uniQure NV
For the period ended Dec 31, 2024. Filed Feb 27, 2025.
View on SEC.govAbout this filing
A company's yearly report to the SEC, with audited financial statements. It is the fullest account a company gives of its business, its finances and the risks it sees.
FY2024 · Filed Feb 27, 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-K
Period ended 2023-12-31; filed 2024-02-28.
0001558370-24-001915
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-K
Period ended 2024-12-31; filed 2025-02-27.
0001558370-25-001699
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 145 previous paragraphs and 145 current paragraphs.
51 unchanged; 45 reworded; 47 added; 47 removed; 2 previous and 2 current paragraphs rearranged.
Tables are not compared: 65 previous and 65 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 (47)
Current 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.
Current report
We are currently conducting the U.S. and European studies. We completed the enrollment of all 26 patients in the first two cohorts of our US study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023. As of February 2025, we have enrolled 12 patients into a third cohort to further investigate both doses of AMT-130 together with perioperative immunosuppression using the current, established stereotactic administration procedure.
Current report
RMAT Designation
Current report
In June 2024, we announced that the FDA granted us RMAT designation for AMT-130.
Current report
Updated Interim Data
Current report
In July 2024, we announced updated interim clinical data, including up to 24 months of follow-up data from 29 patients enrolled in the U.S. study and the European study as of a March 31, 2024 cut-off date.
Current report
Regulatory Alignment
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.
Current report
Other clinical candidates
Current report
In November 2024, we announced that the that the first patient was dosed in the GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mTLE. In addition, the FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere.
Current report
In August 2024, we announced that the first patient was dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease. In February 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.
Current report
In October 2024, we announced the first patient was dosed in a Phase I/IIa clinical trial of AMT-162 for the treatment of SOD1-ALS. In January 2025, we announced that we completed the enrollment of the first cohort of the clinical trial and that we would proceed enrolling the second cohort of the clinical trial.
Current report
Sale of commercial manufacturing activities
Current report
In June 2024, we and Genezen entered into the Lexington Transaction, which closed in July 2024. Genezen acquired manufacturing facility including equipment and related manufacturing operations with a carrying value of $15.2 million, inventory with a carrying value of $8.8 million and certain other assets (including allocated goodwill) with a carrying value of $2.8 million associated with the Lexington Facility on Closing.
Current report
As consideration for the Lexington Transaction, we received from Genezen (i) shares of newly issued Series C preferred stock of Genezen Holdings Inc. valued at $12.5 million, which are convertible into common stock and will accrue an 8.0% per annum cumulative dividend, (ii) a convertible promissory note with a nominal amount of $12.5 million, bearing interest at 8.0% per annum and maturing 63 months following the date of issuance valued at $13.3 million and (iii) a right to purchase HEMGENIX® at terms considered favorable to market terms (the “Consideration”) valued at $16.7 million. We also included a firm purchase commitment liability, valued at $8.8 million for our customer contract with CSL Behring inherently related to the right to purchase HEMGENIX® in the consideration received.
Current report
We recorded a $1.2 million gain from the divestment. We paid a total of $8.3 million to Genezen and CSL Behring related to adjustments of working capital and to obtain consent to proceed with the divestiture.
Current 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.
Current report
At the Closing, we entered into a commercial supply agreement (“CSA”) with Genezen. Pursuant to the CSA, Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX® on our behalf. The CSA includes a minimum term of three years and minimum purchase commitments of HEMGENIX® commercial supplies of $43.3 million over the first three years, unless certain contractual provisions are triggered. Our obligations with respect to the supply of HEMGENIX® to CSL Behring remain in effect notwithstanding the subcontracting to Genezen.
Current report
Organizational restructuring
Current report
In August 2024, we announced the Restructuring to conserve capital and streamline the organization. As a result of the Restructuring and Lexington Transaction, we eliminated approximately 300 positions or 65% of our workforce.
Current report
We substantially completed the Restructuring at the end of 2024. We have incurred $5.2 million of employee severance costs in the year ended December 31, 2024 and estimate that we will incur additional costs in the range of $0.5 million to $1.0 million in 2025.
Current report
As of December 31, 2024 we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date. Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date.
Current report
Hercules loan repayment and amendment
Current report
In connection with the Closing we once more amended the facility. As a condition to Hercules’ consent to the Lexington Transaction we prepaid $50.0 million of the total $100.0 million principal outstanding in July 2024. The remaining $50.0 million principal outstanding will need to be repaid at the Maturity Date.
Current report
Financing
Current report
In January 2025, we raised $70.1 million of net proceeds, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a price to the public of $17.00 per ordinary share. In February 2025, we raised an additional $10.6 million in net proceeds upon the underwriters’ exercise of their option to purchase an additional 0.7 million ordinary shares at the public offering price.
Current report
Intangible asset
Current report
Part of the consideration received in the Lexington Transaction included a right, with an estimated fair market value of $16.7 million, to purchase HEMGENIX® from Genezen up to the termination of our supply agreement with CSL Behring at terms considered favorable to market terms. The determination of the fair value required us to estimate the current and future commercial supply prices of HEMGENIX® to compare these to the contractually agreed terms at Closing. Prices for the commercial supply of HEMGENIX® are not readily observable in the market. Therefore, our estimate of the commercial supply price was based on what a market participant would be willing to pay in a comparable transaction, using industry benchmarks, relevant market data, current and historical commercial supply agreements, as well as historical production cost information as reference points.
Current 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.
Current report
Date at which we expect to satisfy the commitment under the Royalty Financing Agreement
Current report
On May 12, 2023, we entered into the Royalty Financing Agreement with the Purchaser. Under the terms of the Royalty Financing Agreement we received an upfront payment of $375.0 million in exchange for its rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us (refer to Note 13 “Royalty Financing Agreement” for further details). Pursuant to the agreement, the total amount of royalties to be received by the Purchaser is subject to an increasing cap equal to (i) $693.8 million up to the First Hard Cap Date if such thresholds are met or, if such cap is not met by the First Hard Cap Date (ii) $843.8 million through the Second Hard Cap Date. If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to us. We have no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.
