uniQure N.V.
For the period ended Dec 31, 2025. Filed Mar 2, 2026.
View on SEC.govAbout this filing
A company's yearly report to the SEC, with audited financial statements. It is the fullest account a company gives of its business, its finances and the risks it sees.
FY2025 · Filed Mar 2, 2026
QURE$21.70+3.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-K
Period ended 2024-12-31; filed 2025-02-27.
0001558370-25-001699
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-K
Period ended 2025-12-31; filed 2026-03-02.
0001104659-26-021898
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 145 previous paragraphs and 128 current paragraphs.
46 unchanged; 55 reworded; 26 added; 43 removed; 1 previous and 1 current paragraphs rearranged.
Tables are not compared: 65 previous and 63 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (26)
Current report
Clinical Data
Current report
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).
Current report
Regulatory Updates
Current report
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.
Current report
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.
Current report
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.
Current report
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.
Current report
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.
Current report
Public offerings
Current report
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.
Current report
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.
Current report
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.
Current report
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.
Current report
There are no other recently issued accounting pronouncements pending adoption that are applicable or expected to have a material impact.
Current report
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.
Current report
Non-operating items, net
Current report
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.
Current report
Income tax expense
Current report
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.
Current report
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.
Current report
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.
Current report
Commitments related to the uniQure France Acquisition (nominal amounts)
Current report
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.
Current report
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.
Current report
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.
Current report
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.
Removed paragraphs (43)
Previous report
RMAT Designation
Previous report
In June 2024, we announced that the FDA granted us RMAT designation for AMT-130.
Previous report
Updated Interim Data
Previous report
In July 2024, we announced updated interim clinical data, including up to 24 months of follow-up data from 29 patients enrolled in the U.S. study and the European study as of a March 31, 2024 cut-off date.
Previous report
Regulatory Alignment
Previous report
In December 2024, following our initial Type B meeting, we announced that we had reached agreement with FDA on key elements of an accelerated approval pathway for AMT-130.
Previous report
In November 2024, we announced that the that the first patient was dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mTLE. In addition, the FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere.
Previous report
In August 2024, we announced that the first patient was dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease. In February 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.
Previous report
In October 2024, we announced the first patient was dosed in a Phase I/IIa clinical trial of AMT-162 for the treatment of SOD1-ALS. In January 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.
Previous report
Sale of commercial manufacturing activities
Previous report
In June 2024, we and Genezen entered into the Lexington Transaction, which closed in July 2024. Genezen acquired manufacturing facility including equipment and related manufacturing operations with a carrying value of $15.2 million, inventory with a carrying value of $8.8 million and certain other assets (including allocated goodwill) with a carrying value of $2.8 million associated with the Lexington Facility on Closing.
Previous report
As consideration for the Lexington Transaction, we received from Genezen (i) shares of newly issued Series C preferred stock of Genezen Holdings Inc. valued at $12.5 million, which are convertible into common stock and will accrue an 8.0% per annum cumulative dividend, (ii) a convertible promissory note with a nominal amount of $12.5 million, bearing interest at 8.0% per annum and maturing 63 months following the date of issuance valued at $13.3 million and (iii) a right to purchase HEMGENIX® at terms considered favorable to market terms (the “Consideration”) valued at $16.7 million. We also included a firm purchase commitment liability, valued at $8.8 million for our customer contract with CSL Behring inherently related to the right to purchase HEMGENIX® in the consideration received.
Previous report
We recorded a $1.2 million gain from the divestment. We paid a total of $8.3 million to Genezen and CSL Behring related to adjustments of working capital and to obtain consent to proceed with the divestiture.
Previous report
In connection with the Closing, we, Genezen and the landlord of the Lexington Facility entered into an agreement for us to assign and Genezen to assume the existing lease agreement between us and the landlord. We also amended our original July 2013 guarantee to continue guaranteeing rental payments owed by Genezen until the end of the current term on May 31, 2029. In the event of Genezen’s default related to rental payments owed to the landlord, we are entitled to terminate the assignment agreement and step into the original lease agreement.
