Item 7. Management’s Discussion and Analysis
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (“MD&A”) is intended to help the reader understand our results of operations and financial condition. This MD&A is provided as a supplement to, and should be read in conjunction with, our audited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Annual Report on Form 10-K, including the disclosures under “Risk Factors.” Our consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S. GAAP”) and unless otherwise indicated are presented in U.S. dollars.
Except for the historical information contained herein, the matters discussed in this MD&A may be deemed to be forward-looking statements. Forward-looking 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.
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, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1, refractory mesial temporal lobe epilepsy (“mTLE”), and Fabry disease.
Huntington’s disease program (AMT-130)
AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a micro ribonucleic acid (“miRNA”) specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
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.
RMAT Designation
In June 2024, we announced that the FDA granted us RMAT designation for AMT-130.
Updated Interim Data
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.
Regulatory Alignment
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.
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Other clinical candidates
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.
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.
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.
Sale of commercial manufacturing activities
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.
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.
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.
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.
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.
Organizational restructuring
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.
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.
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Financing
Royalty financing agreement
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.
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.
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.
Hercules loan repayment and amendment
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”).
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.
Financing
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.
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Financial Highlights
Key components of our results of operations include the following:
Year ended December 31,
2024
2023
2022
(in thousands)
Total revenues
$
27,119
$
15,843
$
106,483
Cost of license revenues
(1,267)
(65)
(1,254)
Cost of contract manufacturing revenues
(17,060)
(13,563)
(2,089)
Research and development expenses
(143,782)
(214,864)
(197,591)
Selling, general and administrative expenses
(52,657)
(74,591)
(55,059)
Net loss
(239,556)
(308,478)
(126,789)
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).
See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Critical Accounting Policies and Estimates
In preparing our consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the SEC we make assumptions, judgments and estimates that can have a significant impact on our net loss/income and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. On an ongoing basis, we evaluate our assumptions, estimates and judgments, including those related to what we believe to be our critical accounting policies. Refer to Note 2 “Summary of significant accounting policies” for a summary of our significant accounting policies.
We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions, judgments or estimates. We also discuss our critical accounting estimates with the Audit Committee of our Board of Directors.
We consider the following to be our critical accounting estimates in the year ended December 31, 2024:
● Contingent consideration recorded in relation to the uniQure France SAS business combination;
● Valuation of the intangible asset with favorable terms recorded as part of the Lexington Transaction;
● Date at which we expect to satisfy the commitment under the Royalty Financing Agreement.
Contingent consideration
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.
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:
● We had used discount rates ranging from 15.3% to 15.6% to calculate the contingent consideration as of December 31, 2023. As of December 31, 2024 we used discount rates ranging from 15.3% to 16.2% to reflect increases in market interest rates. An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability.
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● We need to regularly update our estimate of when a milestone is expected to be achieved. An achievement of a milestone at a later than currently expected date reduces the fair value of the contingent consideration liability whereas an achievement at an earlier than currently expected date increases the fair value of the contingent consideration liability.
● We initially recorded the contingent consideration liability on the Acquisition Date assuming a 40% probability of advancing the TLE research program into clinical development. We developed this estimate using data from an external study regarding the average likelihood of advancing into clinical development at a certain stage or preclinical development. For the year ended December 31, 2022, we had increased the probability to 66.0% following the commencement of toxicology studies for the TLE program. During the year ended December 31, 2023 we increased the probability to 100.0% following the clearance of the IND application for AMT-260 in August 2023. We did not change the probabilities of any further milestones following the dosing of the first patient in a Phase I/II clinical trial in November 2024. The increase in probabilities resulted in a $14.2 million expense in the year ended December 31, 2023 and a $4.4 million expense in the year ended December 31, 2022. This also resulted in an increase of the probability that AMT-260 may advance to late-stage development and commercialization.
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.
Intangible asset
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.
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.
Date at which we expect to satisfy the commitment under the Royalty Financing Agreement
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.
