Item 2. Management’s Discussion and Analysis
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”). Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S. GAAP”) and unless otherwise indicated are presented in U.S. dollars.
Overview
We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”), refractory mesial temporal lobe epilepsy (“mTLE”) and Fabry disease.
Business Developments
Financing
In January 2025, we received net proceeds of $70.1 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a public offering price of $17.00 per ordinary share. In February 2025, we received an additional $10.6 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.
Huntington’s disease program (AMT-130)
RMAT and Breakthrough Therapy Designations
On April 17, 2025, we announced that the U.S. Food and Drug Administration (“FDA”) granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease. The Breakthrough Therapy designation is supported by clinical data from the ongoing Phase I/II trials of AMT-130 for the treatment of Huntington’s disease showing the potential for meaningful slowing of disease progression. Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). In general, the preliminary clinical evidence should show a clear advantage over available therapy.
In June 2024, we announced that the FDA granted us Regenerative Medicine Advanced Therapy (“RMAT”) designation for AMT-130 based on the potential of AMT-130 to address the major unmet medical need among patients with Huntington’s disease.
In addition to the RMAT and Breakthrough Therapy designations, the FDA previously granted Orphan Drug designation and Fast Track designation to AMT-130.
Regulatory Alignment
In March 2025, we held a Type B meeting with the FDA to discuss chemistry, manufacturing and controls (“CMC”) requirements for AMT-130, in support of a planned Biologics License Application, (“BLA”) submission. An additional Type B meeting focused on the pivotal statistical analysis plan was held in the second quarter of 2025.
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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. In correspondence leading up to and following the Type B meeting, FDA agreed that data from the ongoing Phase I/II studies of AMT-130, compared to a natural history external control, may serve as the primary basis for a BLA, submission under FDA’s accelerated approval pathway. The FDA also agreed that composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain (“NfL”) measured in cerebrospinal fluid (“CSF”) may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.
Recent Developments of other Product Candidates
Temporal lobe epilepsy program (AMT-260)
We are conducting a Phase I/IIa clinical trial (GenTLE) AMT-260 for the treatment of MTLE in the United States. GenTLE consists of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory mTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept data. The FDA-approved study protocol provides that the first three patients to be enrolled in the study are required to have MRI-confirmed unilateral, hippocampal sclerosis.
In November 2024 we announced that the first patient had been dosed in GenTLE. In addition, the FDA in February 2025 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.
Fabry disease program (AMT-191)
We are conducting a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease. The multicenter, open-label clinical trial consists of two dose-escalating cohorts of up to six adult male patients each to assess safety, tolerability, and early signs of efficacy of AMT-191 in patients with Fabry disease.
In February 2025, we announced a favorable recommendation from the independent data monitoring committee (“IDMC”) following a review of safety data from the first two patients treated in the Phase I/IIa study. The IDMC’s review did not identify any significant safety concerns and recommended proceeding with enrollment in the second cohort.
In August 2024, we announced that the first patient has been dosed. In September 2024, we announced the FDA granted Orphan Drug Designation to AMT-191 and in October 2024, the FDA granted Fast Track Designation to AMT-191.
Amyotrophic Lateral Sclerosis (AMT-162)
EPISOD1 is a Phase I/II multi-center, open-label trial of AMT-162 for the treatment of SOD1-ALS being conducted in the United States consisting of three dose-escalating cohorts with up to four patients each receiving a short course of immunosuppression prior to and after an intrathecal infusion of AMT-162. The trial will explore the safety and tolerability of AMT-162 and will assess exploratory signs of efficacy by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein.
In October 2024, we announced the first patient had been dosed in EPISOD1. In January 2025, we announced that the IDMC had recommended proceeding with enrollment of the second cohort after a review of the 28-day safety data from the first cohort. We completed enrollment in the second dose cohort and we expect to initiate enrollment in the third dose cohort in the third quarter of 2025.
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Financial Overview
Key components of our results of operations include the following:
Three months ended March 31,
2025
2024
(in thousands)
Total revenues
$
1,567
$
8,485
Cost of license revenues
(197)
(150)
Cost of contract manufacturing revenues
—
(9,076)
Research and development expenses
(36,140)
(40,692)
Selling, general and administrative expenses
(10,908)
(13,937)
Net loss
(43,637)
(65,618)
As of March 31, 2025 and December 31, 2024, we had cash and cash equivalents and investment securities of $409.0 million and $367.5 million, respectively. We had a net loss of $43.6 million in the three months ended March 31, 2025, compared to a net loss of $65.6 million for the same period in 2024. As of March 31, 2025 and December 31, 2024, we had accumulated deficits of $1,173.6 million and $1,130.0 million, respectively.