Previous report
At the Closing, we entered into a commercial supply agreement (“CSA”) with Genezen. Pursuant to the CSA, Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX® on our behalf. The CSA includes a minimum term of three years and minimum purchase commitments of HEMGENIX® commercial supplies of $43.3 million over the first three years, unless certain contractual provisions are triggered. Our obligations with respect to the supply of HEMGENIX® to CSL Behring remain in effect notwithstanding the subcontracting to Genezen.
Previous report
Organizational restructuring
Previous report
In August 2024, we announced the Restructuring to conserve capital and streamline the organization. As a result of the Restructuring and Lexington Transaction, we eliminated approximately 300 positions or 65% of our workforce.
Previous report
We substantially completed the Restructuring at the end of 2024. We have incurred $5.2 million of employee severance costs in the year ended December 31, 2024 and estimate that we will incur additional costs in the range of $0.5 million to $1.0 million in 2025.
Previous report
Royalty financing agreement
Previous report
In May 2023, we entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. (the “Purchaser”). Under the terms of the Royalty Financing Agreement, we received an upfront payment of $375.0 million in exchange for the Purchaser’s rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us. We will be obligated to pay $25.0 million of the first worldwide sales milestone payment from CSL Behring, if received, to the Purchaser. The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) until June 30, 2032 (“First Hard Cap Date”) if such thresholds are met or, if such cap is not met by June 30, 2032, up to 2.25 times of the upfront payment through December 31, 2038 (“Second Hard Cap Date”). If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to us. We have no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.
Previous report
As of December 31, 2024 we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date. Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date.
Previous report
We retained the rights to all other royalties, as well as contractual milestones totaling up to $1.3 billion, under the terms of the CSL Behring Agreement.
Previous report
Upon entering into the Royalty Financing Agreement in May 2023, we and Hercules amended our existing term loan facility. Through the May 2023 amendment we extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”).
Previous report
In connection with the Closing we once more amended the facility. As a condition to Hercules’ consent to the Lexington Transaction we prepaid $50.0 million of the total $100.0 million principal outstanding in July 2024. The remaining $50.0 million principal outstanding will need to be repaid at the Maturity Date.
Previous report
Financing
Previous report
Intangible asset
Previous report
Part of the consideration received in the Lexington Transaction included a right, with an estimated fair market value of $16.7 million, to purchase HEMGENIX® from Genezen up to the termination of our supply agreement with CSL Behring at terms considered favorable to market terms. The determination of the fair value required us to estimate the current and future commercial supply prices of HEMGENIX® to compare these to the contractually agreed terms at Closing. Prices for the commercial supply of HEMGENIX® are not readily observable in the market. Therefore, our estimate of the commercial supply price was based on what a market participant would be willing to pay in a comparable transaction, using industry benchmarks, relevant market data, current and historical commercial supply agreements, as well as historical production cost information as reference points.
Previous report
If the estimated commercial supply price used in the valuation had increased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received as well as the gain recorded on the divestment would have been increased by $3.0 million. If the estimated commercial supply price used in the valuation had been decreased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received on the divestment would have been decreased by $2.9 million. This would have resulted in a loss on divestment of $1.7 million instead of the $1.2 million gain presented.
Previous report
Date at which we expect to satisfy the commitment under the Royalty Financing Agreement
Previous report
On May 12, 2023, we entered into the Royalty Financing Agreement with the Purchaser. Under the terms of the Royalty Financing Agreement we received an upfront payment of $375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us (refer to Note 13 “Royalty Financing Agreement” for further details). Pursuant to the agreement, the total amount of royalties to be received by the Purchaser is subject to an increasing cap equal to (i) $693.8 million up to the First Hard Cap Date if such thresholds are met or, if such cap is not met by the First Hard Cap Date (ii) $843.8 million through the Second Hard Cap Date. If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to us. We have no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.
Previous report
As of December 31, 2024, we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date. Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date. As a result of this change in estimate we expect that our total payments of interest expense will increase from $318.8 million by $150.0 million to $468.8 million over a term that was extended from 9 years to 15.5 years.
