,177 1,255 922
Programs in preclinical development and platform related expenses 1,883 405 1,478
Total direct research and development expenses $ 17,011 $ 15,230 $ 1,781
Employee and contractor-related expenses 9,111 9,523 (412)
Facility expenses 2,402 3,853 (1,451)
Share-based compensation expense 1,888 2,758 (870)
Information technology costs 966 594 372
Fair value changes related to contingent consideration 1,256 2,254 (998)
Other expenses 1,330 1,171 159
Total other research and development expenses $ 16,953 $ 20,153 $ (3,200)
Total research and development expenses $ 33,964 $ 35,383 $ (1,419)
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Direct research and development expenses
Huntington’s disease (AMT-130)
In the three months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $6.3 million and $10.5 million respectively. The decrease of $4.2 million was primarily related to lower manufacturing process validation costs, which were $0.8 million in the current year period, compared to $4.0 million in the prior year period. Additionally, clinical trials costs decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and Marketing Authorization Application (“MAA”) submissions for AMT-130 in the United States and the United Kingdom, respectively.
Temporal lobe epilepsy (AMT-260)
In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $4.2 million and $2.0 million, respectively. The current year period included $3.7 million of clinical expenses and $0.3 million of CMC expenses, respectively, compared to $1.7 million and $0.2 million, respectively, in the prior year period.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $2.5 million and $1.1 million, respectively. These costs were primarily related to our Phase I/II clinical trial.
Fabry disease (AMT-191)
In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $2.2 million and $1.3 million, respectively. These costs were primarily related to our Phase I/II trial.
Preclinical programs & platform development
In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $1.9 million and $0.4 million, respectively.
Other research & development expenses
● We incurred $9.1 million in personnel and contractor-related expenses in the three months ended June 30, 2026, compared to $9.5 million for the same period in 2025;
● We incurred $2.4 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended June 30, 2026 compared to $3.9 million in the same period in 2025;
● We incurred $1.9 million in share-based compensation expenses in the three months ended June 30, 2026, compared to $2.8 million for the same period in 2025. The decrease of $0.9 million was primarily driven by lower costs related to performance stock units during the current year period; and
● We incurred a $1.3 million loss related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the three months ended June 30, 2026, compared to a $2.3 million loss for the same period in 2025. The $1.0 million favorable change reflects that the prior period included an adjustment to the expected timing of achieving future milestones that did not recur in the current year period.
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Selling, general and administrative expenses
Selling, general and administrative expenses for the three months ended June 30, 2026 were $17.4 million, compared to $13.5 million for the same period in 2025.
Three months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Employee and contractor-related expenses $ 8,878 $ 5,397 $ 3,481
Share-based compensation expense 3,128 2,326 802
Professional fees 1,592 3,428 (1,836)
Intellectual property fees 983 281 702
Depreciation and facility costs 508 578 (70)
Information technology costs 389 193 196
Other expenses 1,889 1,297 592
Total selling, general and administrative expenses $ 17,367 $ 13,500 $ 3,867
● We incurred $8.9 million in personnel and contractor-related expenses in the three months ended June 30, 2026, compared to $5.4 million in the same period in 2025. The $3.5 million increase was primarily driven by of a higher headcount following employee recruitment in the second half of 2025 to support the potential commercial launches of AMT-130;
● We incurred $3.1 million in share-based compensation expenses in the three months ended June 30, 2026, compared to $2.3 million in the same period in 2025. The $0.8 million increase was primarily driven by the higher fair value of awards outstanding during the current year period, in comparison with the prior period;
● We incurred $1.6 million in professional fees in the three months ended June 30, 2026 compared to $3.4 million in the same period in 2025. The $1.8 million decrease was primarily a result of lower costs in connection with the potential commercial launches of AMT-130; and
● We incurred $1.9 million in other expenses in the three months ended June 30, 2026, compared to $1.3 million in the same period in 2025.
