What changed in this filing

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Item 7. Management’s Discussion and Analysis

2024-02-28 compared with 2023-02-27 · 286 added, 288 removed, 259 unchanged (69% of the section changed)

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Words such as “may,” “expect,” “anticipate,” “estimate,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements.

Our actual results and the timing of certain events may differ materially from the results discussed, projected, anticipated, or indicated in any forward-looking statements.

We caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this MD&A.

In addition, even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this MD&A, they may not be predictive of results or developments in future periods.

We caution readers not to place undue reliance on any forward-looking statements made by us, which speak only as of the date they are made.

We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

We are a leader in the field of gene therapy and seek to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results.

We are advancing a pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease and ALS, as well as preclinical product candidates including candidates for the treatment of rTLE and Fabry disease.

In November 2022 and February 2023, our internally-developed HEMGENIX Ô , a gene therapy for the treatment of hemophilia B, was approved for commercialization by the FDA and the EMA, respectively.

In June 2020, we agreed to license HEMGENIX Ô to CSL Behring, which is now responsible for commercialization of HEMGENIX Ô .

We are manufacturing HEMGENIX Ô for CSL Behring and are entitled to specific milestone payments and royalties on net sales.

We believe our validated technology platform and manufacturing capabilities provide us distinct competitive advantages, including the potential to reduce development risk, cost, and time to market.

We produce our AAV-based gene therapies in our own facilities with a proprietary, commercial-scale, cGMP-compliant, manufacturing process.

We believe our Lexington, Massachusetts-based facility is one of the world’s most versatile gene therapy manufacturing facilities.

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.

Business Developments

Below is a summary of our recent significant business developments:

CSL Behring collaboration

In June 2020 we entered into the CSL Behring Agreement pursuant to which CSL Behring received exclusive global rights to the Product.

The transaction became fully effective in May 2021.

In March and April 2022, CSL Behring submitted marketing applications for HEMGENIX Ô in the U.S.

In March and April 2022, we received the $55.0 million owed to us by CSL Behring related to submission of these marketing applications.

In November 2022, the FDA approved the marketing application for the U.S.

and in February 2023 the EMA approved the marketing application for the EU.

We are eligible to receive a $100.0 million payment from CSL Behring following the first sale of the Product in the U.S.

We are also eligible to receive a $75.0 million payment from CSL Behring following the first sale of the Product in any of five major European countries, namely France, Germany, Italy, Spain, and the United Kingdom, provided the first sale occurs prior to July 2, 2023.

We recorded the $100.0 million payment associated with the first sale in the U.S.

as license revenue in the year ended December 31, 2022 as we expect this event to occur in 2023.

We did not record license revenue related to a $75.0 million payment in the year ended December 31, 2022 as the accomplishment of this milestone prior to July 2, 2023 is contingent on factors outside of our control.

Concurrently with the execution of the CSL Behring Agreement, we and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we will supply the Product to CSL Behring at an agreed-upon price commensurate with the stand-alone selling price (“SSP”) until such time that these capabilities are transferred to CSL Behring or its designated contract manufacturing organization.

We completed the validation of the current manufacturing process and in July 2022, following a comprehensive multi-day facility inspection, the EMA notified us that GMP certification can be issued for our Lexington, Massachusetts-based manufacturing site to produce commercial supply of HEMGENIX Ô .

In August 2022, we completed the FDA pre-license inspection of the Lexington facility.

On September 6, 2022 CSL Behring notified us of its intent to transfer manufacturing technology related to the Product in the coming years to a third-party contract manufacturer designated by CSL Behring.

CSL Behring also informed us of its intent to retain us as a source for manufacturing after the completion of the technology transfer.

In August 2022, the GMP certification for the Amsterdam facility was amended to include release testing of the Product in the European Union following inspection by the IGJ.

W e recorded $1.7 million in revenue related to manufacturing the Product in the year ended December 31, 2022.

Huntington’s disease program (AMT-130)

On March 21, 2022, we announced that we have completed the enrollment of all 26 patients in the first two cohorts of our Phase I/II clinical trial of AMT-130 taking place in the U.S.

