uniQure NV
For the period ended Mar 31, 2025. Filed May 9, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2025 · Filed May 9, 2025
QURE$21.70+3.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-05.
0001558370-24-014346
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-09.
0001558370-25-007068
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 182 previous paragraphs and 116 current paragraphs.
50 unchanged; 42 reworded; 24 added; 76 removed; 14 previous and 0 current paragraphs rearranged.
Tables are not compared: 73 previous and 49 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (24)
Current report
Financing
Current report
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.
Current report
RMAT and Breakthrough Therapy Designations
Current report
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.
Current report
In addition to the RMAT and Breakthrough Therapy designations, the FDA previously granted Orphan Drug designation and Fast Track designation to AMT-130.
Current report
Regulatory Alignment
Current report
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.
Current report
In December 2024, following our initial Type B meeting, we announced that we had reached agreement with FDA on key elements of an Accelerated Approval pathway for AMT-130. 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.
Current report
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.
Current report
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.
Current report
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.
Current report
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.
Current report
Contract manufacturing
Current report
General
Current report
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”.
Current report
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.
Current report
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.
Current report
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.
Current report
Our other items, net, for thethree months ended March 31, 2025 and March 31, 2024 were as follows:
Current report
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).
Current report
We recognized interest expense of $1.8 million and $3.7 millionin thethree 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.
Current report
Income tax expense
Current report
Commitments related to the CSA and the DMSA with Genezen
Current report
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.
Removed paragraphs (76)
Previous report
AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying amicro ribonucleic acid (“miRNA”) specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
Previous report
We are currently conducting a multi-center randomized, controlled Phase I/II clinical trial for AMT-130 in the U.S. (“US study”) as well as an open-label Phase Ib/II study in Europe with the same early-manifest criteria for Huntington’s disease as the U.S. study (“European study”). 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.
Previous report
In November 2023, we initiated patient dosing in a third cohort of up to 12 patients across both U.S. and U.K. sites to further investigate both doses of AMT-130 in combination with perioperative immunosuppression. Patient enrollment is ongoing in this cohort with a focus on evaluating near-term safety and tolerability.
Previous report
In July 2024, we announced updated interim clinical data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease.
Previous report
In conjunction with the July 2024 update, we conducted a post-hoc statistical analysis of clinical outcomes for the 21 treated patients at 24 months compared to an expanded, propensity-weighted external control developed in collaboration with the Cure Huntington’s Disease Initiative (CHDI) using data from the TRACK-HD, TRACK-ON and PREDICT-HD natural history studies. The external control includes 154 patients that met the Phase I/II clinical trial eligibility criteria, and whose data contributions were statistically weighted using propensity scoring to closely match the baseline characteristics of patients treated with AMT-130. Disease-related outcomes for these well-balanced cohorts were then compared after 24 months follow-up and are summarized below. Measures of statistical significance in this analysis were based on nominal p values and are unadjusted.
Previous report
Based on data observed as of the July 2024 interim update, AMT-130 remains generally well-tolerated, with a manageable safety profile at both doses. There were no new AMT-130-related serious adverse events reported.
Previous report
Based on the granting of the RMAT designation, we have scheduled a Type B, multi-disciplinary meeting with the U.S. Food and Drug Administration (FDA) in late November 2024 at which we plan to present the most recent clinical data and initiate discussions regarding the potential for an expedited development pathway for AMT-130. We will also discuss with the FDA a future communication plan that is expected to include additional sub-disciplinary meetings to take place in the first half of 2025.
Previous report
Sale of commercial manufacturing activities
Previous report
On June 29, 2024, our affiliates, uniQure Inc. and uniQure biopharma B.V., entered into an asset purchase agreement (the “APA”) with Genezen Holdings Inc. and its subsidiary, Genezen MA, Inc. (together “Genezen”) to sell certain assets and assume certain liabilities related to uniQure’s manufacturing facility (the “Lexington Facility”) and operations in Lexington, Massachusetts (the “Lexington Transaction”). The Lexington Transaction closed on July 22, 2024 (the “Closing”). Our Chief Executive Officer, Matthew Kapusta, joined the board of directors of Genezen in connection with the Closing.