Current report
As of December 31, 2024, we expect to satisfy our commitment to the Purchaser prior to the Second Hard Cap Date. Until December 2024, we assumed that we would satisfy the commitments to the Purchaser prior to the First Hard Cap Date. As a result of this change in estimate we expect that our total payments of interest expense will increase from $318.8 million by $150.0 million to $468.8 million over a term that was extended from 9 years to 15.5 years.
Current report
In the year ended December 31, 2024, we adopted Accounting Standards Update (ASU) 2023-07,Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures (“ASU 2023-07”). This update requires us to disclose segment expenses that are significant and regularly provided to our chief operating decision maker (“CODM”). In addition, ASU 2023-07 requires us to disclose the title and position of its CODM and how the CODM uses segment profit or loss information in assessing segment performance and deciding how to allocate resources. The adoption of this update did not have a material impact on our financial position or results of operations but resulted in expanded disclosures in the segment reporting section of the financial statements. We adopted ASU 2023-07 using a retrospective transition method.
Current report
In 2008, we entered into a license agreement with St. Jude Children’s Research Hospital (“St. Jude”), which we amended in 2012. Under this license agreement, St. Jude has granted us an exclusive license, with a right to sublicense, to patent rights relating to expression of hFIX in gene therapy vectors, to make, import, distribute, use, and commercialize products containing hFIX covered by a valid patent claim in the field of gene therapy for treatment or prophylaxis of hemophilia B. The majority of the U.S. patent rights will expire in 2028 and the European patent rights (along with one U.S. patent) will expire in 2025.
Current report
With respect to our collaboration with CSL Behring, we have agreed with St. Jude on an apportionment of certain amounts we receive from CSL Behring as sublicensing revenue that is equivalent to a low-single digit percentage of such amounts. The agreement will remain in effect until no further payment is due relating to any licensed product under this agreement or either we or St. Jude exercise our rights to terminate it. St. Jude may terminate the agreement in specified circumstances relating to our insolvency. We may terminate the agreement for convenience at any time subject to a specified notice period.
Current report
We provided contract manufacturing service to CSL Behring between April 2022 and July 2024. We transferred these activities to Genezen as part of the Lexington Transaction. Following the Closing of the Lexington Transaction, title to HEMGENIX® supply directly passes from the contract manufacturer, Genezen, to CSL Behring. We do not control HEMGENIX® before it is transferred to CSL Behring. We arrange for HEMGENIX® to be provided by Genezen to CSL Behring. We determined that we are an agent in the sale of HEMGENIX® to CSL Behring.
Current report
As a result of being an agent, we recognize corresponding costs related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring and income related to the release of liabilities associated with expected net losses in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss. We recognized contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring when earned upon sales of HEMGENIX® to CSL Behring. We recognized $6.1 million, $10.8 million and $1.7 million contract manufacturing revenues in the years ended December 31, 2024, 2023 and 2022, respectively.
Current report
Hemophilia B (AMT-061)
Current report
In year ended December 31, 2024, we recognized a $1.2 million net gain related to the Lexington Transaction, with no such amounts recognized in 2023 and 2022.
Current report
We recognized $3.2 million in net other expenses during the year ended December 31, 2024 (nil in 2023 and 2022) related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.
Showing the first 40 of 47 passages. Open the source sections for the rest.
Removed paragraphs (47)
Previous report
Business Developments
Previous report
Below is a summary of our recent significant business developments:
Previous report
We are currently conducting a multi-center randomized, controlled, and blinded Phase I/II clinical trial for AMT-130 in the U.S. in which 26 patients with early-manifest Huntington’s disease have been enrolled. The low-dose cohort of this trial includes 10 patients, of which six patients received treatment with AMT-130 and four patients received imitation surgery. The high-dose cohort includes 16 patients, of which 10 patients received treatment with AMT-130 and six patients received imitation surgery. Patients in the high-dose cohort that received imitation surgery had the option to cross over after 12 months if they met the inclusion criteria for the study. In July 2022, we began crossing over patients in the high-dose cohort who received the imitation surgical procedure. Four of the six control patients in the high-dose cohort have been crossed over to treatment (three patients received the high dose and one patient received the low dose). The remaining two control patients in the high-dose cohort did not meet all the inclusion criteria for the study and were not eligible for crossover. All four crossover patients received a short course of immunosuppression therapy concurrent with the administration of AMT-130.
Previous report
We are also conducting an open-label Phase Ib/II study in the EU and the United Kingdom, which has enrolled 13 patients with the same early-manifest criteria for Huntington’s disease as the U.S. study. Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the subsequent high-dose cohort.
Previous report
In November 2023, we began enrolling patients in a third cohort to further investigate both doses in combination with perioperative immune suppression with a focus on evaluating near-term safety. Up to 12 patients will be treated in this cohort, all of whom will receive AMT-130 using the current, established stereotactic neurosurgical delivery procedure.
Previous report
In June 2023, we announced interim data, including up to 24-month follow-up, from 26 patients enrolled in the ongoing U.S. Phase I/II clinical trial of AMT-130. In December 2023 we announced updated interim data, including up to 30-month follow-up from the 26 patients enrolled in the ongoing U.S. Phase I/II clinical trial of AMT-130 as well as the 13 patients enrolled in the European Phase Ib/II trial. See “Business —Our Development of AMT-130 for Huntington’s Disease” for further information on these interim data and the ongoing clinical trials of AMT-130.
Previous report
Amyotrophic lateral sclerosis program (AMT-162)
Previous report
On January 31, 2023, we announced that we had entered into a global licensing agreement with Apic Bio for a one-time, intrathecally administered investigational gene therapy for ALS caused by mutations in SOD1.
Previous report
We made an initial cash payment in February 2023 of $10.0 million to Apic Bio that was recognized as a research and development expense. We owe up to $43.0 million in milestone payments to Apic Bio if AMT-162 is approved for commercialization in the U.S. and Europe.
Previous report
Temporal lobe epilepsy program (AMT-260)
Previous report
AMT-260 is our gene therapy candidate for refractory MTLE. We acquired AMT-260 through our acquisition of uniQure France SAS in July 2021. In September 2023, following the clearance of an IND for AMT-260 in August 2023, we paid EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS, to settle a milestone payment owed in relation to our 2021 acquisition.