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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.
Revenue recognition related to CSL Behring variable milestone payments
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.
Recently Adopted Accounting Pronouncements
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.
Results of Operations
The following table presents a comparison of the years ended December 31, 2024, 2023 and 2022.
Year ended December 31,
2024
2023
2022
2024 vs 2023
2023 vs 2022
(in thousands)
Total revenues
$
27,119
$
15,843
$
106,483
$
11,276
$
(90,640)
Operating expenses:
Cost of license revenues
(1,267)
(65)
—
(1,202)
(65)
Cost of contract manufacturing
(17,060)
(13,563)
(3,343)
(3,497)
(10,220)
Research and development expenses
(143,782)
(214,864)
(197,591)
71,082
(17,273)
Selling, general and administrative expenses
(52,657)
(74,591)
(55,059)
21,934
(19,532)
Total operating expenses
(214,766)
(303,083)
(255,993)
88,317
(47,090)
Other income
7,926
6,059
7,171
1,867
(1,112)
Other expense
(4,573)
(1,690)
(820)
(2,883)
(870)
Loss from operations
(184,294)
(282,871)
(143,159)
98,577
(139,712)
Non-operating expense, net
(52,833)
(23,686)
14,900
(29,147)
(38,586)
Loss before income tax benefit
$
(237,127)
$
(306,557)
$
(128,259)
69,430
(178,298)
Income tax (expense) / benefit
(2,429)
(1,921)
1,470
(508)
(3,391)
Net loss
$
(239,556)
$
(308,478)
$
(126,789)
$
68,922
$
(181,689)
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Revenues and cost of revenues
Our revenues and associated costs for the years ended December 31, 2024, 2023 and 2022 were as follows:
Year ended December 31,
2024
2023
2022
2024 vs 2023
2023 vs 2022
(in thousands)
License revenues
$
10,133
$
2,758
$
100,000
$
7,375
$
(97,242)
Contract manufacturing revenues
6,114
10,835
1,717
(4,721)
9,118
Collaboration revenues
10,872
2,250
4,766
8,622
(2,516)
Total revenues
$
27,119
$
15,843
$
106,483
$
11,276
$
(90,640)
Cost of license revenues
(1,267)
(65)
(1,254)
(1,202)
1,189
Cost of contract manufacturing revenues
(17,060)
(13,563)
(2,089)
(3,497)
(11,474)
Total cost
$
(18,327)
$
(13,628)
$
(3,343)
$
(4,699)
$
(10,285)
CSL Behring
We sold the exclusive global rights to HEMGENIX® to CSL Behring in 2021 (“License Sale”). We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of HEMGENIX® have been earned. We recognized $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.
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.
With respect to our collaboration with CSL Behring, we have agreed with St. Jude on an apportionment of certain amounts we receive from CSL Behring as sublicensing revenue that is equivalent to a low-single digit percentage of such amounts. The agreement will remain in effect until no further payment is due relating to any licensed product under this agreement or either we or St. Jude exercise our rights to terminate it. St. Jude may terminate the agreement in specified circumstances relating to our insolvency. We may terminate the agreement for convenience at any time subject to a specified notice period.
We treat license payments to St. Jude as contract fulfillment costs associated with license revenue we receive from CSL Behring and recognize these as costs of license revenues. We incurred $1.3 million, $0.1 million and $1.3 million of such cost in the years ended December 31, 2024, 2023 and 2022, respectively.
We recognize collaboration revenues associated with services we provide to CSL Behring. Collaboration revenue is recognized when the performance obligations are satisfied. We recognized $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.
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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.
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.
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.
BMS
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.
Res e arch and development expenses
We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:
● employee-related expenses, including salaries, benefits, travel and share-based compensation expense;
● costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;
● costs incurred to conduct consistency and comparability studies;
● costs incurred for the development and improvement of our manufacturing processes and methods;
● costs associated with research activities for enabling technology platforms;
● costs associated with the rendering of collaboration services;
● payments related to identifiable intangible assets without an alternative future use;
● payments to our licensors for milestones that have been achieved related to our product candidates;
● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and
● changes in the fair value of liabilities recorded in relation to our acquisition of uniQure France SAS.