Previous report
In conjunction with the Lexington Transaction, Genezen extended offers of employment to a significant majority of our employees located at the Lexington Facility, with the remaining employees terminated effective August 30, 2024.
Previous report
As consideration for the Lexington Transaction, we received (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 and (iii) a right to purchase HEMGENIX® at terms considered favorable to market terms (the “Consideration”).
Previous report
In connection with the Closing, we, Genezen and the landlord of the Lexington Facility entered into an agreement for us to assign and Genezen to assume the existing lease agreement between us and the landlord. We also amended our original July 2013 guarantee to continue guaranteeing rental payments owed by Genezen until the end of the current term on May 31, 2029. In the event of Genezen’s default related to rental payments owed to the landlord, we are entitled to terminate the assignment agreement and step into the original lease agreement.
Previous report
At the Closing, we entered into a commercial supply agreement (“CSA”) with Genezen. Pursuant to the terms of the CSA, the parties agreed to subcontract the manufacturing of HEMGENIX® to Genezen. The CSA includes a minimum term of three years and minimum purchase commitments of HEMGENIX® commercial supplies of $43.3 million over the first three years, unless certain contractual provisions are triggered. Our obligations with respect to the supply of HEMGENIX® to CSL Behring remain in effect notwithstanding the subcontracting to Genezen.
Previous report
We recorded a $1.2 million gain from the divestment. We paid a total of $8.3 million to Genezen and a third party related to adjustments of working capital and to obtain consent to proceed with the divestment.
Previous report
Additionally, we entered into a development and other manufacturing services agreement (“DMSA”) with Genezen at Closing. Pursuant to the DMSA, we are entitled to preferred customer status to receive manufacturing and development services to support our investigational gene therapy programs and other services related to HEMGENIX® (other than our manufacturing and supply obligations under the CSA). The DMSA has a minimum term of three years and requires us to purchase services for a total minimum of $14.0 million.
Previous report
Hercules Loan Repayment and Amendment
Previous report
In connection with the Closing on July 22, 2024, we and Hercules amended the 2023 Amended Facility (“2024 Amended Facility”). As a condition to Hercule’s consent to the Lexington Transaction, on July 19, 2024 we prepaid $50.0 million of the total $100.0 million principal outstanding. The remaining $50.0 million principal outstanding will need to be repaid on January 5, 2027 (the “Maturity Date”).
Previous report
Results of the business review
Previous report
On July 23, 2024, we announced the Closing and on August 1, 2024, we announced an organizational restructuring (the “Restructuring”). These actions were the outcome of a recently completed, comprehensive review of our operations with the goals of conserving capital and streamlining the organization. As part of these changes, we eliminated approximately 300 positions or 65% of our workforce. We estimate that we will incur costs in the range of $6.5 million to $7.5 million related to employee severance costs depending on whether or not employees continue to provide services through the residual service periods. The Restructuring was subject to the review by our Amsterdam-based works council, which was completed in the third quarter of 2024. We expect the Restructuring to be substantially completed by the end of 2024 with some employees continuing through second quarter 2025.
Previous report
In August 2023, the FDA cleared the IND application for AMT-260, our investigational gene therapy candidate for refractory MTLE. AMT-260 is comprised of an AAV9 vector that locally delivers two engineered miRNAs designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE.
Previous report
AMT-191 is our investigational gene therapy candidate for the treatment of Fabry disease. AMT-191 is comprised of an AAV5 capsid that incorporates theα-galactosidase A (“GLA”) transgene and a proprietary, highly potent, liver-specific promoter. In November 2023 we announced that the FDA had cleared the IND application for AMT-191.
Previous report
AMT-162 is our investigational gene therapy candidate for a one-time, intrathecally administered investigational gene therapy for SOD1-ALS. AMT-162 is comprised of a recombinant AAVrh10 vector that expresses a miRNA designed to knock down the expression of SOD1 with the goal of slowing down or potentially reversing the progression of ALS in patients with SOD1 mutations. The FDA has cleared the IND application for AMT-162 and has granted Orphan Drug and Fast Track Designation.