Other income and expense
Three months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Research and development grants from Dutch authorities $ 1,164 $ 1,392 $ (228)
Sublease income, net 337 347 (10)
Supply of HEMGENIX® to CSL Behring (7,695) (1,731) (5,964)
Other income, net (169) 404 (573)
Total other items, net $ (6,363) $ 412 $ (6,775)
In April 2026, we entered into an agreement to terminate the Genezen CSA. In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset within Other expense.
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Other non-operating items, net
Our other non-operating items, net, for the three months ended June 30, 2026 and June 30, 2025 were as follows:
Three months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Interest income $ 5,051 $ 3,524 $ 1,527
Interest expense - Royalty Financing Agreement (12,963) (13,770) 807
Interest expense - Hercules loan facility (1,403) (1,821) 418
Foreign currency (losses) / gains, net (1,730) 18,638 (20,368)
Changes in fair value of liability related to pre-funded warrants (15,970) — (15,970)
Total non-operating items, net $ (27,015) $ 6,571 $ (33,586)
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $5.1 million in interest income in the three months ended June 30, 2026, compared to $3.5 million in the same period in 2025. The $1.5 million increase was primarily due to higher average balances of our investment securities held during the current year period, compared to the prior year period.
In May 2023, uniQure biopharma B.V. entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. to sell certain current and future royalties due to uniQure biopharma B.V. from CSL Behring from the net sales of HEMGENIX® pursuant to the CSL Behring Agreement. We recognized non-cash interest expenses related to the Royalty Financing Agreement of $13.0 million and $13.8 million in the three months ended June 30, 2026 and 2025, respectively.
We recognized interest expense related to the Hercules loan facility of $1.4 million and $1.8 million in the three months ended June 30, 2026 and June 30, 2025, respectively. The $0.4 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.
We conduct transactions and hold monetary assets and liabilities denominated in foreign currencies, principally the euro and the U.S. dollar. Monetary balances are remeasured at period-end exchange rates, and resulting foreign currency transaction gains and losses are recorded in the Unaudited Consolidated Statements of Operations and Comprehensive Loss as incurred.
We recognized a net foreign currency loss of $1.7 million during the three months ended June 30, 2026, compared to a net gain of $18.6 million during the same period in 2025. The foreign currency movements relate to our Hercules loan facility, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.
In connection with our September 2025 follow-on public offering, we issued pre-funded warrants that are classified as a liability and measured at fair value each reporting period. We recognized a $16.0 million loss in the three months ended June 30, 2026, reflecting an increase in the fair value of this liability, compared to nil for the same period in 2025.
Income tax expense
We recognized $1.8 million of deferred tax expense in the three months ended June 30, 2026, and $0.4 million of deferred tax expense for the same period in 2025.
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Results of Operations
Comparison of the six months ended June 30, 2026 and 2025
The following table presents a comparison of our results of operations for the six months ended June 30, 2026 and 2025:
Six months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Total revenues $ 9,403 $ 6,829 $ 2,574
Operating expenses:
Cost of license revenues (569) (853) 284
Research and development expenses (63,140) (71,523) 8,383
Selling, general and administrative expenses (37,435) (24,408) (13,027)
Total operating expenses (101,144) (96,784) (4,360)
Other income 3,230 10,903 (7,673)
Other expense (9,412) (4,144) (5,268)
Loss from operations (97,923) (83,196) (14,727)
Non-operating (expense) / income, net (34,342) 2,761 (37,103)
Loss before income tax expense $ (132,265) $ (80,435) (51,830)
Income tax expense (2,329) (921) (1,408)
Net loss $ (134,594) $ (81,356) $ (53,238)
License revenues
We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the six months ended June 30, 2026 and 2025, we recognized $9.4 million and $6.8 million of license revenues, respectively.
R&D expense
R&D expenses for the six months ended June 30, 2026 were $63.1 million, compared to $71.5 million for the same period in 2025. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.