Previous report
Reorganization
Previous report
On October 5, 2023, we announced that we are implementing a Reorganization. As a result of the Reorganization, we discontinued investments in more than half of our research programs, including AMT-210 for the treatment of Parkinson’s disease and certain technology projects. Following the Reorganization, we are prioritizing advancing our clinical-stage programs to clinical proof of concept.
Previous report
As a result of the Reorganization, we eliminated approximately 20% of our total workforce and closed our research laboratory in Lexington, Massachusetts. We completed the Reorganization by the end of fiscal year 2023 and incurred costs of approximately $2.5 million for cash expenditures related to employee severance costs. We also incurred costs associated with the closure of a research laboratory in Lexington and associated fixed assets of which the carrying values were determined to not be recoverable as of the cease-use date in November 2023. As part of the Reorganization, we also consolidated all GMP manufacturing into our Lexington manufacturing facility and consolidated process and analytical development into our Amsterdam, Netherlands facility.
Previous report
As a result of the significant reduction in research activities, Ricardo Dolmetsch, Ph.D., departed as Chief Scientific Officer, effective October 4, 2023. Dr. Dolmetsch served as a scientific consultant through the end of the year. In connection with Dr. Dolmetsch’s transition, the Board of Directors of the Company have appointed Richard Porter, Ph.D., our current Chief Business Officer, to serve as the Company’s Chief Business and Scientific Officer, effective as of October 5, 2023.
Previous report
Hercules amendment
Previous report
Investment in debt securities
Previous report
In July and September 2023, we invested $272.0 million and EUR 87.0 million (or a total of $366.4 million as of the investment dates) of our cash and cash equivalents into short-term U.S. and European government bonds that are U.S. dollar and euro denominated, respectively. Our investment policy requires us to invest in bonds with the highest investment grade credit rating. As of December 31, 2023, the bonds have remaining maturities ranging from less than one month to seven months. We classify these bonds as held-to-maturity.
Previous report
In June 2023, the first sale of HEMGENIX® in the U.S. occurred, and in July 2023 we collected the $100.0 million owed to us under the CSL Behring Agreement.
Previous report
Compared to the year ended December 31, 2023, we expect that our research and development expenses will decline following the Reorganization, until such time we decide to advance one of our gene therapy product candidates into late-stage clinical development.
Previous report
In prior periods, we have considered the following to be critical accounting estimates but have determined that these are no longer considered critical for the current year:
Previous report
Valuation allowance related to Dutch deferred tax assets
Previous report
We are subject to corporate taxes in the Netherlands. We have been incurring net operating losses in accordance with the corporate tax laws in almost all years since we founded our business.
Previous report
As of December 31, 2022, the total amount of net operating losses carried forward under the Dutch tax regime was $264.0 million (December 31, 2021: $228.5 million). We have historically recorded a full valuation allowance. We evaluate all positive and negative evidence including future income from the CSL Behring Agreement in assessing the need for such a full valuation allowance. We concluded that as of December 31, 2022 it is more likely than not that the remaining deferred tax assets will not be realized. If we would have released the full valuation allowance as of December 31, 2022, then we would have recorded up to $74.5 million of deferred tax income during the year ended December 31, 2022. This is no longer considered a critical accounting estimate as future income from the CSL Behring Agreement is not expected to result in a taxable profit.
Previous report
On June 24, 2020 (“ Signing Date”), we entered into the CSL Behring Agreement. The transaction became effective on Closing.
Previous report
As of Closing, we identified two material performance obligations related to the CSL Behring Agreement:
Previous report
We determined that the fixed upfront payment of $450.0 million and the $12.4 million that we received in relation to the certain reimbursable activities to fulfill the transfer of global rights (“Additional Covenants”) in the year ended December 31, 2021 should be allocated to the License Sale. In addition, we concluded that $255.0 million of variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well. We determined that the License Sale was completed on May 6, 2021, when we transferred the license and CSL Behring assumed full responsibility for the development and commercialization of the Product. Upon the Closing, we evaluated the amounts of potential payments and the likelihood that the payments will be received. We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since we cannot control the achievement of regulatory and first commercial sales milestones, we concluded that all potential payments were constrained as of Closing. We determined that we would recognize revenue related to these payments, only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter. We will include payments related to sales milestones in the transaction price when their achievement becomes probable, and we will include royalties on the sale of Product once these have been earned.
Previous report
None.
Previous report
We recognize contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring. Contract manufacturing revenues are realized when earned upon sales of HEMGENIX® to CSL Behring. We recognized $10.8 million and $1.7 million contract manufacturing revenues in the year ended December 31, 2023 and 2022, respectively. We did not recognize such revenues in 2021 as we started contract manufacturing activities to supply CSL Behring with HEMGENIX® following their submission of a BLA and MAA in the spring of 2022.
Previous report
We recognized collaboration revenues associated with certain pre-clinical analytical development and process development activities that were reimbursable to us by Bristol-Myers Squibb (“BMS”) under the initial collaboration, research and license agreements (“BMS CLA”) and the amended agreements (“amended BMS CLA”) as well as other related agreements. Collaboration revenue related to these contracted services were recognized when performance obligations were satisfied. We recognized nil, $1.8 million and $4.2 million of collaboration revenue for the years ended December 31, 2023, 2022 and 2021, respectively.
Previous report
Etranacogene dezaparvovec (AMT-060/061)
Previous report
The decrease of $3.8 million in externals cost in the year ended December 31, 2023 compared to 2022 is a result of transitioning the activities to CSL Behring in December 2022 and winding down the support in early 2023. The decrease of $6.3 million external cost in the year ended December 31, 2022 compared to 2021 is a result of CSL Behring being responsible from May 2021 onwards.
Previous report
We recognized nil in other income of employee retention credit under the U.S. CARES Act in the year ended December 31, 2023, compared to nil and $2.6 million of such income for the same periods in 2022 and 2021, respectively.
Previous report
Deferred tax income recorded in 2022 results from deferred tax benefits recorded related to the buildup of net operating losses by the French entity which are partially offset by deferred tax expense recorded in the U.S. as a result of the consumption of net operating losses. In addition, we recorded deferred tax expense resulting from the release of valuation allowance for the tax benefit of share issuance costs within the Netherlands in 2022.
Previous report
Deferred tax expense recorded in 2021 results from the consumption of net operating tax losses by our U.S. entity as well as deferred tax expense resulting from the release of valuation allowance for the tax benefit of share issuance costs within the Netherlands.