Previous report
In October 2024, we announced the first patient has been dosed in a Phase I/IIa clinical trial of AMT-162 for the treatment of SOD1-ALS. The first-in-human Phase I/II clinical trial is a U.S.-based, multi-center, open-label trial consisting of three cohorts with up to four patients each receiving a one-time intrathecal infusion with immunosuppression. Safety, tolerability and early signs of efficacy will be evaluated in the study.
Previous report
Intangible asset
Previous report
We entered into the APA with Genezen on June 29, 2024 to divest our commercial manufacturing activities in return for the Consideration. The Lexington Transaction closed on July 22, 2024. Part of the Consideration received was 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 market value required us to estimate the current and future commercial supply prices of HEMGENIX® to compare these to the contractually agreed terms. 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 and relevant market data as reference points.
Previous report
If the estimated commercial supply price used in the valuation had increased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received as well as the gain recorded on the divestment would have been increased by $3.0 million. If the estimated commercial supply price used in the valuation had been decreased by 5% per drug product batch, then the fair market value of the intangible asset and the consideration received on the divestment would have been decreased by $2.9 million. This would have resulted in a loss on divestment of $1.7 million instead of the $1.2 million gain presented.
Previous report
Revenues
Previous report
Our revenues for the three months ended September 30, 2024 and 2023 were as follows:
Previous report
Cost of contract manufacturing
Previous report
R&D expenses
Previous report
In the three months ended September 30, 2024 and September 30, 2023, we incurred costs of $1.6 million and $4.7 million respectively, for the development of AMT-260. In August 2023, the FDA cleared our IND application, and we continue to incur costs for the preparation of a Phase I clinical trial.
Previous report
Preclinical programs and platform development
Previous report
On January 31, 2023, we entered into a global licensing agreement with Apic Bio for AMT-162. In the three months ended September 30, 2024 and September 30, 2023, we incurred costs of $0.9 million and $2.4 million, respectively, to initiate a Phase I/II clinical trial.
Previous report
Selling, general and administrative expenses for the three months ended September 30, 2024 were $11.6 million, compared to $18.1 million for the same period in 2023.
Previous report
We recognized $1.2 million net gain resulting from the sale of the commercial manufacturing activities located in Lexington, MA in July 2024 in other income.
Previous report
We recognized $1.1 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the three months ended September 30, 2024, compared to $1.4 million for the same period in 2023.
Previous report
Other items, net for the periods presented primarily related to income from the subleasing of our Amsterdam facility and Lexington research and development facility and expenses we incur in relation to the subleasing facility.
Previous report
We recognized $16.6 million in interest expense for the three months ended September 30, 2024 and $15.4 million for the three months ended September 30, 2023. Our interest expense in 2024 increased due to an increase of $1.0 million in non-cash interest expense related to the Royalty Financing Agreement that we entered into in May 2023.
Previous report
Income tax benefit
Previous report
We recognized $0.0 million of deferred tax benefit in the three months ended September 30, 2024, and $0.1 million of deferred tax benefit for the same period in 2023.
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Reworded paragraphs (42)
Previous wording
Huntington’s disease program (AMT-130) updated interim clinical data
Current wording
Huntington’s disease program (AMT-130)
Previous wording
In June 2024, we announced that the U.S. Food and Drug Administration (“FDA”) granted 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. The designation follows the FDA’s review of interim Phase I/II clinical data for AMT-130 announced in December 2023 and is based on an analysis comparing these 24-month clinical data to a non-concurrent criteria-matched natural history cohort.
Current wording
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.
Previous wording
We are initiating a Phase I/IIa clinical trial that will be conducted in the United States and consist 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 (“POC”) data. The first patient has been enrolled into the observational phase of the Phase I/II clinical trial of AMT-260 for the treatment of mTLE. 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. Due to the more restrictive inclusion criteria for these sentinel patients, enrollment has taken longer than expected. We are rapidly activating recruitment sites with 10 centers currently open and an additional two sites expected to be activated by the end of 2024.
Current wording
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.
Previous wording
In August 2024, we announced that the first patient has been dosed in 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.
Current wording
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.
Previous wording
In September 2024, we announced the FDA has granted Orphan Drug Designation to AMT-191 and in October 2024, the FDA granted Fast Track Designation to AMT-191.