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Our research and development expenses primarily consist of costs incurred for the research and development of our product candidates, which include:
● AMT-130 (Huntington’s disease). We have been incurring costs related to our U.S. study since February 2019. Since 2021, we have also incurred costs related to our European study;
● AMT-260 (Temporal lobe epilepsy) . We have incurred costs related to the development of AMT-260. We acquired this program in July 2021;
● AMT-191 (Fabry disease) . We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease;
● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ) We have incurred costs related to the acquisition in addition to costs incurred to initiate and conduct a Phase I/II clinical trial;
● AMT-061 (Hemophilia B) . 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 periods ended December 31, 2023 and 2024 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.
● Preclinical research programs . We incurred costs related to the research of multiple preclinical gene therapy product candidates with the potential to treat certain rare and other serious medical conditions; and
● Technology platform development and other related research . We incurred significant research and development costs related to manufacturing prior to the Closing of the Lexington Transaction and other enabling technologies that are applicable across all our programs.
Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgement 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 candidate.
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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.
Year ended December 31,
2024
2023
2022
2024 vs 2023
2023 vs 2022
(in thousands)
Huntington's disease (AMT-130)
$
17,989
$
12,991
$
19,846
$
4,998
$
(6,855)
Temporal lobe epilepsy (AMT-260)
12,053
13,037
16,199
(984)
(3,162)
Amyotrophic lateral sclerosis (AMT-162)
7,306
16,039
—
(8,733)
16,039
Fabry disease (AMT-191)
4,814
2,659
2,862
2,155
(203)
Programs in preclinical development and platform related expenses
3,927
11,005
7,157
(7,078)
3,848
Hemophilia B (AMT-061)
—
(1,336)
2,474
1,336
(3,810)
Total direct research and development expenses
$
46,089
$
54,395
$
48,538
$
(8,306)
$
5,857
Employee and contractor-related expenses
49,818
74,514
64,935
(24,696)
9,579
Facility expenses
21,627
29,490
23,582
(7,863)
5,908
Share-based compensation expense
9,295
16,881
18,402
(7,586)
(1,521)
Disposables
5,084
13,232
17,830
(8,148)
(4,598)
Severance costs
3,967
2,188
—
1,779
2,188
Impairment charge
—
1,438
—
(1,438)
1,438
Fair value changes related to contingent consideration
(1,817)
15,895
7,081
(17,712)
8,814
Other expenses
9,719
6,831
17,223
2,888
(10,392)
Total other research and development expenses
$
97,693
$
160,469
$
149,053
$
(62,776)
$
11,416
Total research and development expenses
$
143,782
$
214,864
$
197,591
$
(71,082)
$
17,273
Direct research and development expenses
Huntington disease (AMT-130)
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.
Temporal lobe epilepsy (AMT-260)
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.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In January 2023, we entered into a global licensing agreement with Apic Bio for AMT-162. In the years ended December 31, 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.
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Fabry disease (AMT-191)
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.
Preclinical programs & platform development
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.
Hemophilia B (AMT-061)
We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022. Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 and 2022 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.