The low-dose cohort includes 10 patients, of which six patients received treatment with AMT-130 and four patients received imitation surgery.

The higher-dose cohort includes 16 patients, of which 10 patients received treatment with AMT-130 and six patients received imitation surgery.

In July 2022 we crossed over one of these six patients and treated the patient with the lower dose of AMT-130.

On June 23, 2022, we announced encouraging safety and biomarker data from the 10 patients enrolled in the low-dose U.S.

AMT-130 was generally well-tolerated with no serious adverse events related to AMT-130 reported in the treated patients.

In the four treated patients with evaluable data, mean levels of CSF mHTT declined at all timepoints compared to baseline and decreased by 53.8% at 12 months of follow-up.

In the six treated patients, measurements of CSF NfL initially increased as expected following the AMT-130 surgical procedure and declined thereafter, nearing baseline at 12 months of follow-up.

In August 2022, we announced a voluntary postponement of AMT-130 higher-dose procedures due to SUSARs reported in three of the 14 patients that were treated with the higher dose of AMT-130.

In October 2022, after completing a comprehensive safety investigation, the DSMB recommended resuming treatment at the higher dose of AMT-130 for the remaining five European patients and any patients in the U.S.

trial eligible to cross over from the control arm to the treatment.

All three patients have experienced full resolution of the reported SUSARs.

Acquisition of Corlieve Therapeutics

On June 21, 2021, we entered into a Sale and Purchase Agreement (“SPA”) to acquire all outstanding ordinary shares of Corlieve SAS (“Corlieve”), a privately held French gene therapy company (the “Corlieve Transaction”).

Upon the closing of the Corlieve Transaction on July 30, 2021 (the “Acquisition Date”), we acquired 97.7% of the outstanding ordinary shares of Corlieve in return for EUR 44.9 million ($53.3 million as of the Acquisition Date).

We paid EUR 1.8 million ($1.9 million) to acquire the remaining shares of Corlieve in 2022.

We financed the Corlieve Transaction from cash on hand.

In addition to the payments to acquire 100% of the outstanding ordinary shares, Corlieve’s former and remaining shareholders are eligible to receive up to EUR 40.0 million (or $42.8 million as of December 31, 2022) upon the achievement of development milestones through Phase I/II and EUR 160.0 million (or $171.3 million as of December 31, 2022) upon the achievement of milestones associated with Phase III development and obtaining approval to commercialize AMT-260 in the U.S.

W e expect these obligations will become payable between 2023 and 2031.

If and when due, w e may elect to pay up to 25% of such milestone payments through the issuance of our ordinary shares.

Termination of Bristol-Myers Squibb Agreement

In May 2015 we and BMS entered the BMS CLA.

The initial four-year research term under the collaboration terminated in May 2019.

On December 1, 2020, we and BMS amended the BMS CLA.

As a result of the Amended BMS CLA, we recognized the remaining balance of license revenue of $27.8 million not previously recognized during the year ended December 31, 2020.

The Amended BMS CLA did not extend the initial research term.

For a period of one-year from the effective date of the Amended BMS CLA, BMS was able to replace up to two of the four active collaboration targets with two new targets in the field of cardiovascular disease.

BMS did not replace any of the active collaboration targets.

On December 17, 2020, BMS designated one of the four collaboration targets as a candidate to advance into Investigational New Drug-enabling studies (“IND-enabling studies”) entitling us to receive a $4.4 million research milestone payment.

We recorded the $4.4 million as license revenue in the year ended December 31, 2020.

On November 21, 2022 we received the Termination Notice whereby the Amended BMS CLA terminated on February 21, 2023.

The Amended BMS CLA terminated two warrants that, when in effect, provided BMS the right to increase its ownership in the Company up to 19.9% upon the exercise of both warrants, with the exercise of such warrants being conditioned on the designation of a seventh, and a tenth Collaboration Target, respectively.