Previous report
Revenue recognition related to CSL Behring variable milestone payments
Previous report
We sold the exclusive global rights HEMGENIX™ to CSL Behring in 2021. The consideration received included variable milestone payments which we recorded as revenue once probable. We recorded $100.0 million of variable milestone revenue related to a first sale of HEMGENIX™ as license revenue in the year ended December 31, 2022 following the November 2022 BLA approval. We collected the payment in July 2023. We do not consider this to be a critical accounting estimate for the years ended December 31, 2024 and 2023 because we currently do not expect to recognize any regulatory milestone payments within the next 12 months.
Previous report
In the year ended December 31, 2024, we adopted Accounting Standards Update (ASU) 2023-07,Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures (“ASU 2023-07”). This update requires us to disclose segment expenses that are significant and regularly provided to our chief operating decision maker (“CODM”). In addition, ASU 2023-07 requires us to disclose the title and position of its CODM and how the CODM uses segment profit or loss information in assessing segment performance and deciding how to allocate resources. The adoption of this update did not have a material impact on our financial position or results of operations but resulted in expanded disclosures in the segment reporting section of the financial statements. We adopted ASU 2023-07 using a retrospective transition method.
Previous report
BMS
Previous report
Following the termination of the collaboration, research and license agreements and the amended agreements with Bristol-Myers Squibb (“BMS”) in February 2023, we did not recognize any further revenue for services rendered. We recognized nil collaboration revenue for the years ended December 31, 2024 and 2023 and $1.8 million for the year ended December 31, 2022.
Previous report
Other non-operating items, net
Previous report
In 2024 and 2023, we recognized no Other non-operating gains. In 2022, we recognized a $2.8 million net gain related to a decrease in the fair value market value of a derivative financial liability
Previous report
Income tax
Previous report
From 2022 up to mid-2023 we recognized deferred tax income in France as a result of the buildup of net operating losses by the French entity. The deferred tax income in France in 2024 is almost fully offset with an increase in valuation allowance.
Showing the first 40 of 43 passages. Open the source sections for the rest.
Reworded paragraphs (55)
Previous wording
Except for the historical information contained herein, the matters discussed in this MD&A may be deemed to be forward-looking statements. Forward-looking statement 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.
Current wording
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.
Previous wording
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, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1, refractory mesial temporal lobe epilepsy (“mTLE”), and Fabry disease.
Current wording
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.
Previous wording
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 amicro ribonucleic acid (“miRNA”) specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
Current wording
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.
Previous wording
We are currently conducting the U.S. and European studies. We completed the enrollment of all 26 patients in the first two cohorts of our US study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023. As of February 2025, we have enrolled 12 patients into a third cohort to further investigate both doses of AMT-130 together with perioperative immunosuppression using the current, established stereotactic administration procedure.
Current wording
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.
Previous wording
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 $10.6 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.
Current wording
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.* *
Previous wording
As of December 31, 2024, we had $367.5 million in cash and cash equivalents and investment securities (December 31, 2023: $617.9 million cash and cash equivalents and investment securities). We had a net loss of $239.6 million in 2024 and a net loss of $308.5 million and $126.8 million in 2023 and 2022, respectively. As of December 31, 2024, we had an accumulated deficit of $1,130.0 million (December 31, 2023: $890.4 million).
Current wording
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).
Previous wording
See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Current wording
Refer to “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Previous wording
We consider the following to be our critical accounting estimates in the year ended December 31, 2024:
Current wording
We consider the following to be our critical accounting estimate in the year ended December 31, 2025:
Previous wording
In July 2021 (“Acquisition Date”), we acquired uniQure France SAS (“Acquisition Date”) and we recorded contingent consideration related to amounts potentially payable to uniQure France SAS’s former shareholders. The amounts payable in accordance with the share and purchase agreement (“SPA”) are contingent upon realization of certain milestones associated with AMT-260. Contingent consideration was measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.
Current wording
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.