See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Critical Accounting Policies and Estimates
In preparing our consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the “SEC”) we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. A summary of our critical accounting policies as well as a discussion of our critical accounting estimates are presented in our Annual Report . There were no material changes to our critical accounting policies during the three months ended March 31, 2025.
Contract manufacturing
We entered into a development and commercial supply agreement with CSL Behring LLC (“CSL Behring”) in June 2020 for the commercial supply of HEMGENIX®. In July 2024, we divested our commercial manufacturing activities located in Lexington, MA (“Lexington Transaction”). Prior to the Lexington Transaction, we recognized contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring and the cost to manufacture HEMGENIX® under such an agreement as cost of contract manufacturing.
Research and development expenses
We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:
● employee-related expenses, including salaries, benefits, travel and share-based compensation expense;
● costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;
● costs incurred to conduct consistency and comparability studies;
● costs incurred for the development and improvement of our manufacturing processes and methods;
● costs associated with research activities for enabling technology platforms;
● costs associated with the rendering of collaboration services;
● payments related to identifiable intangible assets without an alternative future use;
● payments to our licensors for milestones that have been achieved related to our product candidates;
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● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and
● changes in the fair value of liabilities recorded in relation to our acquisition of uniQure France SAS.
Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable 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.
Selling, general and administrative expenses
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.
Other items, net
Our other income generally consists of payments received to subsidize our research and development efforts, income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility, and for the three months ended March 31, 2025, income from a one-time sale of critical reagents.
Following the Lexington Transaction, our other expense primarily consists of costs incurred under our commercial supply agreement (“CSA”) with Genezen Holdings Inc. and its affiliate Genezen MA, Inc. (together “Genezen”). These include costs related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA . Additionally, other expenses also consist of expenses we incur in relation to our subleasing income.
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Results of Operations
Comparison of the three months ended March 31, 2025 and 2024
The following table presents a comparison of our results of operations for the three months ended March 31, 2025 and 2024:
Three months ended March 31,
2025
2024
2025 vs 2024
(in thousands)
Total revenues
$
1,567
$
8,485
$
(6,918)
Operating expenses:
Cost of license revenues
(197)
(150)
(47)
Cost of contract manufacturing
—
(9,076)
9,076
Research and development expenses
(36,140)
(40,692)
4,552
Selling, general and administrative expenses
(10,908)
(13,937)
3,029
Total operating expenses
(47,245)
(63,855)
16,610
Other income
8,306
1,376
6,930
Other expense
(1,959)
(234)
(1,725)
Loss from operations
(39,331)
(54,228)
14,897
Non-operating expense, net
(3,810)
(10,734)
6,924
Loss before income tax benefit
$
(43,141)
$
(64,962)
21,821
Income tax expense
(496)
(656)
160
Net loss
$
(43,637)
$
(65,618)
$
21,981
Revenue
Our revenue for the three months ended March 31, 2025 and 2024 was as follows:
Three months ended March 31,
2025
2024
2025 vs 2024
(in thousands)
License revenues
$
1,567
$
1,202
$
365
Contract manufacturing revenues
—
3,990
(3,990)
Collaboration revenues
—
3,293
(3,293)
Total revenues
$
1,567
$
8,485
$
(6,918)
License revenues
We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended March 31, 2025 and March 31, 2024, we recognized $1.6 million and $1.2 million of license revenues, respectively.
Collaboration revenues
We provide services to CSL Behring in accordance with the CSL Behring Agreement. Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied. For the three months ended March 31, 2025 and 2024 we recognized nil and $3.3 million of collaboration revenue for CSL Behring, respectively. The decrease in collaboration revenue of $3.3 million in the three months ended March 31, 2025 compared to the same period in 2024 resulted from us not providing any development and other services to CSL Behring in relation to the CSL Behring Agreement in 2025.
Contract manufacturing revenues
We recognized contract manufacturing revenues related to the manufacturing of HEMGENIX® for CSL Behring prior to the Lexington Transaction. Contract manufacturing revenues were realized when earned upon sales of HEMGENIX® drug product to CSL Behring. We recognized nil contract manufacturing revenues in the three months ended March 31, 2025 , compared to $4.0 million for the same period in 2024.
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Cost of contract manufacturing
We incurred nil costs of contract manufacturing in the three months ended March 31, 2025. In the three months ended March 31, 2024 we incurred $9.1 million costs of contract manufacturing related to the manufacture of HEMGENIX® prior to the Lexington Transaction in July 2024.