In the Amended BMS CLA, we and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to the earlier of (i) December 1, 2026 and (ii) BMS’ delivery of a target cessation notice for all four Collaboration Targets, we (or our third party acquirer) would pay to BMS a one-time, non-refundable, non-creditable cash payment of $70.0 million, provided that (x) if $70.0 million is greater than five percent of the net proceeds (as contractually defined) from such change of control transaction, the payment would be an amount equal to five percent of such net proceeds, and (y) if $70.0 million is less than one percent of such net proceeds, the change of control payment would be an amount equal to one percent of such net proceeds.

We had not consummated any change of control transaction as of the Termination Notice and as of December 31, 2022, assessed the probability as of the Termination Date and released the liability related to the change of control payment to other income during the year ended December 31, 2022.

The Termination Notice did not change any of the provisions of the Investor Agreement with BMS that we entered into in 2015, but various provisions of the Investor Agreement have been terminated.

We have granted BMS certain registration rights that allow BMS to require us to register our securities beneficially held by BMS under the Securities Act.

BMS may make up to two such demands for us to register the shares, subject to customary limitations.

In addition, independent of their demand registration rights, upon the occurrence of certain events, we must also provide BMS the opportunity to include their ordinary shares in any registration statement that we effect.

We also continue to grant BMS certain information rights under the Investor Agreement, although these requirements may be satisfied by our public filings required by U.S.

securities laws.

BMS also continues to be subject to a lock up pursuant to the Investor Agreement for as long as BMS holds more than 4.9% of our ordinary shares (as of December 31, 2022 BMS held 5.08%).

Without our prior consent, BMS may not sell or dispose any of its current ordinary shares.

The Investor Agreement also continues to require BMS to vote all of our ordinary shares it beneficially holds in favor of all items on the agenda for the relevant general meeting of shareholders of our company as proposed on behalf of our company, unless, in the context of a change of control or similar transaction, BMS has itself made an offer to our company or our board in connection with the transaction that is the subject of the vote, in which case it is free to vote its shares at its discretion.

This voting provision will terminate upon the later of the date on which BMS no longer beneficially owns at least 4.9% of our outstanding ordinary shares or the closing of a transaction that provides BMS exclusive and absolute discretion to vote our shares it beneficially holds.

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.

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.

Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the subsequent high-dose cohort.

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.

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.

Amyotrophic lateral sclerosis program (AMT-162)

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.

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.

Temporal lobe epilepsy program (AMT-260)

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.

Reorganization

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.

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.

As a result of the significant reduction in research activities, Ricardo Dolmetsch, Ph.D., departed as Chief Scientific Officer, effective October 4, 2023.

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.

Royalty Financing Agreement

On May 12, 2023, we entered into the Royalty Financing Agreement with the Purchaser.

Under the terms of the Royalty Financing Agreement, we received an upfront payment of $375.0 million in exchange for 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.

We retained the rights to all other royalties, as well as contractual milestones totaling up to $1.3 billion, under the terms of the CSL Behring Agreement.

Hercules amendment

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.

Investment in debt securities

In December 2022, we invested $100.0 million and EUR 80.0 million (or total of $183.9 million as of investment date) of our cash on hand into short-term and long-term euro and dollar denominated sovereign bonds.

The bonds have remaining maturities ranging from two to 14 months.

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.

We made no such investments in 2021 and 2020.

Covid pandemic

The coronavirus disease (“Covid”) caused by the severe acute respiratory syndrome coronavirus 2 (“Sars-CoV 2 virus”) was characterized as a pandemic by the WHO on March 11, 2020.

Since then, various variants of the Sars-CoV 2 virus causing Covid have been identified.

The broader implications of Covid, including the implications from the various variants, on our results of

operations and overall financial performance remain uncertain.

We have experienced and continue to experience increased lead times in the delivery of equipment and disposables that we use to manufacture materials for our various programs.

Currently, these have not materially impacted our development timelines and we continue to adapt to the current environment to minimize the effect to our business.

However, we may experience more pronounced disruptions in our operations in the future.

Russian-Ukrainian war

Our business is not directly impacted by the war as we do not operate in either Russia or the Ukraine.

However, the war might potentially amplify the disruptive impact of the Covid pandemic.

CSL Behring collaboration

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.