Previous wording
Changes in contingent consideration can result from changes in the assumed achievement and timing of estimated milestones and the discount rate used to estimate the fair value of the liability:
Current wording
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:
Previous wording
The fair value of the contingent consideration liability as of December 31, 2024 was EUR 10.5 million ($10.9 million) and as of December 31, 2023 was EUR 39.0 million ($43.0 million). The reduction was primarily driven by a EUR 26.8 million ($28.2 million) milestone payment we made in December 2024 related to the first patient dosed in the AMT-260 clinical trial. If as of December 31, 2024, we 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 33.7 million ($35.0 million). If as of December 31, 2024 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.
Current wording
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.
Previous wording
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 $10.1 million, $2.8 million and $100.0 million of license revenue for the years ended December 31, 2024, 2023 and 2022, respectively. We recognized $10.1 million and $2.8 million of license revenue in 2024 and 2023 related to royalty payments owed on HEMGENIX® sales, when earned. We recognized $100.0 million of license revenue in 2022 related to a milestone payment owed on the first sale of HEMGENIX® in the U.S. in 2023.
Current wording
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.
Previous wording
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) will expire in 2025.
Current wording
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.
Previous wording
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 $1.3 million, $0.1 million and $1.3 million of such cost in the years ended December 31, 2024, 2023 and 2022, respectively.
Current wording
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.
Previous wording
We recognize collaboration revenues associated with services we provide to CSL Behring. Collaboration revenue is recognized when the performance obligations are satisfied. We recognized $10.9 million, $2.3 million and $3.0 million of collaboration revenue for the years ended December 31, 2024, 2023 and 2022, respectively. 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. The decrease in collaboration revenue in 2023 of $0.7 million compared to 2022 was primarily related to a reduction in services requested by CSL Behring following the submissions of the BLA and MAA for HEMGENIX® in 2022.
Current wording
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.
Previous wording
We provided contract manufacturing service to CSL Behring between April 2022 and July 2024. We transferred these activities to Genezen as part of the Lexington Transaction. 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.
Current wording
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.
Previous wording
As a result of 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 contract manufacturing HEMGENIX® for CSL Behring when earned upon sales of HEMGENIX® to CSL Behring. We recognized $6.1 million, $10.8 million and $1.7 million contract manufacturing revenues in the years ended December 31, 2024, 2023 and 2022, respectively.
Current wording
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.
Previous wording
We incurred $17.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the year ended December 31, 2024 and $13.6 million and $2.1 million of cost of contract manufacturing revenues, in the years ended December 31, 2023 and 2022 respectively. 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. Costs of contract manufacturing revenues increased in the year ended December 31, 2023 compared to the year ended December 31, 2022 due to the increase in sales of HEMGENIX® and a write-down of inventory that had a cost basis in excess of its expected net realizable value.
Current wording
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.
Previous wording
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 judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:
Current wording
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:
Previous wording
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 candidate.
Current wording
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.
Previous wording
Research and development expenses for the year ended December 31, 2024 were $143.8 million, compared to $214.9 million and $197.6 million for the years ended December 31, 2023 and 2022, 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.
Current wording
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.
Previous wording
In the years ended December 31, 2024, 2023 and 2022, we incurred $18.0 million, $13.0 million and $19.8 million 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 $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. The decrease of $6.8 million in external cost in 2023 compared to 2022 is a result of the completion of U.S. patient enrollment in March 2022.
Current wording
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.
Previous wording
In the years ended December 31, 2024, 2023 and 2022 we incurred $12.1 million, $13.0 million and $16.2 million expenses respectively. In August 2023, the FDA cleared our IND application, and we started incurring costs for the preparation of a Phase I clinical trial.
Current wording
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.
Previous wording
In the years ended December 31, 2024, 2023 and 2022 we incurred $4.8 million, $2.7 million and $2.9 million expenses, respectively, related to our development of AMT-191. In November 2023, the FDA cleared the IND application, and we started incurring additional costs for Phase I/II clinical trial preparation. Costs incurred in the year ended December 31, 2022 primarily related to our preclinical activities.
Current wording
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.