Previous report
On March 1, 2021, we entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at-the-market (“ATM”) offering program, under which we, from time to time in our sole discretion, could offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million. In the year ended December 31, 2021, we received net proceeds of $29.6 million from the issuance of 921,730 ordinary shares under the Sales Agreement that took place during March and April of that year. We paid SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement. We did not issue any ordinary shares under the Sales Agreement for the 12-month periods ended December 31, 2023 and December 31, 2022.
Previous report
On January 29, 2021, we drew down $35 million under a facility agreement with Hercules. We drew down a further $30 million under our 2021 Restated Facility with Hercules in December 2021.
Previous report
Net cash generated from operating activities was $288.0 million for the year ended December 31, 2021, and consisted of net income of $329.6 million adjusted for non-cash items, including depreciation and amortization expense of $7.3 million, share-based compensation expense of $25.6 million, a change in fair value of contingent consideration of $6.8 million, unrealized foreign exchange gains of $31.3 million, a change in deferred taxes of $3.2 million and other non-cash items, net, of $2.8 million. Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $50.3 million, which includes $55.0 million recognized as a contract asset related to probable CSL Behring milestone payments. Additionally, these changes also related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $4.0 million primarily related to an increase in various prepaids, including those related to clinical trials, partially offset by decrease in receivables as a result of collection of the BMS milestone that was recorded as of December 31, 2020 and collection of the CSL Behring receivables recorded as of December 31, 2020 for expenses for which we had a right of reimbursement and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $8.5 million primarily related to an increase in various accruals for goods received from and services provided by vendors and an increase in personnel accruals. Net income primarily consisted of $462.4 million license revenue recognized on Closing and $55.0 million license revenue related to CSL Behring’s global regulatory submissions for etranacogene dezaparvovec that occurred in March and April 2022 and were considered probable on December 31, 2021.
Previous report
In the years ended December 31, 2023 and 2022, we invested $366.4 million and $163.1 million, respectively, of our cash on hand into euro and dollar denominated government bonds. We made no such investments in 2021. In the year ended December 31, 2023 we received $167.9 million in proceeds from the maturing of investments securities.
Previous report
In 2023, we invested $3.4 million in the build out of our Amsterdam site compared to $11.9 million in 2022 and $12.4 million in 2021. Our investments in 2023 and 2022 related to investments into equipment while in 2021 investments primarily related to the construction of additional laboratories to support the expansion of our research and development activities as well as the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.
Showing the first 40 of 47 passages. Open the source sections for the rest.
Reworded paragraphs (45)
Previous wording
We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devasting 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, ALS, refractory MTLE and Fabry disease. Our internally developed HEMGENIX®, a gene therapy for the treatment of hemophilia B, was approved for commercialization by the FDA in November 2022 and by the EMA in February 2023. In June 2020, we entered into the Commercialization and License Agreement to license HEMGENIX® to CSL Behring, which is responsible for its commercialization. We are manufacturing HEMGENIX® for CSL Behring and are entitled to specific milestone payments and royalties on net sales under the Commercialization and License Agreement. In May 2023, we entered into the Royalty Purchase Agreement for the sale of a portion of the royalty rights due to us from CSL Behring under the Commercialization and License Agreement. On October 5, 2023, we announced a Reorganization (see definition below) of our research and technology programs.
Current wording
We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1, refractory mesial temporal lobe epilepsy (“mTLE”), and Fabry disease.
Previous wording
On May 12, 2023, we entered into the Royalty Financing Agreement with the Purchaser. Under the terms of the Royalty Financing Agreement, we received an upfront payment of $375.0 million in exchange for the Purchaser’s rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us. We are also eligible to receive an additional $25.0 million milestone payment under the Royalty Financing Agreement if 2024 net sales of HEMGENIX® exceed a pre-specified threshold. The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) and 1.85 times the $25.0 million milestone payment (if paid) prior to the First Hard Cap Date or, if such cap is not met, up to 2.25 times the upfront and milestone payment (if paid) through December 31, 2038. If 2024 net sales do not exceed a pre-specified threshold, we will be obligated to pay $25.0 million to the Purchaser but only to the extent that we achieve a future sales milestone under the CSL Behring Agreement. If such milestone payment is not due from CSL Behring, we are not obligated to pay any amounts to the Purchaser.
Current wording
In May 2023, we entered into a royalty purchase agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. (the “Purchaser”). Under the terms of the Royalty Financing Agreement, we received an upfront payment of $375.0 million in exchange for the Purchaser’s rights to the lowest royalty tier on CSL Behring’s worldwide net sales of HEMGENIX® for certain current and future royalties due to us. We will be obligated to pay $25.0 million of the first worldwide sales milestone payment from CSL Behring, if received, to the Purchaser. The Purchaser will receive 1.85 times the upfront payment (or $693.8 million) until June 30, 2032 (“First Hard Cap Date”) if such thresholds are met or, if such cap is not met by June 30, 2032, up to 2.25 times of the upfront payment through December 31, 2038 (“Second Hard Cap Date”). If, on or prior to the defined dates for each cap amount, the total amount of royalty payments received by the Purchaser equals or exceeds the cap amount applicable to such date, the Royalty Financing Agreement will automatically terminate and all rights to the HEMGENIX® royalty payments will revert back to us. We have no obligation to repay any amounts received from the Purchaser in the event that the applicable cap amount is not reached during the term of the Royalty Financing Agreement.
Previous wording
Upon entering into the Royalty Financing Agreement, we and Hercules amended the 2021 Restated Facility on May 12, 2023. The 2023 Amended Facility extends the maturity date and interest-only period from December 1, 2025 to January 5, 2027.
Current wording
Upon entering into the Royalty Financing Agreement in May 2023, we and Hercules amended our existing term loan facility. Through the May 2023 amendment we extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027 (the “Maturity Date”).
Previous wording
As of December 31, 2023, we had $617.9 million in cash and cash equivalents and investment securities (December 31, 2022: $392.8 million cash and cash equivalents and investment securities). We had a net loss of $308.5 million in 2023, a net loss of $126.8 million in 2022 and net income of $329.6 million in 2021. As of December 31, 2023, we had an accumulated deficit of $890.4 million (December 31, 2022: $581.9 million).