Financial Highlights

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Total revenues

Cost of revenues

Cost of license revenues

Cost of contract manufacturing revenues

Research and development expenses

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As of December 31, 2023, we had $617.9 million in cash and cash equivalents and investment securities (December 31, 2022:

$556.3 million cash and cash equivalents).

We had a net loss of $126.8 million in 2022, and net income of $329.6 million in 2021 and a net loss of $125.0 million in 2020.

$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).

We anticipate that our expenses will increase for the foreseeable future and will include costs related to:

● advancing AMT-130 for our Huntington’s disease gene therapy program into phase III clinical study;

● advancing our gene therapy programs rTLE, SOD1-ALS and Fabry disease into Phase I/II clinical studies;

● potentially acquiring or in-licensing rights to new therapeutic targets, product candidates and technologies;

● making potential future milestone payments related to the acquisition of Corlieve, if any;

● advancing preclinical research and development for gene therapy product candidates targeting other diseases;

● continuing to invest in expanding, developing and optimizing our manufacturing capabilities and other enabling technologies, such as next-generation viral vectors, promoters and re-dosing.

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.

See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.

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We also discuss our critical accounting estimates with the Audit Committee of our Board of Directors.

We consider the following to be our critical accounting estimates:

● Recognition of revenue including the CSL Behring milestones in relation to the CSL Behring Agreement;

● Contingent consideration recorded in relation to the Corlieve business combination;

● Valuation allowance related to Dutch and U.S.

deferred tax assets.

Revenue recognition related to CSL Behring milestones

On the Signing Date we entered into the CSL Behring Agreement.

The transaction became effective on Closing.

As of Closing, we identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“License Sale”);

(ii) Generate information to support the regulatory approval of the current and next generation manufacturing process of Product and to provide any such information generated to CSL Behring (“Manufacturing Development”).

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.

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™ in the U.S.

as we consider this to be probable following the November 2022 FDA approval of HEMGENIX™.

Despite the approval of the MAA for HEMGENIX™ by the EMA in February 2023, we did not record the $75.0 million variable milestone payment related to a first sale of HEMGENIX™ in the one of five major European countries, namely France, Germany, Italy, Spain, and the United Kingdom, as license revenue in the year ended December 31, 2022 as the payment is contingent on the milestone being achieved prior to July 2, 2023, which is contingent on factors outside our control.

We consider the following to be our critical accounting estimate:

● Contingent consideration recorded in relation to the uniQure France SAS business combination;

Contingent consideration

On the Acquisition Date of Corlieve we recorded contingent consideration related to amounts potentially payable to Corlieve’s former shareholders.

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.

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An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability.

● As of December 31, 2021, we had estimated that AMT-260 would advance into the clinical development by early 2024, and as of December 31, 2022, we revised that estimate to late 2023.

● We need to develop an estimate of when a milestone is expected to be achieved.

An achievement of a milestone at a later than currently expected date reduces the fair value of the contingent consideration liability whereas an achievement at an earlier than currently expected date increases the fair value of the contingent consideration liability.

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We developed this estimate using data from an external study regarding the average likelihood of advancing into clinical development at a certain stage or preclinical development.

For the year ended December 31, 2021, we had increased the probability to 55.0% following the designation of a lead candidate by us in late October 2021.

During the year ended December 31, 2022 we increased the probability to 66.0% following the commencement of toxicology studies for the TLE program.

For the year ended December 31, 2022, we had increased the probability to 66.0% following the commencement of toxicology studies for the TLE program.

During the year ended December 31, 2023 we increased the probability to 100.0% following the clearance of the IND application for AMT-260 in August 2023.

The increase in probabilities resulted in a $14.2 million expense in the year ended December 31, 2023 and a $4.4 million expense in the year ended December 31, 2022.

This also resulted in an increase of the probability that AMT-260 may advance to late-stage development and commercialization.

The fair value of the contingent consideration liability as of December 31, 2023 was $43.0 million and as of December 31, 2022 was $35.3 million.

The increase was primarily driven by an increase in the probability of the TLE program advancing into clinical development.