Previous wording
In January 2023, we entered into a global licensing agreement with Apic Bio for AMT-162. In the years ended December 31, 2024 and 2023, we incurred $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial in which enrolled the first patient in October 2024. 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.
Current wording
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.
Previous wording
In the years ended December 31, 2024, 2023 and 2022 we incurred $3.9 million, $11.0 million and $7.2 million expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Current wording
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.
Previous wording
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 and 2022 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.
Current wording
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.
Previous wording
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio. Our selling costs included employee expenses as well as advisory fees related to obtaining the commercialization and license agreement (“CSL Behring Agreement”) between us and CSL Behring LLC (“CSL Behring”).
Current wording
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.
Previous wording
Selling, general and administrative expenses for the year ended December 31, 2024 were $52.7 million, compared to $74.6 million and $55.1 million for the years ended December 31, 2023 and 2022, respectively.
Current wording
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.
Previous wording
We recognized nil income related to our equity stake in VectorY B.V. in the year ended December 31, 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. We recognized other income of $0.3 million related to the equity stake in 2022.
Current wording
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.
Previous wording
In 2024, we recognized $5.6 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands and France compared to $5.3 million in 2023 and $6.1 million in 2022.
Current wording
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.
Previous wording
In year ended December 31, 2024, we recognized a $1.2 million net gain related to the Lexington Transaction, with no such amounts recognized in 2023 and 2022.
Current wording
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.
Previous wording
We recognized $3.2 million in net other expenses during the year ended December 31, 2024 (nil in 2023 and 2022) 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.
Current wording
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.
Previous wording
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized interest income of $21.4 million in 2024, $19.6 million in 2023 and $0.6 million in 2022. Our interest income increased in 2024 by $1.9 million compared to 2023 primarily due to the interest income earned on investment securities and cash on hand. Interest income increased by $19.0 million in 2023, compared to 2022, as a result of receiving $370.1 million of net proceeds from the May 2023 royalty financing agreement and a $100.0 million milestone payment from CSL Behring in July 2023.
Current wording
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.
Previous wording
We recognized non-cash interest expenses related to the May 2023 royalty financing agreement of $50.9 million in 2024, $26.9 million in 2023 and nil in 2022.
Current wording
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.
Previous wording
We recognized interest expense of $12.9 million in 2024, $14.6 million in 2023 and $11.7 million in 2022 related to the Hercules debt facility. 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. Interest expense increased by $2.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt.
Current wording
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.
Previous wording
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 2024, we recognized a net foreign currency loss of $10.5 million related to our borrowings from Hercules, the May 2023 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 loss of $1.7 million in 2023 and a net gain of $23.2 million in 2022.
Current wording
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.
Previous wording
We recognized $2.3 million of deferred tax expense in 2024 compared to $1.9 million of deferred tax expense in 2023 and $1.5 million of deferred tax income in 2022. In 2024 and 2023 deferred tax expense recorded in the U.S., related to the consumption of net operating losses.
Current wording
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.
Previous wording
As of December 31, 2024, we had cash and cash equivalents, restricted cash and investment securities of $368.9 million. Until such time, if ever, as we can generate substantial cash flows from successfully commercializing our proprietary product candidates, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution, and licensing arrangements. Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe that our cash and cash equivalents and investment securities will fund our operations through the second half of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. The amount and timing of our actual expenditures may vary significantly depending on numerous factors, including the timing of a potential BLA submission, approval and commercialization of AMT-130. We expect that we will require additional funding if we decide to advance any of our other clinical product candidates into late-stage clinical development. Our material cash requirements include the following contractual and other obligations:
Current wording
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.
Previous wording
We entered into lease arrangements for facilities, including corporate, laboratory and office space. As of December 31, 2024, we had fixed lease payment obligations of $23.0 million, with $4.1 million payable within 12 months. Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen. This reduced our fixed lease payment obligations by $20.7 million. We continue to guarantee such payments until the end of the lease term in May 2029.
Current wording
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.
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Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
Our material cash requirements include the following contractual and other obligations:
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
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 $10.6 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.