Six months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Huntington's disease (AMT-130) $ 14,260 $ 18,747 $ (4,487)
Temporal lobe epilepsy (AMT-260) 6,995 4,030 2,965
Fabry disease (AMT-191) 3,716 3,442 274
Amyotrophic lateral sclerosis (AMT-162) 3,397 2,929 468
Programs in preclinical development and platform related expenses 3,017 1,685 1,332
Total direct research and development expenses $ 31,385 $ 30,833 $ 552
Employee and contractor-related expenses 18,171 19,682 (1,511)
Facility expenses 6,021 8,403 (2,382)
Share-based compensation expense 3,865 5,221 (1,356)
Information technology costs 2,056 1,276 780
Fair value changes related to contingent consideration (82) 3,470 (3,552)
Other expenses 1,724 2,638 (914)
Total other research and development expenses $ 31,755 $ 40,690 $ (8,935)
Total research and development expenses $ 63,140 $ 71,523 $ (8,383)
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Direct research and development expenses
Huntington’s disease (AMT-130)
In the six months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $14.3 million and $18.7 million respectively. The decrease of $4.5 million was primarily driven by lower costs related to clinical trials of $9.0 million in the current period, compared to $11.2 million in the prior year period. Additionally, manufacturing process validation costs were decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and MAA submissions for AMT-130 in the United States and the United Kingdom, respectively.
Temporal lobe epilepsy (AMT-260)
In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $7.0 million and $4.0 million, respectively. The current period included costs of $6.3 million related to clinical trials and $0.5 million related to CMC development, compared to $3.3 million and $0.7 million, respectively, in the prior year period.
Fabry disease (AMT-191)
In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $3.7 million and $3.4 million, respectively. These costs primarily related to the Phase I/II trial.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $3.4 million and $2.9 million, respectively. These costs primarily related to the Phase I/II trial.
Preclinical programs & platform development
In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $3.0 million and $1.7 million, respectively.
Other research & development expenses
● We incurred $18.2 million in personnel and contractor-related expenses in the six months ended June 30, 2026, compared to $19.7 million for the same period in 2025. The decrease of $1.5 million was primarily due to lower personnel-related expenses in the current year period;
● We incurred $6.0 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the six months ended June 30, 2026 compared to $8.4 million in the same period in 2025. The decrease of $2.4 million was primarily driven by a revision to the estimated useful lives of certain assets, resulting in higher depreciation recognized during the prior year period;
● We incurred $3.9 million in share-based compensation expenses in the six months ended June 30, 2026, compared to $5.2 million for the same period in 2025. The decrease of $1.3 million was primarily driven by lower costs related to performance stock units during the current year period;
● We incurred a $0.1 million gain related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the six months ended June 30, 2026, compared to a $3.5 million loss for the same period in 2025. The $3.6 million favorable change was primarily driven by movements in the discount rate and the timing of future milestone achievements; and
● We incurred $1.7 million of other expenses for the six months ended June 30, 2026, compared to $2.6 million for the same period in 2025.
Selling, general and administrative expenses
Selling, general and administrative expenses for the six months ended June 30, 2026 were $37.4 million, compared to $24.4 million for the same period in 2025.
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Six months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Employee and contractor-related expenses $ 18,733 $ 10,882 $ 7,851
Share-based compensation expense 6,241 4,273 1,968
Professional fees 5,247 5,331 (84)
Intellectual property fees 1,855 564 1,291
Information technology costs 909 474 435
Depreciation and facility costs 845 958 (113)
Other expenses 3,605 1,926 1,679
Total selling, general and administrative expenses $ 37,435 $ 24,408 $ 13,027
● We incurred $18.7 million in personnel and contractor-related expenses in the six months ended June 30, 2026, compared to $10.9 million in the same period in 2025. The $7.8 million increase was primarily driven by higher headcount following employee recruitment in the second half of 2025 to support the potential commercial launches of AMT-130;
● We incurred $5.2 million in professional fees in the six months ended June 30, 2026 compared to $5.3 million in the same period in 2025;
● We incurred $6.2 million in share-based compensation expenses in the six months ended June 30, 2026, compared to $4.3 million in the same period in 2025. The $1.9 million increase was primarily driven by the higher fair value of awards outstanding during the current year period, in comparison with the prior period;
● We incurred $1.9 million in intellectual property fees in the six months ended June 30, 2026 compared to $0.6 million in the same period in 2025. The $1.3 million increase was primarily driven by higher registration and professional fees; and
● We incurred $3.6 million in other expenses in the six months ended June 30, 2026, compared to $1.9 million in the same period in 2025.