If as of December 31, 2022 we had assumed TLE was certain (i.e.

100% probability) to advance into clinical development, then the fair value of the contingent consideration liability would have increased from $35.3 million to $48.8 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.

Valuation allowance related to Dutch and U.S.

deferred tax assets

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:

● Valuation allowance related to Dutch deferred tax assets;

● Revenue recognition related to CSL Behring milestones

Valuation allowance related to Dutch deferred tax assets

We are subject to corporate taxes in the Netherlands.

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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.

We are also subject to corporate taxes in the U.S..

While our operations in the U.S.

had initially been incurring net operating tax losses, our subsidiary in the U.S.

generated taxable income in the past few years starting with 2018.

Based on the design of our worldwide operations, we determined as of December 31, 2020 that we expect to continue to generate taxable income in the U.S.

during the foreseeable future and therefore determined that it had become more likely than not that our U.S.

deferred tax assets will be realized.

Accordingly, we recorded $16.4 million of deferred tax income in the year ended December 31, 2020 from releasing the full valuation allowance against our net deferred tax assets in the U.S..

We generated taxable income in the U.S.

during the years ended December 31, 2021 and December 31, 2022 and therefore continue to expect that it is more likely than not that our U.S.

deferred tax assets will be realized.

We would be required to record deferred tax expense to recognize a valuation allowance on a portion of or possibly even our full U.S.

deferred tax asset of $21.1 million as of December 31, 2022, if we would expect not to meet the above threshold.

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.

Revenue recognition related to CSL Behring milestones

On June 24, 2020 (“ Signing Date”), we entered into the CSL Behring Agreement.

The transaction became effective on Closing.

As of Closing, we identified two material performance obligations related to the CSL Behring Agreement:

(i) Sale of the exclusive global rights to the Product (“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.

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.

Recently Adopted Accounting Pronouncements

Government Assistance

In November 2018, the Financial Accounting Standards Board (“FASB”) issued ASU 2021-10, Government Assistance (Topic 832) which discussed the requirements for disclosures, to be applied prospectively or retrospectively, related to transactions with a government.

ASU 2021-10 is effective for fiscal years beginning after December 15, 2021.

The new disclosure requirements required disclosures around 1) information about the nature of the transactions and the related accounting policy used to account for the transactions, 2) the line items on the balance sheet and income statement that are affected by the transactions, and the amounts applicable to each financial statement line item, and 3) significant terms and conditions of the transactions, including commitments and contingencies.

The Company currently includes information on government grants and the adoption of ASU 2021-10 on January 1, 2022 has not had a material impact on the Company’s consolidated financial statements.

Results of Operations

The following table presents a comparison of the twelve months ended December 31, 2022, 2021 and 2020.

The following table presents a comparison of the years ended December 31, 2023, 2022 and 2021.

Year ended December 31,

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(Loss) / income from operations

Non-operating items, net

(Loss) / income before income tax benefit / (expense)

Income tax benefit / (expense)

Non-operating (expense) / income, net

(Loss) / income before income tax benefit

Income tax (expense) / benefit

Net (loss) / income

Revenues and cost of revenues

Our revenue and associated costs for the years ended December 31, 2022, 2021 and 2020 was as follows:

Our revenues and associated costs for the years ended December 31, 2023, 2022 and 2021 were as follows:

Year ended December 31,

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License revenues

Collaboration revenues

Contract manufacturing revenues

Collaboration revenues

Total revenues

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Cost of contract manufacturing revenues

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 $100.0 million and $517.4 million of license revenue for the years ended December 31, 2022 and 2021.

We recognized $100.0 million of license revenue in 2022 related to a milestone payment we expect to collect following the first sale of HEMGENIX™ in the U.S.

We recognized $517.4 million license revenue in 2021 related to the fixed upfront payment of $450.0 million and the $12.4 million we received in relation to the Additional Covenants 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 the February 25, 2022 filing of the 2021 financial statements.

We expense contract fulfillment costs associated with license revenue recognized as costs of license contract revenues.

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.

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 $1.3 million and $25.0 million of such cost in the years ended December 31, 2022 and 2021, respectively.