Other research & development expenses
● We incurred $49.8 million in employee and contractor expenses in the year ended December 31, 2024 compared to $74.6 million in 2023 and $64.9 million in 2022. Our cost decreased in 2024 by $24.7 million compared to 2023 primarily as a result of the July 2024 Lexington Transaction as well as the October 2023 and August 2024 reorganizations. The increase in 2023 of $9.6 million, compared to 2022, primarily related to the hiring of new personnel and contractors in 2022 through mid-2023 to support our clinical-stage programs and manufacturing operations;
● We incurred $21.6 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the year ended December 31, 2024 compared to $29.4 million in 2023 and $23.6 million in 2022. The decrease in 2024 compared to 2023 of $7.9 million is primarily a result of the July 2024 Lexington Transaction. Our costs increased by $5.9 million in 2023 compared to 2022 as a result of incurring additional operating and depreciation expenses in both our Lexington and Amsterdam facilities;
● We incurred $9.3 million in share-based compensation expenses in the year ended December 31, 2024 compared to $16.9 million in 2023 and $18.4 million in 2022. The decrease in 2024 of $7.6 million, compared to 2023, is primarily a result of the July 2024 Lexington Transaction, the reorganizations in October 2023 and August 2024 and a decrease in the fair value of the long-term incentive awards granted. The decrease in 2023 of $1.5 million, compared to 2022, is primarily due to the October 2023 reorganization and a decrease in the fair value of long-term incentive awards granted. This was partially offset by cost related to performance share units granted in 2021, for which achievement of related performance conditions was deemed probable in 2023 and 2024;
● We incurred $5.1 million in disposables costs in the year ended December 31, 2024 compared to $13.2 million in 2023 and $17.8 million in 2022. The decrease in 2024 primarily related to the July 2024 Lexington Transaction as well as the reorganizations in October 2023 and August 2024. The decrease in 2023 compared to 2022 was related to the October 2023 reorganization;
● We incurred $4.0 million of severance costs in the year ended December 31, 2024 related to the July 2024 Lexington Transaction and the August 2024 reorganization, compared to $2.1 million of severance cost in 2023 related to the October 2023 reorganization. No such costs were incurred in 2022;
● We incurred nil and $1.4 million of costs in the years ended December 31, 2024 and 2023, respectively, related to the impairment of the Lexington, MA facility right-of-use asset and related leasehold improvements, compared to nil in 2022;
● We recognized a $1.8 million gain in the year ended December 31, 2024 related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to losses of $15.9 million in 2023 and $7.1 million in 2022. The increase in the 2023 loss was primarily due to an increase in the probability of making future milestone payments due upon dosing of the first patient in Phase I/II clinical trial of AMT-260 after receiving IND acceptance in August 2023; and
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● We incurred $9.7 million in other expenses in the year ended December 31, 2024 compared to $6.8 million in 2023 and $17.2 million in 2022. The increase in costs in 2024 of $2.9 million, compared to 2023, is primarily a result of an increase in information technology expenses partially offset by decreases in contractual license expenses and consultant-related expenses. The decrease in costs in 2023 of $10.4 million, compared to 2022, is due to decreases in contractual license expenses and consultant-related expenses.
Selling, general and administrative expenses
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio. 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”).
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.
Year ended December 31,
2024
2023
2022
2024 vs 2023
2023 vs 2022
(in thousands)
Employee and contractor-related expenses
$
21,343
$
24,399
$
21,065
$
(3,056)
$
3,334
Share-based compensation expense
11,928
17,386
15,479
(5,458)
1,907
Professional fees
7,261
11,673
7,133
(4,412)
4,540
Depreciation and facility costs
2,110
2,051
1,181
59
870
Intellectual property fees
1,744
3,819
1,480
(2,075)
2,339
Severance costs
1,223
361
—
862
361
Information technology costs
1,101
6,526
3,190
(5,425)
3,336
Financial advisory fees
—
3,800
1,000
(3,800)
2,800
Other expenses
5,947
4,576
4,531
1,371
45
Total selling, general and administrative expenses
$
52,657
$
74,591
$
55,059
$
(21,934)
$
19,532
● We incurred $21.3 million in personnel and contractor expenses in the year ended December 31, 2024 compared to $24.4 million in 2023 and $21.4 million in 2022. The decrease in 2024 of $3.1 million, compared to 2023, was primarily related to the reduction in personnel and contractors as a result of the Lexington Transaction and the October 2023 and August 2024 reorganizations. The increase in 2023 of $3.3 million, compared to 2022, was primarily related to the hiring of personnel and contractors to support our business operations;