R&D expense
R&D expenses for the three months ended March 31, 2025 were $36.1 million, compared to $40.7 million for the same period in 2024. Other research and development expenses are separately classified in the table below. These other expenses are not allocated as they are deployed across multiple projects under development.
Three months ended March 31,
2025
2024
2025 vs 2024
(in thousands)
Huntington's disease (AMT-130)
$
8,230
$
2,426
$
5,804
Fabry disease (AMT-191)
2,187
1,370
817
Temporal lobe epilepsy (AMT-260)
2,075
2,254
(179)
Amyotrophic lateral sclerosis (AMT-162)
1,831
1,977
(146)
Programs in preclinical development and platform related expenses
1,280
449
831
Total direct research and development expenses
$
15,603
$
8,476
$
7,127
Employee and contractor-related expenses
10,159
16,923
(6,764)
Facility expenses
4,550
7,095
(2,545)
Share-based compensation expense
2,463
3,425
(962)
Fair value changes related to contingent consideration
1,216
165
1,051
Disposables
827
1,921
(1,094)
Other expenses
1,322
2,687
(1,365)
Total other research and development expenses
$
20,537
$
32,216
$
(11,679)
Total research and development expenses
$
36,140
$
40,692
$
(4,552)
Direct research and development expenses
General
Prior to the Lexington Transaction, we supplied Good Manufacturing Processes (“GMP”) material utilized in our clinical programs for Huntington’s disease and Fabry disease as well as GMP material needed for our preclinical programs using our in-house manufacturing facility. The related expenses were part of “other research and development expenses”. Following the closing of the Lexington Transaction we rely on third party suppliers and present associated expenses for these activities within “direct research and development expenses”.
Huntington’s disease (AMT-130)
In three months ended March 31, 2025 and March 31, 2024, we incurred costs of $8.2 million and $2.4 million 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 $5.8 million in external cost in 2025 compared to 2024 was primarily driven by activities related to the preparation of the BLA submission for AMT-130 in the United States.
Fabry disease (AMT-191)
In the three months ended March 31, 2025 and March 31, 2024, we incurred costs of $2.2 million and $1.4 million, respectively, related to our development of AMT-19. We incurred $2.1 million and $0.1 million related to clinical trials and CMC development, respectively, compared to $1.3 million and $0.0 million in the comparative period. In November 2023, the FDA cleared the IND application, and we started incurring additional costs for the Phase I/II clinical trial preparation.
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Temporal lobe epilepsy (AMT-260)
In three months ended March 31, 2025 and March 31, 2024, we incurred costs of $2.1 million and $2.3 million respectively, for the development of AMT-260. We incurred $1.6 million and $0.4 million related to clinical trials and CMC development, respectively, compared to $0.5 million and $1.7 million in the comparative period.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the three months ended March 31, 2025 and March 31, 2024, we incurred $1.8 million and $2.0 million of expenses, respectively, to initiate a Phase I/II clinical trial which enrolled the first patient in October 2024. We incurred $1.7 million and $0.1 million related to clinical trials and CMC development, respectively, compared to $0.9 million and $1.1 million in the comparative period.
Preclinical programs & platform development
In the three months ended March 31, 2025 and March 31, 2024 , we incurred $1.3 million and $0.4 million of costs, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Other research & development expenses
● We incurred $10.2 million in personnel and contractor-related expenses in the three months ended March 31, 2025 , compared to $16.9 million for the same period in 2024. The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024 as well as a reduction in expense from the restructuring that occurred in August 2024;
● We incurred $4.6 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the three months ended March 31, 2025 , compared to $7.1 million in the same period in 2024. The decrease was primarily related to the divestment of our commercial manufacturing activities in July 2024;
● We incurred $2.5 million in share-based compensation expenses in the three months ended March 31, 2025 , compared to $3.4 million for the same period in 2024. The decrease was primarily related to the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;
● We recognized a $1.2 million loss in the three months ended March 31, 2025, related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $0.2 million loss for the same period in 2024;
● We incurred $0.8 million in disposable costs in the three months ended March 31, 2025 , compared to $1.9 million for the same period in 2024; and
● We incurred $1.3 million of other expenses for the three months ended March 31, 2025 , compared to $2.7 million for the same period in 2024.
Selling, general and administrative expenses
Selling, general and administrative expenses for the three months ended March 31, 2025 were $10.9 million, compared to $13.9 million for the same period in 2024.