We did not incur such costs in the years ended December 31, 2020.

We recognize collaboration revenues associated with services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring between Closing and December 2022, when CSL Behring fully assumed these activities as well as in relation to additional development services that CSL Behring requests.

These services are provided by our employees.

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.

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 $3.0 million, $2.4 million, and nil of collaboration revenue for the years ended December 31, 2022, 2021 and 2020, respectively.

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.

We recognize contract manufacturing revenue related to contract manufacturing HEMGENIX TM for CSL Behring.

Contract manufacturing revenue is realized when earned upon sales of HEMGENIX TM to CSL Behring.

We recognized $1.7 million contract manufacturing revenues in the year ended December 31, 2022.

We did not recognize such revenues in 2021 and 2020, as we started contract manufacturing activities to supply CSL Behring with launch supplies of HEMGENIX TM following their submission of a BLA and MAA in the spring of 2022.

We incurred $2.1 million of cost of contract manufacturing revenues related to the manufacture of the Product in the year ended December 31, 2022, compared to nil cost of contract manufacturing revenues in the years ended December 31, 2021 and 2022, respectively.

We recognized license revenues associated with the amortization of the non-refundable upfront payment and target designation fees we received from BMS in 2015 until December 1, 2020.

We evaluated our outstanding performance obligation following the amendment of the BMS CLA on December 1, 2020 and determined that our remaining performance obligation is immaterial.

We updated our measure of progress accordingly and amortized the remaining balance of unrecognized revenue of $27.8 million as of December 1, 2020 as license revenue from a related party.

On December 17, 2020 BMS designated one of the four Collaboration Targets as a candidate to advance into IND-enabling studies under the amended BMS CLA entitling us to receive a $4.4 million research milestone payment, which we recorded as license revenue in the year ended December 31, 2020.

We recognized no license revenues in the years ended December 31, 2022 and December 31, 2021.

We recognized collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that were reimbursable by BMS under the BMS CLA and the 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 $2.3 million, $3.0 million and $2.4 million of collaboration revenue for the years ended December 31, 2023, 2022 and 2021, respectively.

Following the termination of the amended BMS CLA on February 22, 2023 we do not expect to recognize any further revenue for services rendered in accordance with the BMS CLA.

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.

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.

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.

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.

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.

Res e arch and development expenses

We expense R&D as incurred.

We expense research and development (“R&D”) expenses as incurred.

R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development.

Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:

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● costs associated with research activities for enabling technology platforms, such as next-generation vectors, promoters and re-administration of gene therapies;

● costs associated with the rendering of collaboration services as well as the continued development of the Product;

● costs associated with the rendering of collaboration services;

● payments related to identifiable intangible assets without an alternative future use;

● payments to our licensors for milestones that have been achieved related to our product candidates, including approval of the BLA;

● payments to our licensors for milestones that have been achieved related to our product candidates, including approval of the MAA and BLA for HEMGENIX ® ;

● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies;

● changes in the fair value of liabilities recorded in relation to our acquisition of Corlieve.

● changes in the fair value of liabilities recorded in relation to our acquisition of uniQure France SAS.

Our research and development expenses primarily consist of costs incurred for the research and development of our product candidates, which include:

● AMT-130 (Huntington’s disease).

We have incurred costs related to preclinical and nonclinical studies of AMT-130 and have been incurring costs related to our Phase I/II trial since February 2019.

We have incurred costs related to preclinical and nonclinical studies of AMT-130 and have been incurring costs related to our U.S.

Phase I/II clinical trial since February 2019.

Since 2021, we have also incurred costs related to our Phase Ib/II clinical trial in Europe;

● AMT-260 (Temporal lobe epilepsy) .

We have incurred costs related the preclinical development of temporal lobe epilepsy, which we acquired from Corlieve on July 30, 2021;

We have incurred costs related to the preclinical development of AMT-260.

We acquired this program on July 30, 2021;

● AMT-191 (Fabry disease) .