● We incurred $11.9 million of share-based compensation expenses in the year ended December 31, 2024 compared to $17.4 million in 2023 and $15.5 million in 2022. The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the Lexington Transaction, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of long-term incentive awards granted, partially offset by an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2024. The increase in 2023 of $1.9 million, compared to 2022, was primarily related to an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2023. These increases were partially offset by a decrease in expense related to a reduction in the fair value of long-term incentive awards granted;
● We incurred $7.3 million in professional fees in the year ended December 31, 2024 compared to $11.7 million in 2023 and $7.1 million in 2022. We regularly incur accounting, audit and legal fees associated with operating as a public company. In 2024, we incurred additional professional fees related to the Lexington Transaction. In 2023, we incurred additional professional fees related to our global licensing agreement with Apic Bio, the Royalty Purchase Agreement and other corporate initiatives;
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● We incurred $1.7 million in intellectual property fees including registration and professional fees in the year ended December 31, 2024 compared to $3.8 million in 2023 and $1.5 million in 2022. The decrease of $2.1 million in 2024, compared to 2023, is primarily due to a decrease in professional fees. The increase of $2.3 million in 2023, compared to 2022, is mainly related to an increase in professional fees;
● We incurred $1.2 million of severance costs in the year ended December 31, 2024 related to the Lexington Transaction and the August 2024 reorganization and $0.4 million of severance costs in 2023 related to the October 2023 reorganization. No such costs were incurred in 2022; and
● We incurred nil, $3.8 million and $1.0 million in financial advisory fees pertaining to our licensing transaction with CSL Behring in the years ended December 31, 2024, 2023 and 2022, respectively.
Other items, net
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.
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.
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.
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.
Other income for the years ended December 31, 2024, 2023, and 2022 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility. We present expenses related to such income as other expense.
Other non-operating items, net
Our non-operating items, net, for the years ended December 31, 2024, 2023 and 2022 were as follows:
Year ended December 31,
2024
2023
2022
2024 vs 2023
2023 vs 2022
(in thousands)
Interest income
$
21,415
$
19,562
$
609
$
1,853
$
18,953
Interest expense - Royalty Financing Agreement
(50,865)
(26,933)
—
(23,932)
(26,933)
Interest expense - Hercules debt facility
(12,876)
(14,624)
(11,704)
1,748
(2,920)
Foreign currency (losses) / gains, net
(10,507)
(1,691)
23,235
(8,816)
(24,926)
Other non-operating gains
—
—
2,760
—
(2,760)
Total non-operating (expense) / income, net
$
(52,833)
$
(23,686)
$
14,900
$
(29,147)
$
(38,586)
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.
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.
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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.
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.
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
Income tax
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.
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.
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Financial Position, Liquidity and Capital Resources
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:
Debt
As of December 31, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc. (“Hercules”) for an aggregate principal amount of $50.0 million. In July 2024, in connection with the Closing of the Lexington Transaction, we repaid $50.0 million of the $100.0 million of principal outstanding as well as $3.1 million in end-of-term fees. Future interest payments and financing fees associated with the loan total $16.0 million, with $8.6 million payable within 12 months. We are contractually required to repay the $50.0 million principal outstanding in full in January 2027.
Leases
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.
Commitments related to acquisition of uniQure France SAS (nominal amounts)
In relation to our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractual milestones. The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. In September 2023, we made a payment of EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS following the FDA’s clearance of the IND application for AMT-260. In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS following the dosing of the first patient in Phase I/II clinical trial for AMT-260. As of December 31, 2024, our remaining commitment amounts include a EUR 160.0 million ($166.2 million) in potential milestone payments associated with Phase III development and the approvals 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 milestone will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of December 31, 2024, of $1.04/€1.00. As of December 31, 2024, we expect these obligations will become payable between 2029 and 2033. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.
Commitments related to licensors and financial advisors
We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our License Sale to CSL Behring. We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.