Other income and expense
Six months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Research and development grants from Dutch authorities 2,122 3,111 (989)
Sublease income, net 606 371 235
Sale of critical reagents to Genezen $ — $ 6,000 $ (6,000)
Supply of HEMGENIX® to CSL Behring (8,880) (3,256) (5,624)
Other income, net (30) 533 (563)
Total other items, net $ (6,182) $ 6,759 $ (12,941)
In April 2026, we entered into an agreement to terminate the Genezen CSA. In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset in Other expense.
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Other non-operating items, net
Our other non-operating items, net, for the six months ended June 30, 2026 and June 30, 2025 were as follows:
Six months ended June 30,
2026 2025 2026 vs 2025
(in thousands)
Interest income $ 10,280 $ 7,651 $ 2,629
Interest expense - Royalty Financing Agreement (25,584) (27,079) 1,495
Interest expense - Hercules loan facility (2,814) (3,621) 807
Foreign currency (losses) / gains, net (4,024) 25,810 (29,834)
Changes in fair value of liability related to pre-funded warrants (12,201) — (12,201)
Total Other non-operating items, net $ (34,342) $ 2,761 $ (37,103)
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $10.3 million in interest income in the six months ended June 30, 2026, compared to $7.7 million in the same period in 2025. The $2.6 million increase was primarily due to higher average balances of investment securities held during the current year period, compared to the prior year period.
We recognized interest expense related to the Hercules loan facility of $2.8 million in the six months ended June 30, 2026, compared to $3.6 million in the same period in 2025. The $0.8 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.
We recognized a net foreign currency loss of $4.0 million in the six months ended June 30, 2026, compared to a net gain of $25.8 million in the same period in 2025. The foreign currency movements related to our borrowings from Hercules, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.
Income tax expense
We recognized $2.3 million of deferred tax expense in the six months ended June 30, 2026, compared to $0.9 million in the same period in 2025.
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Financial Position, Liquidity and Capital Resources
As of June 30, 2026, we had cash and cash equivalents, restricted cash and investment securities of $811.9 million. We believe these resources will be sufficient to fund our projected operating expenses into 2030, including costs associated with the commercial launches and confirmatory study of AMT-130, ongoing clinical trials of AMT-130, AMT-191 and AMT-260, and potential investments to advance certain pipeline candidates into late stage development. The amount and timing of our actual expenditures may vary significantly depending on the commencement and design of any new clinical studies that we may conduct. We have based our estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, and as such, we may require additional funding.
This includes up to $100.0 million in additional availability under our Hercules senior secured term loan facility, which becomes accessible upon BLA approval for AMT-130 prior to September 30, 2027, subject to confirmatory trial requirements remaining on track. Additional funding could also include a combination of public equity offerings, collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible. 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 June 30, 2026, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million. The loan has an interest-only period until October 1, 2028 and we are contractually required to repay the $50.0 million in equal installments between October 1, 2028 and October 1, 2030. The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to October 1, 2028. The interest-only period will be further extended to October 1, 2030 if certain commercial milestones are met prior to March 31, 2029. Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $19.5 million, with $5.4 million payable within the next 12 months.
Leases
We have entered into lease arrangements for facilities, including corporate, laboratories and office space. As of June 30, 2026, we had fixed lease payment obligations of $22.9 million, with $5.2 million payable within the next 12 months. Following the closing of the Lexington Transaction, we assigned our lease for our prior manufacturing facility in Lexington, MA to Genezen. As of June 30, 2026, we remain obligated under a guarantee of lease payments of $14.0 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.