We have incurred costs related to the preclinical development of Fabry disease;

We have incurred costs related to the preclinical development of AMT-191 for the treatment of Fabry disease;

● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ) We have incurred costs related to the acquisition in addition to costs incurred to initiate a Phase I/II clinical trial;

● Etranacogene dezaparvovec (hemophilia B) .

We have incurred costs related to the research, development, and production of etranacogene dezaparvovec for the treatment of hemophilia B.

During 2020 and up to the Closing of the CSL Behring Agreement we incurred costs related to the preparation of a BLA and MAA and for commercialization of the Product.

Up to the Closing of the CSL Behring Agreement, we incurred costs related to the preparation of a BLA and MAA and for commercialization of the Product.

We also incurred costs for manufacturing development.

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We incurred significant research and development costs related to manufacturing and other enabling technologies that are applicable across all our programs.

Our research and development 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.

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 judgment due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

● the scope, rate of progress and expense of our research and development activities;

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● the timing of regulatory approvals.

● our ability to agree to ongoing development budgets with collaborators who share the costs of our development programs.

A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidate.

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(in thousands)

Huntington's disease (AMT-130)

Amyotrophic lateral sclerosis (AMT-162)

Temporal lobe epilepsy (AMT-260)

Huntington's disease (AMT-130)

Fabry disease (AMT-191)

Hemophilia B (AMT-060/061)

Etranacogene dezaparvovec (AMT-060/061)

Programs in preclinical development and platform related expenses

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Facility expenses

Share-based compensation expenses

Other expenses

Share-based compensation expense

Fair value changes related to contingent consideration

Other expenses

Impairment related to the Reorganization

Total other research and development expenses

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Direct research and development expenses

Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

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.

Temporal lobe epilepsy (AMT-260)

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.

Huntington disease (AMT-130)

We incurred $19.8 million, $10.5 million and $6.9 million in the years ended December 2022, 2021 and 2020 respectively.

In the year ended December 31, 2022, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the U.S.

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.

In the years ended December 31, 2021 and December 31, 2020 our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the U.S.

and in the year ended December 31, 2021, costs were incurred for the preparation of the Phase I/IIb clinical trial in Europe .

Temporal lobe epilepsy (AMT-260)

In years ended December 31, 2022, December 31, 2021 and December 31, 2020, we incurred $16.2 million, $0.9 million and nil, respectively, for the preclinical development of temporal lobe epilepsy, which we acquired from Corlieve on July 30, 2021.

The increase in development cost in the year ended December 31, 2022 related to cost incurred in relation to a toxicology study as well as manufacturing supplies for clinical development.

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.

Fabry disease (AMT-191)

In the years ended December 31, 2022 December 31, 2021 and December 31, 2020, we incurred $2.9 million, $0.9 million and $0.7 million, respectively, primarily related to our preclinical activities for the treatment of Fabry disease (AMT-191).

Hemophilia B (AMT-060/061)

In the years ended December 31, 2022, December 31, 2021 and December 31, 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial and Manufacturing Development.

During 2020 and up to the Closing of the CSL Behring Agreement in May 2021, we also incurred costs related to the preparation of the global regulatory submissions and to prepare for commercialization of the Product.

After the Closing, CSL Behring is responsible for the clinical and regulatory activities and commercialization of the Product.

We managed the existing trials on behalf of CSL Behring until December 2022, at which point the trials began to be managed by CSL Behring.

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.

Etranacogene dezaparvovec (AMT-060/061)

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.

Our Phase IIb dose-confirmation study was initiated in January 2018 and dosing occurred in July and August 2018.

Patients were dosed as part of our Phase I/II clinical trial of AMT-060 in 2015 and 2016.

These costs are presented net of reimbursements due from CSL Behring.

These costs are also presented net of reimbursements due from CSL Behring.

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.

Preclinical programs & platform development

In the year ended December 31, 2022, we incurred $7.2 million of costs primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

In the year ended December 31, 2021, we incurred $8.0 million of costs related to related to our preclinical activities for product candidates including SCA3 (AMT-150) as well as various other research programs and technology innovation projects compared to $5.8 million in 2020.