Commitments related to the CSA and DMSA with Genezen
Our CSA with Genezen requires us to take or pay contract manufacturing services for HEMGENIX® during a three-year period ending July 2027. As of December 31, 2024, our remaining minimum purchase commitments to Genezen amount to $36.5 million, with $15.8 million to be paid within the next 12 months. CSL Behring’s minimum purchase commitments to us over the next 12 months are $13.6 million.
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Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 2027. As of December 31, 2024 our remaining minimum purchase commitments amount to $12.5 million with $4.0 million to be paid within the next 12 months.
The table below summarizes our consolidated cash flow data for the years ended December 31:
Year ended December 31,
2024
2023
2022
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period
$
244,544
$
231,173
$
559,353
Net cash used in operating activities
(182,728)
(145,929)
(145,060)
Net cash generated from / (used in) investing activities
162,968
(205,686)
(182,734)
Net cash (used in) / generated from financing activities
(59,486)
362,721
1,445
Foreign exchange impact
(4,969)
2,265
(1,831)
Cash, cash equivalents and restricted cash at the end of period
$
160,329
$
244,544
$
231,173
We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V. in 1998, with the exception of 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 $239.6 million for the year ended December 31, 2024, and net loss of $308.5 million in 2023, and a net loss of $126.8 million in 2022. As of December 31, 2024, we had an accumulated deficit of $1,130.0 million.
Sources of liquidity
From our first institutional venture capital financing in 2006 through the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023. Between July 2021 and July 2023, we have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL and other milestones collected from CSL, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX®.
In 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.
On May 12, 2023 we and Hercules amended our debt facility. The amendment extends the Maturity Date and interest-only period from December 2025 to January 2027. In connection with the Closing of the Lexington Transaction in July 2024 we amended the debt facility again and repaid $50.0 million of the principal balance outstanding. We are required to repay the remaining principal balance of $50.0 million on the Maturity Date. The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum. We owe a back-end fee of $2.4 million on December 1, 2025 and a back-end fee of $0.6 million on the Maturity Date.
We are subject to certain covenants under the debt facility 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 2024 Amended Facility may limit our ability to obtain debt financing. The debt facility permits us to issue up to $500.0 million of convertible debt.
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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
Year ended December 31,
2024
2023
2022
(in thousands)
Cash flows from operating activities
Net loss
$
(239,556)
$
(308,478)
$
(126,789)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation, amortization and impairment
12,641
11,900
8,537
Amortization of discount on investment securities
(10,901)
(10,917)
—
Share-based compensation expense
22,258
35,093
34,204
Royalty financing agreement interest expense, net of interest paid
44,203
25,616
—
Deferred tax expense / (income)
2,429
1,921
(1,470)
Change in fair value of contingent consideration and derivative financial instrument, net
(1,817)
15,895
4,320
Unrealized foreign exchange losses / (gains), net
15,415
(2,206)
(22,083)
Other items, net
(3,848)
4,721
1,605
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables
(2,243)
(1,323)
(4,083)
Contract asset related to CSL Behring milestone payments
-
100,000
(45,000)
Inventories
2,421
(6,740)
(6,924)
Accounts payable
1,520
(4,169)
9,238
Accrued expenses, other liabilities, and operating leases
(5,642)
(5,328)
3,385
Contingent consideration milestone payment
(19,608)
(1,914)
—
Net cash used in operating activities
$
(182,728)
$
(145,929)
$
(145,060)
Net cash used in operating activities was $182.7 million for the year ended December 31, 2024, and consisted of a net loss of $239.6 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $12.6 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $22.3 million, $44.2 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $2.4 million, $1.8 million change in the fair value of contingent consideration and unrealized foreign exchange losses of $15.4 million. Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.2 million. There was an decrease in inventory balances of $2.4 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to a increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals. Net cash used in operating activities also includes a payment for a contingent consideration milestone of $19.6 million.