Current wording
As of December 31, 2024, we had $367.5 million in cash and cash equivalents and investment securities (December 31, 2023: $617.9 million cash and cash equivalents and investment securities). We had a net loss of $239.6 million in 2024 and a net loss of $308.5 million and $126.8 million in 2023 and 2022, respectively. As of December 31, 2024, we had an accumulated deficit of $1,130.0 million (December 31, 2023: $890.4 million).
Previous wording
We consider the following to be our critical accounting estimate:
Current wording
We consider the following to be our critical accounting estimates in the year ended December 31, 2024:
Previous wording
On the Acquisition Date of uniQure France SAS we recorded contingent consideration related to amounts potentially payable to uniQure France SAS’s former shareholders. The amounts payable in accordance with the SPA are contingent upon realization of certain milestones associated with the TLE research program. Contingent consideration was measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.
Current wording
In July 2021 (“Acquisition Date”), we acquired uniQure France SAS (“Acquisition Date”) and we recorded contingent consideration related to amounts potentially payable to uniQure France SAS’s former shareholders. The amounts payable in accordance with the share and purchase agreement (“SPA”) are contingent upon realization of certain milestones associated with AMT-260. Contingent consideration was measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.
Previous wording
The fair value of the contingent consideration liability as of December 31, 2023 was $43.0 million and as of December 31, 2022 was $35.3 million. The increase was primarily driven by the increase in the probability of TLE advancing into clinical development to 100% following the clearance of the IND application for AMT-260 in August 2023. If as of December 31, 2023 we had assumed TLE was certain (i.e., 100% probability) to advance into late-stage development and commercialization, then the fair value of the contingent consideration liability would have increased from $43.0 million to $75.9 million. If as of December 31, 2023 we had assumed that we would discontinue development of the TLE program, then we could have released the contingent consideration liability to income.
Current wording
The fair value of the contingent consideration liability as of December 31, 2024 was EUR 10.5 million ($10.9 million) and as of December 31, 2023 was EUR 39.0 million ($43.0 million). The reduction was primarily driven by a EUR 26.8 million ($28.2 million) milestone payment we made in December 2024 related to the first patient dosed in the AMT-260 clinical trial. If as of December 31, 2024, we had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 33.7 million ($35.0 million). If as of December 31, 2024 the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.
Previous wording
Revenue recognition related to CSL Behring milestones
Current wording
Revenue recognition related to CSL Behring variable milestone payments
Previous wording
In March and April 2022, we collected the $55.0 million of variable milestone payments related to the submissions of a BLA and MAA which we had recorded as license revenue in the year ended December 31, 2021. During the year ended December 31, 2022, we recorded $100.0 million of variable milestone revenue related to a first sale of HEMGENIX™ and collected this payment in July 2023. We no longer consider this a critical accounting estimate because we currently do not expect to recognize any regulatory milestone payments within the next two years. The recognition of license revenue related to sales milestone payments and royalties does not require significant judgement as we recognize these as sales of HEMGENIX™ occur.
Current wording
We sold the exclusive global rights HEMGENIX™ to CSL Behring in 2021. The consideration received included variable milestone payments which we recorded as revenue once probable. We recorded $100.0 million of variable milestone revenue related to a first sale of HEMGENIX™ as license revenue in the year ended December 31, 2022 following the November 2022 BLA approval. We collected the payment in July 2023. We do not consider this to be a critical accounting estimate for the years ended December 31, 2024 and 2023 because we currently do not expect to recognize any regulatory milestone payments within the next 12 months.
Previous wording
Effective on Closing of the CSL Behring Agreement we sold the exclusive global rights to the Product (“License Sale”). We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of Product have been earned. We recognized $2.8 million, $100.0 million and $517.4 million of license revenue for the years ended December 31, 2023, 2022 and 2021, respectively. We recognized $2.8 million of license revenue in 2023 related to royalty payments owed on HEMGENIX® sales, when earned. We recognized $100.0 million of license revenue in 2022 related to a milestone payment following the first sale of HEMGENIX® in the U.S. in 2023, which we considered probable as of December 31, 2022. We recognized $517.4 million license revenue in 2021 related to the fixed upfront payment of $450.0 million and an additional payment of $12.4 million we received after the Closing as well as a total of $55.0 million of payments related to milestone payments owed on submission of the MAA in March 2022 and the BLA in April 2022, which we had considered probable as of December 31, 2021.
Current wording
We sold the exclusive global rights to HEMGENIX® to CSL Behring in 2021 (“License Sale”). We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of HEMGENIX® have been earned. We recognized $10.1 million, $2.8 million and $100.0 million of license revenue for the years ended December 31, 2024, 2023 and 2022, respectively. We recognized $10.1 million and $2.8 million of license revenue in 2024 and 2023 related to royalty payments owed on HEMGENIX® sales, when earned. We recognized $100.0 million of license revenue in 2022 related to a milestone payment owed on the first sale of HEMGENIX® in the U.S. in 2023.
Previous wording
We expense contract fulfillment costs associated with license revenue we receive from CSL Behring, which are recognized as costs of license revenues. These expenses primarily consist of payments we owe to our licensors in relation to license payments we receive from CSL Behring. We incurred $0.1 million, $1.3 million and $25.0 million of such cost in the years ended December 31, 2023, 2022 and 2021, respectively.
Current wording
We treat license payments to St. Jude as contract fulfillment costs associated with license revenue we receive from CSL Behring and recognize these as costs of license revenues. We incurred $1.3 million, $0.1 million and $1.3 million of such cost in the years ended December 31, 2024, 2023 and 2022, respectively.
Previous wording
We recognize collaboration revenues associated with 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. We recognized $2.3 million, $3.0 million and $2.4 million of collaboration revenue for the years ended December 31, 2023, 2022 and 2021, respectively. The decrease in collaboration revenue in 2023 of $0.7 million compared to 2022 was primarily related to a reduction in services requested by CSL Behring following the submissions of the BLA and MAA for HEMGENIX®. The increase in collaboration revenue in 2022 of $0.6 million compared to 2021 was primarily related to revenues related to full-time-employee (“FTE”) recharges of $3.0 million recognized from the CSL Behring Agreement as a result of additional development services that CSL Behring requested from us.