Current wording
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.
Previous wording
As of September 30, 2024 and December 31, 2023, we had cash and cash equivalents and investment securities of $435.2 million and $617.9 million, respectively. We had a net loss of $44.4 million and $166.3 million in the three and nine months ended September 30, 2024, respectively, compared to a net loss of $89.6 million and $235.3 million for the same periods in 2023. As of September 30, 2024 and December 31, 2023, we had accumulated deficits of $1,056.7 million and $890.4 million, respectively.
Current wording
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.
Previous wording
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 nine months ended September 30, 2024 with the exception of determining the valuation of the intangible asset with favorable terms recorded as part of the Lexington Transaction.
Current wording
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.
Previous wording
We entered into a development and commercial supply agreement with CSL Behring in June 2020. Prior to Closing, we recognized the cost to manufacture HEMGENIX® under such agreement as cost of contract manufacturing.
Current wording
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.
Previous wording
Our other income primarily consists of the net gain resulting from the sale of the commercial manufacturing activities located in Lexington, MA in July 2024, payments received to subsidize our research and development efforts, and income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility which, commenced in September 2024.
Current wording
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.
Previous wording
Our other expense primarily consists of costs incurred under our commercial supply agreement with 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 expense also consists ofexpenses we incur in relation to our subleasing income.
Current wording
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 ofexpenses we incur in relation to our subleasing income.
Previous wording
Our revenue for the nine months ended September 30, 2024 and 2023 was as follows:
Current wording
Our revenue for thethree months ended March 31, 2025 and 2024 was as follows:
Previous wording
We recognize license revenues from CSL Behring, related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended September 30, 2024, we recognized $2.1 million of license revenues, compared to $0.5 million for the same period in 2023.
Current wording
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.
Previous wording
For the nine months ended September 30, 2024 and 2023 we recognized $10.6 million and $1.3 million of collaboration revenue for CSL Behring, respectively. The increase in collaboration revenue of $9.3 million in the nine months ended September 30, 2024 compared to the same period in 2023 was primarily related to additional development and other services provided to CSL Behring in relation to the CSL Behring Agreement.
Current wording
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.
Previous wording
We recognized contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring prior to the Closing of 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 September 30, 2024, compared to $0.3 million for the same period in 2023.
Current wording
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 thethree months ended March 31, 2025, compared to $4.0 million for the same period in 2024.
Previous wording
We incurred $0.8 million of cost of contract manufacturing related to the manufacture of HEMGENIX® prior to the Closing of the Lexington Transaction in the three months ended September 30, 2024, compared to $1.0 million cost of contract manufacturing in the three months ended September 30, 2023.
Current wording
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.
Previous wording
R&D expenses for the three months ended September 30, 2024 were $30.6 million, compared to $65.4 million for the same period in 2023. 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.
Current wording
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.
Previous wording
In the three months ended September 30, 2024 and September 30, 2023, we incurred costs of $4.5 million and $3.1 million respectively. Our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trials in the U.S. and in Europe. We completed enrollment of the first two cohorts in the U.S. in March 2022 and in Europe in June 2023.
Current wording
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.
Previous wording
In the three months ended September 30, 2024 and September 30, 2023, we incurred costs of $0.8 million and $0.4 million, respectively, related to our development of AMT-191. In November 2023, the FDA cleared the IND application, and we started incurring additional costs for the Phase I/II clinical trial preparation.
Current wording
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.
Previous wording
In the three months ended September 30, 2024 and September 30, 2023, we incurred $1.0 million and $3.1 million of costs, respectively, primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Current wording
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.
Previous wording
We recognized $1.5 million net in other expenses during the three months ended September 30, 2024 (nil in three months ended September 30, 2023) related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.
Current wording
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.
Previous wording
Our other non-operating items, net, for the three months ended September 30, 2024 and 2023 were as follows:
Current wording
Our other non-operating items, net, for thethree months ended March 31, 2025 and March 31, 2024 were as follows:
Previous wording
We recognize interest income associated with our cash and cash equivalents, investment securities and convertible note. We recognized $17.2 million in interest income in the nine months ended September 30, 2024, compared to $12.4 million in the same period in the prior year. Our interest income increased by $4.8 million due to the interest income earned on investment securities as well as cash on hand during the nine months ended September 30, 2024.