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Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million. Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $80.5 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $1.3 million. There was a net decrease in contract assets of $100.0 million related to the collection of the $100.0 million milestone due from CSL Behring in July 2023 . There was an increase in inventory balances of $6.7 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $9.5 million, primarily related to a decrease of $4.2 million in accounts payable and a decrease of $5.3 million related to various accruals. Net cash used in operating activities also includes a payment for a contingent consideration milestone of $1.9 million.
Net cash used in operating activities was $145.1 million for the year ended December 31, 2022, and consisted of a net loss of $126.8 million adjusted for non-cash items, including depreciation and amortization expense of $8.5 million, share-based compensation expense of $34.2 million, changes in the fair value of contingent consideration and the derivative financial liability of $4.3 million, unrealized foreign exchange gains of $22.1 million and a change in deferred taxes of $1.5 million. Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $43.4 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $4.1 million. There was a net increase in contract assets related to CSL Behring milestone payments of $45.0 million. The net increase related to $100.0 million recognized as a contract asset in the current period and collection of $55.0 million of the contract asset related to the CSL milestones of $55.0 million in March 2022 and April 2022. There was an increase in inventories of $6.9 million related to the production of HEMGENIX ® under the CSL Behring Agreement. These changes also relate to a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $12.6 million, primarily related to an increase in accounts payable.
Net cash generated from / (used in) investing activities
In 2024, we generated $163.0 million from our investing activities compared to using $205.7 million in 2023 and $182.7 million in 2022.
Year ended December 31,
2024
2023
2022
(in thousands)
Proceeds from maturity of debt securities
$
534,498
$
167,907
—
Investment in debt securities
(359,841)
(366,439)
$
(163,146)
Divestment of commercial manufacturing facility
(8,321)
—
—
Capital expenditures - European sites
(1,783)
(3,389)
(11,904)
Capital expenditures - Lexington site
(1,585)
(3,765)
(5,784)
Acquisition of uniQure France SAS, net of cash acquired
—
—
(1,900)
Net cash generated from / (used in) investing activities
$
162,968
$
(205,686)
$
(182,734)
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Net cash (used in) / generated from financing activities
Year ended December 31,
2024
2023
2022
(in thousands)
Cash flows from financing activities
Proceeds from Royalty Financing Agreement, net of debt issuance costs
$
-
$
370,062
$
-
Proceeds from issuance of shares related to employee stock option and purchase plans
2,123
308
1,445
Repayment of long-term debt
(53,050)
-
-
Contingent consideration milestone payment
(8,559)
(7,649)
-
Net cash (used in) / generated from financing activities
$
(59,486)
$
362,721
$
1,445
In December 2024, following the dosing of the first patient in Phase I/II clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS based on contractually defined milestones. EUR 26.8 million ($28.2 million) of this payment related to a contingent consideration of which EUR 8.2 million ($8.6 million) was classified as cash flows from financing activities and EUR 18.7 million ($19.6 million) was classified as a net cash flow used in operating activities.
In September 2023, following the FDA’s clearance of the IND application for AMT-260, we made a payment of EUR 10.0 million ($10.7 million) to the former shareholders of uniQure France SAS based on contractually defined milestones. EUR 8.9 million ($9.6 million) of this payment related to a contingent consideration of which EUR 7.2 million ($7.6 million) was classified as cash flows from financing activities and EUR 1.8 million ($1.9 million) was classified as a net cash flow used in operating activities.
Funding requirements
Our future capital requirements will depend on many factors, including but not limited to:
● activities related to submission of a BLA for AMT-130 in Huntington’s disease;
● activities to prepare for the commercialization of AMT-130 in Huntington’s disease in the United States;
● investments to launch AMT-130 in Huntington’s disease in the United States including the amounts of revenue we generate following the launch;
● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones;
● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;
● the scope, timing, results, and costs of our current and planned clinical trials;
● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements;
● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;
● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;
● the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates;
● the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies;
● the cost, timing and outcome of regulatory reviews associated with our product candidates;
● our ability to enter into collaboration arrangements in the future; and
● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims.
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