Current wording
We recognize collaboration revenues associated with services we provide to CSL Behring. Collaboration revenue is recognized when the performance obligations are satisfied. We recognized $10.9 million, $2.3 million and $3.0 million of collaboration revenue for the years ended December 31, 2024, 2023 and 2022, respectively. The increase in collaboration revenue in 2024 of $8.6 million compared to 2023 was primarily related to additional development and other services provided in relation to the transfer of manufacturing responsibility to CSL Behring. The decrease in collaboration revenue in 2023 of $0.7 million compared to 2022 was primarily related to a reduction in services requested by CSL Behring following the submissions of the BLA and MAA for HEMGENIX® in 2022.
Previous wording
We incurred $13.6 million and $2.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the years ended December 31, 2023 and 2022 respectively, compared to nil cost of contract manufacturing revenues in the year ended December 31, 2021. Costs of contract manufacturing revenues has increased in the year ended December 31, 2023 compared to the year ended December 31, 2022 due to the increase in sales of HEMGENIX® and a write-down of inventory that has a cost basis in excess of its expected net realizable value.
Current wording
We incurred $17.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the year ended December 31, 2024 and $13.6 million and $2.1 million of cost of contract manufacturing revenues, in the years ended December 31, 2023 and 2022 respectively. Costs of contract manufacturing revenues increased in the year ended December 31, 2024 compared to the year ended December 31, 2023 primarily due to expensing costs that could not be recovered from selling HEMGENIX® under the terms of our development and commercial supply agreement with CSL Behring. Costs of contract manufacturing revenues increased in the year ended December 31, 2023 compared to the year ended December 31, 2022 due to the increase in sales of HEMGENIX® and a write-down of inventory that had a cost basis in excess of its expected net realizable value.
Previous wording
Following the termination of the amended BMS CLA on February 22, 2023 we did not recognize any further revenue for services rendered in accordance with the BMS CLA.
Current wording
Following the termination of the collaboration, research and license agreements and the amended agreements with Bristol-Myers Squibb (“BMS”) in February 2023, we did not recognize any further revenue for services rendered. We recognized nil collaboration revenue for the years ended December 31, 2024 and 2023 and $1.8 million for the year ended December 31, 2022.
Previous wording
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:
Current wording
Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:
Previous wording
Research and development expenses for the year ended December 31, 2023 were $214.9 million, compared to $197.6 million and $143.5 million for the years ended December 31, 2022 and 2021, respectively. Other research and development expenses are separately classified in the table below. These are not allocated as they are deployed across multiple projects under development.
Current wording
Research and development expenses for the year ended December 31, 2024 were $143.8 million, compared to $214.9 million and $197.6 million for the years ended December 31, 2023 and 2022, respectively. Other research and development expenses are separately classified in the table below. These are not allocated as they are deployed across multiple projects under development.
Previous wording
We incurred $13.0 million, $19.8 million and $10.5 million expenses in the years ended December 31, 2023, 2022 and 2021 respectively. Our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II(b) clinical trials in the U.S. and in Europe. We enrolled 26 patients into our U.S. clinical trial between June 19, 2020 and March 21, 2022 and enrolled 13 patients into our European clinical trial between June 23, 2022 and June 21, 2023. The decrease of $6.8 million in external cost in the year ended December 31, 2023 compared to 2022 is a result of enrolling less patients into the clinical trial during 2023, following completion of U.S. enrollment in March 2022. The increase of $9.3 million in external cost in the year ended December 31, 2022 compared to 2021 is a result of increased enrolling activities in 2022, including cross-over patients from our U.S. trial and starting to enroll patients into our European trial in 2022, as well as follow-up activities related to patients enrolled in prior periods.
Current wording
In the years ended December 31, 2024, 2023 and 2022, we incurred $18.0 million, $13.0 million and $19.8 million expenses respectively. Our external costs for the development of AMT-130 were primarily related to the execution of our U.S. and European studies. The increase of $5.0 million in external cost in 2024 compared to 2023 is a result of enrolling patients into a third cohort between November 2023 and December 2024 as well as expenses we incurred associated with the RMAT application and in preparation of the Type B meeting with the FDA in November 2024. The decrease of $6.8 million in external cost in 2023 compared to 2022 is a result of the completion of U.S. patient enrollment in March 2022.
Previous wording
In the years ended December 31, 2023, December 31, 2022 and December 31, 2021, we incurred $13.0 million, $16.2 million and $0.9 million, respectively, expenses for the preclinical development of AMT-260, which we acquired on July 30, 2021. In August 2023, the FDA cleared our IND application, and we started incurring costs for the preparation of a Phase I clinical trial. The decrease in development costs in the year ended December 31, 2023 compared to 2022, results from completing the IND enabling studies, initiated during the year ended December 31, 2022, in the first half of 2023. The increase in development cost in the year ended December 31, 2022, compared to 2021, related to costs incurred in relation to the toxicology study we initiated during the year as well as the manufacturing of supplies for clinical development.
Current wording
In the years ended December 31, 2024, 2023 and 2022 we incurred $12.1 million, $13.0 million and $16.2 million expenses respectively. In August 2023, the FDA cleared our IND application, and we started incurring costs for the preparation of a Phase I clinical trial.
Previous wording
On January 31, 2023, we entered into a global licensing agreement with Apic Bio for AMT-162. We have incurred $10.0 million of expenses related to the acquisition. In addition, we have incurred $6.0 million of costs to initiate a Phase I/II clinical trial in 2024.
Current wording
In January 2023, we entered into a global licensing agreement with Apic Bio for AMT-162. In the years ended December 31, 2024 and 2023, we incurred $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial in which enrolled the first patient in October 2024. During the year ended December 31, 2023, we incurred expenses of $10.0 million related to the upfront consideration paid to Apic Bio for the global licensing rights to AMT-162.
Previous wording
In the years ended December 31, 2023, December 31, 2022 and December 31, 2021, we incurred $2.7 million, $2.9 million and $0.9 million expenses, respectively, primarily related to our preclinical activities. In November 2023, the FDA cleared the IND application, and we started incurring additional costs for Phase I/II clinical trial preparation. The decrease of $0.2 million in external costs in the year ended December 31, 2023 compared to 2022 is a result of completing the IND enabling studies by November 2023. The increase of $2.0 million external costs in the year ended December 31, 2022 compared to 2021 is a result of initiating IND-enabling toxicology studies in August 2022.