Current wording
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.
Previous wording
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 $7.6 million during the three months ended September 30, 2024, compared to a net gain of $0.2 million during the same period in 2023.
Current wording
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.
Previous wording
We recognized $1.6 million of deferred tax expense in thenine months ended September 30, 2024, and $1.1 million of deferred tax benefit for the same period in 2023.
Current wording
We recognized $0.5 million of deferred tax expense in thethree months ended March 31, 2025, and $0.7 million of deferred tax expense for the same period in 2024.
Previous wording
As of September 30, 2024, we had cash and cash equivalents, restricted cash and investment securities of $436.7 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 end 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. We expect that we will require additional funding if we decide to advance AMT-130 for our Huntington’s disease gene therapy program or any of our other product candidates into late-stage clinical development. Our material cash requirements include the following contractual and other obligations:
Current wording
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:
Previous wording
As of September 30, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc. (“Hercules”) for an aggregate principal amount of $50.0 million. On July 19, 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 $18.2 million, with $6.5 million payable within 12 months. We are contractually required to repay the $50.0 million in full in January 2027.
Current wording
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.
Previous wording
We entered into lease arrangements for facilities, including corporate, manufacturing and office space. As of September 30, 2024, we had fixed lease payment obligations of $25.3 million, with $4.3 million payable within 12 months. Following the closing of the Lexington Transaction on July 22, 2024 we assigned our lease for the Lexington Facility to Genezen. This reduced our fixed lease payment obligations by $21.8 million, including $4.4 million payable within 12 months. We continue to guarantee such payments until the end of the lease term in May 2029.
Current wording
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.
Previous wording
In relation to our acquisition of uniQure France SAS in 2021, 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. As of September 30, 2024, our remaining commitment amounts include a EUR 30.0 million ($33.5 million) milestone payment due upon treating the first patient in a Phase I/II clinical trial for AMT-260 and EUR 160.0 million ($178.6 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 milestones will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of September 30, 2024, of $1.12/€1.00. As of September 30, 2024, we expect these obligations will become payable between 2024 and 2033. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.
Current wording
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.
Previous wording
Our CSA with Genezen requires us to take or pay contract manufacturing services for HEMGENIX® during a three-year period ending July 22, 2027. As of September 30, 2024, our remaining minimum purchase commitments to Genezen amount to $39.9 million, with $14.7 million to be paid within the next 12 months. CSL Behring’s minimum purchase commitments to us over the next 12 months are $11.9 million.
Current wording
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.
Previous wording
Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 22, 2027. As of September 30, 2024 our remaining minimum purchase commitments amount to $13.3 million with $3.5 million to be paid within the next 12 months.
Current wording
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.
Previous wording
The table below summarizes our consolidated cash flow data for the nine months ended September 30, 2024 and 2023:
Current wording
The table below summarizes our consolidated cash flow data for the three months ended March 31, 2025 and 2024:
Previous wording
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 $44.4 million and $166.3 million in the three and nine months ended September 30, 2024, respectively, compared to a net loss of $89.6 million and $235.3 million during the same periods in 2023. As of September 30, 2024, we had an accumulated deficit of $1,056.7 million.
Current wording
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.
Previous wording
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 from selling a portion of royalties due from our collaboration partner CSL Behring. We collected $100.0 million in July 2023 related to the first sale milestone of HEMGENIX® in the U.S., and are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from royalty financing) on net sales of HEMGENIX®.
Current wording
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®.
Previous wording
On May 12, 2023 we and Hercules amended the 2021 Restated Facility. The 2023 Amended Facility extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027. In connection with the closing of the Lexington Transaction on July 22, 2024 we amended the 2023 Amended Facility and repaid $50.0 million of the principal. 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. Under the 2024 Amended Facility, 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.
Current wording
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.
Previous wording
We are subject to certain covenants under the 2024 Amended 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 2024 Amended Facility permits us to issue up to $500.0 million of convertible debt.
Current wording
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.
Previous wording
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 financing, 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.