Three months ended March 31,
2025
2024
2025 vs 2024
(in thousands)
Employee and contractor-related expenses
$
5,485
$
6,219
$
(734)
Share-based compensation expense
1,947
3,421
(1,474)
Professional fees
1,903
2,041
(138)
Depreciation and facility costs
380
442
(62)
Intellectual property fees
283
588
(305)
Information technology costs
281
267
14
Other expenses
629
959
(330)
Total selling, general and administrative expenses
$
10,908
$
13,937
$
(3,029)
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● We incurred $5.5 million in personnel and contractor-related expenses in the three months ended March 31, 2025, compared to $6.2 million in the same period in 2024. The decrease was primarily related to the reduction in personnel and contractors as a result of the divestment of our commercial manufacturing activities and the organizational restructuring in August 2024;
● We incurred $1.9 million in share-based compensation expenses in the three months ended March 31, 2025 , compared to $3.4 million in the same period in 2024. The decrease was primarily related to the divestment of our commercial manufacturing activities and a reduction in expense from the restructuring that occurred in August 2024;
● We incurred $1.9 million in professional fees in the three months ended March 31, 2025 , compared to $2.0 million in the same period in 2024. We regularly incur accounting, audit and legal fees associated with operating as a public company;
● We incurred $0.3 million in intellectual property fees including registration and professional fees in the three months ended March 31, 2025 compared to $0.6 million in the same period in 2024. The decrease mainly related to a decrease in professional fees; and
● We incurred $0.6 million in other expenses in the three months ended March 31 , 2025, compared to $1.0 million in the same period in 2024.
Other items, net
Our other items, net, for the three months ended March 31, 2025 and March 31, 2024 were as follows:
Three months ended March 31,
2025
2024
2025 vs 2024
(in thousands)
Sale of critical reagents to Genezen
$
6,000
$
—
$
6,000
Research and development grants from Dutch authorities
1,719
1,224
495
Sublease income
458
152
306
Supply of HEMGENIX® to CSL Behring
(1,525)
—
(1,525)
Sublease expense
(434)
(234)
(200)
Other income, net
129
—
129
Total other income, net
$
6,347
$
1,142
$
5,205
In the three months ended March 31, 2025 we recognized $6.0 million in income related to a one-time sale of critical reagents to Genezen (nil in the prior period).
We recognized $1.5 million in net other expenses during the three months ended March 31, 2025, 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.
Other non-operating items, net
Our other non-operating items, net, for the three months ended March 31, 2025 and March 31, 2024 were as follows:
Three months ended March 31,
2025
2024
2025 vs 2024
(in thousands)
Interest income
$
4,127
$
6,508
$
(2,381)
Interest expense - Royalty Financing Agreement
(13,309)
(12,401)
(908)
Interest expense - Hercules debt facility
(1,800)
(3,696)
1,896
Foreign currency gains / (losses), net
7,172
(1,145)
8,317
Total non-operating expense, net
$
(3,810)
$
(10,734)
$
6,924
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $4.1 million in interest income in the three months ended March 31, 2025, compared to $6.5 million in the same period in the prior year. Our interest income decreased by $2.4 million due to the interest income earned on investment securities as well as cash on hand during the three months ended March 31, 2025.
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We recognized interest expense of $1.8 million and $3.7 million in the three months ended March 31, 2025 , and March, 31, 2024, respectively, related to the Hercules Capital, Inc. (“Hercules”) debt facility. Interest expense decreased by $1.9 million in 2025, compared to 2024, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates.
We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S. dollars. We recognize foreign exchange results related to changes in these foreign currencies.
We recognized a net foreign currency gain, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $7.2 million during the three months ended March 31, 2025, compared to a net loss of $1.1 million during the same period in 2024 .
Income tax expense
We recognized $0.5 million of deferred tax expense in the three months ended March 31, 2025 , and $0.7 million of deferred tax expense for the same period in 2024 .
Financial Position, Liquidity and Capital Resources
As of March 31, 2025, we had cash and cash equivalents, restricted cash and investment securities of $410.4 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, the progress of regulatory interactions, approval (if any) and commercialization efforts with respect to 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 March 31, 2025, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million. Future interest payments and financing fees associated with the loan total $14.5 million, with $8.6 million payable within 12 months. We are contractually required to repay the $50.0 million in full in January 2027.
Leases
We have entered into lease arrangements for facilities, including corporate, laboratories and office space. As of March 31, 2025, we had fixed lease payment obligations of $22.8 million, with $4.2 million payable within 12 months. Following the Lexington Transaction, we assigned our lease for the Lexington Facility to Genezen. As of March 31, 2025, we continue to guarantee $19.6 million of fixed lease payments under the lease for the Lexington Facility until the end of the lease term in May 2029.