Current wording
In the years ended December 31, 2024, 2023 and 2022 we incurred $4.8 million, $2.7 million and $2.9 million expenses, respectively, related to our development of AMT-191. In November 2023, the FDA cleared the IND application, and we started incurring additional costs for Phase I/II clinical trial preparation. Costs incurred in the year ended December 31, 2022 primarily related to our preclinical activities.
Previous wording
In the years ended December 31, 2023, 2022 and 2021 we incurred $11.0 million, $7.2 million and $8.0 million, respectively, of costs related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Current wording
In the years ended December 31, 2024, 2023 and 2022 we incurred $3.9 million, $11.0 million and $7.2 million expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Previous wording
In the years ended December 31, 2023, December 31, 2022 and December 31, 2021, we incurred $1.4 million income, $2.5 million expenses and $8.7 million expenses, respectively which were primarily related to the Phase II development of the hemophilia B program. After the Closing of the CSL Behring Agreement in May 2021, CSL Behring was responsible for the clinical and regulatory activities and commercialization of the Product. We managed the existing trials on behalf of CSL Behring until such responsibilities were transitioned to CSL Behring in December 2022. Direct research and development expenses related to clinical development incurred in the year ended December 31, 2022 and 2021 are presented net of reimbursements due from CSL Behring. In the same periods, we also incurred costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec. These costs are also presented net of reimbursements due from CSL Behring.
Current wording
We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022. Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 and 2022 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.
Previous wording
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio. Our selling costs include employee expenses as well as professional fees related to the preparation of a commercial launch of HEMGENIX®and advisory fees related to obtaining the CSL Behring Agreement.
Current wording
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio. Our selling costs included employee expenses as well as advisory fees related to obtaining the commercialization and license agreement (“CSL Behring Agreement”) between us and CSL Behring LLC (“CSL Behring”).
Previous wording
Selling, general and administrative expenses for the year ended December 31, 2023 were $74.6 million, compared to $55.1 million and $56.3 million for the years ended December 31, 2022 and 2021, respectively.
Current wording
Selling, general and administrative expenses for the year ended December 31, 2024 were $52.7 million, compared to $74.6 million and $55.1 million for the years ended December 31, 2023 and 2022, respectively.
Previous wording
In the year ended December 31, 2023, we recognized $0.8 million in other expense in relation to a reduction in the fair market value of our equity stake in VectorY B.V. following an October 2023 financing round. We recognized other income of $0.3 million and $3.0 million related to the equity stake for the years ended December 31, 2022 and 2021, respectively. We received the equity stake in VectorY B.V. in conjunction with a settlement agreement that the Company and VectorY B.V. entered into in April 2021.
Current wording
We recognized nil income related to our equity stake in VectorY B.V. in the year ended December 31, 2024. In 2023, we recognized $0.8 million in other expense related to a reduction in the fair market value of our equity stake in VectorY B.V. following an October 2023 financing round. We recognized other income of $0.3 million related to the equity stake in 2022.
Previous wording
In 2023, we recognized $5.0 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands compared to $5.6 million in 2022 and $5.3 million in 2021.
Current wording
In 2024, we recognized $5.6 million in income related to payments received from European authorities to subsidize our research and development efforts in the Netherlands and France compared to $5.3 million in 2023 and $6.1 million in 2022.
Previous wording
Other income for the years ended December 31, 2023, 2022, and 2021 also includes income from the subleasing of a portion of our Amsterdam facility. We present expenses related to such income as other expense.
Current wording
Other income for the years ended December 31, 2024, 2023, and 2022 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility. We present expenses related to such income as other expense.
Previous wording
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $19.6 million interest income in 2023, $0.6 million in 2022 and $0.2 million in 2021. Interest income increased by $19.0 million in 2023 compared to 2022 as a result of earning interest income from investing the proceeds of our May 2023 Royalty Financing Agreement as well as the $100.0 million milestone payment collected from CSL Behring in July 2023 into debt securities. In addition, our interest income in 2023 and 2022 compared to 2021 benefited from an increase in interest rates on cash on hand. Our interest income increased in 2022 by $0.4 million compared to 2021 primarily due to the interest income earned on investment securities.
Current wording
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized interest income of $21.4 million in 2024, $19.6 million in 2023 and $0.6 million in 2022. Our interest income increased in 2024 by $1.9 million compared to 2023 primarily due to the interest income earned on investment securities and cash on hand. Interest income increased by $19.0 million in 2023, compared to 2022, as a result of receiving $370.1 million of net proceeds from the May 2023 royalty financing agreement and a $100.0 million milestone payment from CSL Behring in July 2023.
Previous wording
We recognized $41.6 million interest expense in 2023, $11.7 million in 2022 and $7.5 million in 2021. Interest expense increased by $29.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt as well as the recognition of $26.9 million of accrued interest expense related to the Royalty Financing Agreement that we entered into in May 2023. Our interest expense in 2022 primarily increased by $4.2 million compared to 2021 due to an increase in market interest rates in 2022.
Current wording
We recognized interest expense of $12.9 million in 2024, $14.6 million in 2023 and $11.7 million in 2022 related to the Hercules debt facility. Interest expense decreased by $1.7 million in 2024, compared to 2023, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates. Interest expense increased by $2.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt.
Previous wording
We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S. dollars. We recognize foreign exchange results related to changes in these foreign currencies. In 2023, we recognized a net foreign currency loss of $1.7 million related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, compared to a net gain of $23.2 million in 2022 and a net gain of $29.7 million in 2021.
Current wording
We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S. dollars. We recognize foreign exchange results related to changes in these foreign currencies. In 2024, we recognized a net foreign currency loss of $10.5 million related to our borrowings from Hercules, the May 2023 royalty financing agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group. We recognized a net loss of $1.7 million in 2023 and a net gain of $23.2 million in 2022.
Previous wording
In 2023, we recognized nil within Other non-operating gains /(losses). In 2022, we recognized a $2.8 million net gain within Other non-operating gains / (losses) related to a decrease in the fair value market value of a derivative financial liability related to the change of control payment (“CoC-payment”) compared to a net loss of $0.2 million in 2021. We recorded a net gain in 2022 as no change of control event had occurred as of the February 22, 2023 Termination Date of the amended BMS CLA. We had recorded a net loss of $0.2 million in 2021 for the increase in the fair market value of the derivative financial liability related to the CoC-payment.