Current wording
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.
Previous wording
Net cash used in operating activities was $129.9 million for the nine months ended September 30, 2024 and consisted of net loss of $166.3 million adjusted for non-cash items, including depreciation and amortization expense of $8.3 million, amortization of the discount on investment securities of $8.5 million, share-based compensation expense of $17.5 million, $37.7 million of interest expense related to the Royalty Financing Agreement, a change in deferred taxes of $1.6 million, changes in the fair value of contingent consideration of $2.0 million, a provision for inventory write-downs of $6.3 million and unrealized foreign exchange gains of $5.7 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $16.2 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $5.8 million. There was an increase in inventory balances of $3.9 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $6.6 million.
Current wording
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.
Previous wording
Net cash used in operating activities was $96.4 million for the nine months ended September 30, 2023 and consisted of net loss of $235.3 million adjusted for non-cash items, including depreciation and amortization expense of $7.7 million, amortization of the premium/discount on investment securities of $6.5 million, share-based compensation expense of $28.1 million, changes in the fair value of contingent consideration of $15.4 million, unrealized foreign exchange losses of $3.1 million, $14.9 million of interest expense related to the Royalty Financing Agreement and a change in deferred taxes of $1.1 million. Net cash used in operating activities also included favorable changes in operating assets and liabilities of $77.0 million. There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $96.6 million, primarily 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 $8.7 million.
Current wording
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.
Previous wording
Net cash generated from / (used in) investing activities
Current wording
Net cash generated from investing activities
Previous wording
In the nine months ended September 30, 2024, we generated $189.6 million in our investing activities compared to using $265.2 million for the same period in 2023.
Current wording
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.
Showing the first 40 of 42 pairs. Open the source sections for the rest.
Rearranged previous paragraphs (14)
Previous report — wording carried elsewhere
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.
Previous report — wording carried elsewhere
For the three months ended September 30, 2024 and 2023 we recognized $0.2 million and $0.6 million of collaboration revenue for CSL Behring, respectively.
Previous report — wording carried elsewhere
We recognize interest income associated with our cash and cash equivalents, investment securities and convertible promissory note. We recognized $4.9 million in interest income in the three months ended September 30, 2024, compared to $7.5 million in the same period in the prior year. Our interest income decreased by $2.6 million due to less interest income earned on investment securities during the three months ended September 30, 2024.
Previous report — wording carried elsewhere
The following table presents a comparison of our results of operations for the nine months ended September 30, 2024 and 2023:
Previous report — wording carried elsewhere
We recognize license revenues from CSL Behring, related to royalty payments owed on HEMGENIX® sales, when earned. For thenine months ended September 30, 2024, we recognized $5.2 million of license revenues, compared to $1.3 million for the same period in 2023.
Previous report — wording carried elsewhere
We recognized contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring prior to the Closing of the Lexington Transaction. Contract manufacturing revenues were realized when earned upon sales of HEMGENIX® drug product to CSL Behring. We recognized $6.1 million contract manufacturing revenues in thenine months ended September 30, 2024, compared to $6.6 million for the same period in 2023.
Previous report — wording carried elsewhere
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.
Previous report — wording carried elsewhere
R&D expenses for the nine months ended September 30, 2024 were $104.9 million, compared to $172.2 million for the same period in 2023. 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.
Previous report — wording carried elsewhere
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
Previous report — wording carried elsewhere
In the nine months ended September 30, 2024 and September 30, 2023, we incurred costs of $3.6 million and $1.8 million, respectively, related to our development of AMT-191. In November 2023, the FDA cleared the IND application, and we started incurring additional costs for the Phase I/II clinical trial preparation.
Previous report — wording carried elsewhere
In the nine months ended September 30, 2024 and September 30, 2023, 2023, we incurred $2.3 million and $7.2 million of costs, respectively, primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Previous report — wording carried elsewhere
We recognized $1.5 million in net other expenses during the nine months ended September 30, 2024 (nil in same period in 2023) related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.
Previous report — wording carried elsewhere
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.
Previous report — wording carried elsewhere
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 $5.4 million during the nine months ended September 30, 2024, compared to a net loss of $1.8 million during the same period in 2023.