Commitments related to uniQure France SAS acquisition (nominal amounts)
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. As of June 30, 2026, our remaining commitment amounts include EUR 160.0 million ($182.3 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and European Union. The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestones will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of June 30, 2026 of $1.14/€1.00. As of June 30, 2026, we expect these obligations will become payable between 2030 and 2034. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.
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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 will collect under the CSL Behring Agreement.
The table below summarizes our consolidated cash flow data for the six months ended June 30, 2026 and 2025:
Six months ended June 30,
2026 2025
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period $ 81,801 $ 160,329
Net cash used in operating activities (66,819) (83,995)
Net cash generated from investing activities 151,819 93,172
Net cash generated from financing activities 249,666 80,714
Foreign exchange impact (1,906) 5,052
Cash, cash equivalents and restricted cash at the end of period $ 414,561 $ 255,272
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 continued to incur losses in the current period. We recorded a net loss of $81.1 million and $134.6 million in the three and six months ended June 30, 2026, compared to a net loss of $37.7 million and $81.4 million during the same period in 2025. As of June 30, 2026, we had an accumulated deficit of $1,463.5 million.
Sources of liquidity
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 June 2026, we received aggregate net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, following the completion of a follow-on public offering of 5.7 million ordinary shares at a public offering price of $45.50 per ordinary share.
We are subject to certain covenants under the senior secured term loan facility with Hercules and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business. 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.
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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 $66.8 million for the six months ended June 30, 2026 and consisted of a net loss of $134.6 million, adjusted for non-cash items including depreciation and amortization expense of $7.9 million, amortization of the discount on investment securities of $7.9 million, share-based compensation expense of $10.1 million, $17.4 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $2.3 million, changes in the fair value of contingent consideration of $0.1 million, changes in the fair value of the liability related to pre-funded warrants of $12.2 million and unrealized foreign exchange losses of $8.3 million. Net cash used in operating activities also included favorable changes in operating assets and liabilities of $20.0 million. There was a net decrease in accounts receivable, accrued income, prepaid expenses, and other current assets and receivables of $8.8 million. There was a net increase in accounts payable, accrued expenses, and operating leases of $11.1 million.
Net cash used in operating activities was $84.0 million for the six months ended June 30, 2025 and consisted of net loss of $81.4 million adjusted for non-cash items, including depreciation and amortization expense of $8.2 million, amortization of the discount on investment securities of $4.2 million, share-based compensation expense of $9.5 million, $20.6 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $0.9 million, changes in the fair value of contingent consideration of $3.5 million, and unrealized foreign exchange gains of $25.2 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $12.3 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $6.8 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $5.6 million.
Net cash generated from investing activities
In the six months ended June 30, 2026, we generated $151.8 million from our investing activities compared to $93.2 million generated during the same period in 2025.
Six months ended June 30,
2026 2025
(in thousands)
Cash flows from investing activities
Proceeds from maturity of debt securities $ 384,981 $ 213,763
Investment in debt securities (232,550) (120,205)
Capital expenditures (612) (386)
Net cash generated from investing activities $ 151,819 $ 93,172
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Net cash generated from financing activities
In the six months ended June 30, 2026, net cash generated from financing activities was $249.7 million, compared to net cash generated from financing activities of $80.7 million in the same period in 2025.
Six months ended June 30,
2026 2025
(in thousands)
Cash flows from financing activities
Proceeds from follow-on public offering of ordinary shares, net of issuance costs $ 242,681 $ 80,511
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans 6,985 203
Net cash generated from financing activities $ 249,666 $ 80,714
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;
● investments required to support the MAAs of AMT-130 or generate confirmatory evidence in conjunction with obtaining accelerated approval;
● investments in the late stage development of AMT-191 and AMT-260;
● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to achieving specific development and regulatory milestones;
● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;
● 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.