Current wording
In 2024 and 2023, we recognized no Other non-operating gains. In 2022, we recognized a $2.8 million net gain related to a decrease in the fair value market value of a derivative financial liability
Previous wording
We recognized $1.9 million of deferred tax expense in 2023, compared to $1.5 million of deferred tax income in 2022 and $3.2 million of deferred tax expense in 2021. In 2023, deferred tax expense recorded in the U.S., related to the consumption of net operating losses, more than offset the deferred tax income recorded as a result of the buildup of net operating losses by the French entity.
Current wording
We recognized $2.3 million of deferred tax expense in 2024 compared to $1.9 million of deferred tax expense in 2023 and $1.5 million of deferred tax income in 2022. In 2024 and 2023 deferred tax expense recorded in the U.S., related to the consumption of net operating losses.
Previous wording
As of December 31, 2023, we had cash and cash equivalents, restricted cash and investment securities of $621.1 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 and following the Reorganization, we believe that our cash and cash equivalents and investment securities will fund our operations into second quarter 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 December 31, 2024, we had cash and cash equivalents, restricted cash and investment securities of $368.9 million. Until such time, if ever, as we can generate substantial cash flows from successfully commercializing our proprietary product candidates, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution, and licensing arrangements. Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe that our cash and cash equivalents and investment securities will fund our operations through the second half of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. The amount and timing of our actual expenditures may vary significantly depending on numerous factors, including the timing of a potential BLA submission, approval and commercialization of AMT-130. We expect that we will require additional funding if we decide to advance any of our other clinical product candidates into late-stage clinical development. Our material cash requirements include the following contractual and other obligations:
Previous wording
As of December 31, 2023, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $100.0 million. Future interest payments and financing fees associated with the loan total $47.6 million, with $13.4 million payable within 12 months. We are contractually required to repay the $100.0 million in full in January 2027.
Current wording
As of December 31, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc. (“Hercules”) for an aggregate principal amount of $50.0 million. In July 2024, in connection with the Closing of the Lexington Transaction, we repaid $50.0 million of the $100.0 million of principal outstanding as well as $3.1 million in end-of-term fees. Future interest payments and financing fees associated with the loan total $16.0 million, with $8.6 million payable within 12 months. We are contractually required to repay the $50.0 million principal outstanding in full in January 2027.
Previous wording
We entered into lease arrangements for facilities, including corporate, manufacturing and office space. As of December 31, 2023, we had fixed lease payment obligations of $53.1 million, with $8.3 million payable within 12 months.
Current wording
We entered into lease arrangements for facilities, including corporate, laboratory and office space. As of December 31, 2024, we had fixed lease payment obligations of $23.0 million, with $4.1 million payable within 12 months. Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen. This reduced our fixed lease payment obligations by $20.7 million. We continue to guarantee such payments until the end of the lease term in May 2029.
Previous 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 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 December 31, 2023, our remaining commitment amounts include a EUR 30.0 million ($33.1 million) milestone payment due upon treating the first patient in a Phase I/II clinical trial for AMT-260 and EUR 160.0 million ($176.6 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 milestone will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of December 31, 2023, of $1.10/€1.00. As of December 31, 2023, we expect these obligations will become payable between the first half of 2024 and 2031. 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 September 2023, we made a payment of EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS following the FDA’s clearance of the IND application for AMT-260. In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS following the dosing of the first patient in Phase I/II clinical trial for AMT-260. As of December 31, 2024, our remaining commitment amounts include a EUR 160.0 million ($166.2 million) in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the U.S. and European Union. The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestone will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of December 31, 2024, of $1.04/€1.00. As of December 31, 2024, we expect these obligations will become payable between 2029 and 2033. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.
Previous wording
We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement. We continue to incur losses in the current period. We recorded a net loss of $308.5 million for the year ended December 31, 2023, and net loss of $126.8 million in 2022, and a net income of $329.6 million in 2021. As of December 31, 2023, we had an accumulated deficit of $890.4 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 Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement. We continued to incur losses in the current period. We recorded a net loss of $239.6 million for the year ended December 31, 2024, and net loss of $308.5 million in 2023, and a net loss of $126.8 million in 2022. As of December 31, 2024, we had an accumulated deficit of $1,130.0 million.
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. In May 2021, we received a $462.4 million cash payment due from CSL Behring. We have collected $55.0 million related to CSL Behring’s global regulatory submissions for etranacogene dezaparvovec in March and April 2022, and $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 the Royalty Financing Transaction) 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 have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL and other milestones collected from CSL, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX®.
Previous wording
We are required to repay the entire principal balance of $100.0 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 2023 Amended Facility, we owe a back-end fee of $4.9 million on December 1, 2025 and a back-end fee of $1.3 million on January 5, 2027.
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 Closing of the Lexington Transaction in July 2024 we amended the debt facility again and repaid $50.0 million of the principal balance outstanding. We are required to repay the remaining principal balance of $50.0 million on the Maturity Date. The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum. We owe a back-end fee of $2.4 million on December 1, 2025 and a back-end fee of $0.6 million on the Maturity Date.
Previous wording
We are subject to certain covenants under the 2023 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 2023 Amended Facility may limit our ability to obtain debt financing. The 2023 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.
Showing the first 40 of 45 pairs. Open the source sections for the rest.
Rearranged current paragraphs (2)
Current report — wording carried from elsewhere
Net Cash used in operating activities
Current report — wording carried from elsewhere
In December 2024, following the dosing of the first patient in Phase I/II clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS based on contractually defined milestones. EUR 26.8 million ($28.2 million) of this payment related to a contingent consideration of which EUR 8.2 million ($8.6 million) was classified as cash flows from financing activities and EUR 18.7 million ($19.6 million) was classified as a net cash flow used in operating activities.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
We and Hercules amended the 2021 Restated Facility on May 12, 2023. The 2023 Amended Facility extends the maturity date and interest-only period from December 1, 2025 to January 5, 2027.
Previous report — wording carried elsewhere
Net cash generated from financing activities