Commitments related to uniQure France SAS acquisition (nominal amounts)
In 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 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 March 31, 2025, our remaining commitment amounts include EUR 160.0 million ($166.2 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and European Union. The timing of achieving these milestones and consequently the timing of payments, as well as whether the 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 March 31, 2025, of $1.08/€1.00. As of March 31, 2025, 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.
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Commitments related to licensors and financial advisors
We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our sale of the exclusive global rights of HEMGENIX® to CSL Behring. We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.
Commitments related to the CSA and the DMSA with Genezen
Our CSA with Genezen requires us to take or pay contract manufacturing services for HEMGENIX® during a three-year period ending July 2027. As of March 31, 2025, our remaining minimum purchase commitments to Genezen amount to $33.1 million, with $17.0 million to be paid within the next 12 months. CSL Behring’s minimum purchase commitments to us over the next 12 months are $16.2 million.
Our development and other manufacturing services agreement (“DMSA”) with Genezen requires us to take or pay contract development services during a three-year period ending July 2027. As of March 31, 2025 our remaining minimum purchase commitments amount to $11.6 million with $4.5 million to be paid within the next 12 months.
The table below summarizes our consolidated cash flow data for the three months ended March 31, 2025 and 2024:
Three months ended March 31,
2025
2024
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period
$
160,329
$
244,544
Net cash used in operating activities
(44,100)
(60,575)
Net cash generated from investing activities
20,099
63,985
Net cash generated from financing activities
80,670
—
Foreign exchange impact
1,660
(1,725)
Cash, cash equivalents and restricted cash at the end of period
$
218,658
$
246,229
We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement. We continue to incur losses in the current period. We recorded a net loss of $43.6 million in the three months ended March 31, 2025, respectively, compared to a net loss of $65.6 million during the same period in 2024. As of March 31, 2025, we had an accumulated deficit of $1,173.6 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 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 net proceeds of $70.1 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a 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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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 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.
To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all. If financing is not available when needed, including through debt or equity financings, or is available only on unfavorable terms, we may be unable to meet our cash needs. If we raise additional funds through collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.
Net cash used in operating activities
Net cash used in operating activities was $44.1 million for the three months ended March 31, 2025 and consisted of net loss of $43.6 million adjusted for non-cash items, including depreciation and amortization expense of $4.5 million, amortization of the discount on investment securities of $2.1 million, share-based compensation expense of $4.4 million, $8.4 million of interest expense net of interest paid related to May 2023 Royalty Financing Agreement, a change in deferred taxes of $0.5 million, changes in the fair value of contingent consideration of $1.2 million, and unrealized foreign exchange gains of $7.1 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $6.3 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $3.0 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $3.3 million.
Net cash used in operating activities was $60.6 million for the three months ended March 31, 2024 and consisted of net loss of $65.6 million adjusted for non-cash items, including depreciation and amortization expense of $2.6 million, amortization of the discount on investment securities of $3.7 million, share-based compensation expense of $7.2 million, $10.9 million of interest expense net of interest paid related to May 2023 Royalty Financing Agreement, a change in deferred taxes of $0.7 million, changes in the fair value of contingent consideration of $0.2 million and unrealized foreign exchange losses of $1.4 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $17.0 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $10.4 million. There was a decrease in inventory balances of $2.2 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $8.8 million, primarily related to a decrease of $6.7 million from personnel-related accruals.
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Net cash generated from investing activities
In the three months ended March 31, 2025, we generated $20.1 million in our investing activities compared to generating $64.0 million for the same period in 2024.
Three months ended March 31,
2025
2024
(in thousands)
Cash flows from investing activities
Proceeds from maturity of debt securities
$
85,000
$
150,107
Investment in debt securities
(64,775)
(83,778)
Capital expenditures - European sites
(110)
(1,051)
Capital expenditures - Lexington site
(16)
(1,293)
Net cash generated from investing activities
$
20,099
$
63,985
Net cash generated from financing activities
In the three months ended March 31, 2025, we generated $80.6 million in financing activities compared to generating nil for the same period in 2024.
Three months ended March 31,
2025
2024
(in thousands)
Cash flows from financing activities
Proceeds from public offering of shares in connection with follow on, net of issuance costs
$
80,511
$
—
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans
159
—
Net cash generated from financing activities
$
80,670
$
—
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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Text extracted from the filing as submitted to EDGAR. Formatting, tables and exhibits are simplified for reading; the original document is authoritative for anything you rely on.