Costs for the year ended December 31, 2020 also include costs for Hemophilia A (AMT-180), which was deprioritized in June 2020.

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.

Other research & development expenses

● We incurred $76.7 million in employee and contractor expenses in the year ended December 31, 2023 compared to $64.9 million in 2022 and $55.7 million in 2021.

Our cost increased in 2022 by $9.2 million compared to 2021 primarily as a result of an increase in personnel and contractor related expenses to support our growth.

Costs increased by $14.0 million in 2021 compared to 2020 as a result of the recruitment of personnel to support the preclinical and clinical trial development of our product candidates;

The increase in 2023 of $11.8 million, compared to 2022, primarily related to the hiring of new personnel and contractors to support our clinical-stage programs and manufacturing operations as well as incurring $2.2 million in severance costs related to the Reorganization with no such cost incurred in 2022.

Our cost increased in 2022 by $9.2 million compared to 2021 primarily as a result of an increase in personnel and contractor related expenses to support the preclinical and clinical trial development of our product candidates;

● We incurred $29.4 million in operating expenses and depreciation expenses related to our rented facilities in the year ended December 31, 2023 compared to $23.6 million in 2022 and $18.8 million in 2021.

The increase in 2022 compared to 2021 of $4.8 million primarily related to additional sites in Lexington and increased depreciation expense related to the expansion of the Amsterdam facility in prior year.

The increase in 2021 compared to 2020 of $1.4 million primarily related to expansion of the Amsterdam facility;

Our costs increased by $5.9 million in 2023 compared to 2022 as a result of incurring additional operating and depreciation expenses in both our Lexington and Amsterdam facilities.

The increase in 2022 compared to 2021 of $4.8 million primarily related to operating expenses and depreciation expense incurred from additional sites in Lexington and increased depreciation expense related to the expansion of the Amsterdam facility in 2021;

● We incurred $13.2 million in disposables costs in the year ended December 31, 2023 compared to $17.8 million in the year ended December 31, 2022 and $14.7 million in the year ended December 31, 2021.

The increases are primarily related to the expansion of our activities to support the development of our product candidates;

The decrease in 2023 related to the reduction in activities in the second half of 2023 due to our Reorganization.

The increase in 2022 related to the expansion of our activities to support the development of our product candidates;

● We incurred $16.9 million in share-based compensation expenses in the year ended December 31, 2023 compared to $18.4 million in 2022 and $12.8 million in 2021.

The decrease in 2023 of $1.5 million, compared to 2022, is primarily due to forfeitures as a result of the Reorganization and other severance, and a decrease in the fair value of awards granted.

This was partially offset by recognizing compensation cost related to performance share units granted in 2021, for which achievement of related performance conditions was deemed probable during the year ended December 31, 2023.

The increase in 2022 compared to 2021 of $5.6 million was primarily driven by the increase in awards granted, including those to newly recruited personnel as well as an increase in expense related to performance share units that were deemed probable;

The increase in 2021 compared to 2020 of $0.8 million was driven primarily by grants to newly recruited personnel offset by share-based compensation expenses recorded in relation to the termination of one of our executives in 2020;

● We incurred $15.9 million of expenses for the year ended December 31, 2023 related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $7.1 million and $6.7 million for the same periods in 2022 and 2021.

The increase in 2023 was primarily due to an increase in the probability of making future milestone payments following the dosing of the first patient in Phase I/II clinical trial of AMT-260 after receiving IND acceptance in August 2023;

● We incurred $1.4 million of costs related to the impairment of the Lexington, MA facility right-of-use asset and related leasehold improvements for the year ended December 31, 2023 compared to nil in prior periods;

● We incurred $6.8 million in other expenses in the year ended December 31, 2023 compared to $17.2 million in 2022 and $5.8 million in 2021.

The increase in 2022 compared to 2021 of $11.4 million is primarily due to $7.0 million of contractual payments we owed to licensors upon FDA approval of HEMGENIX™ and $1.1 million owed to a licensor for a valid patent claim granted within the EU.

The decrease in 2021 compared to 2020 of $0.4 million is a combination of not incurring any expenses related to license payments without an alternative future use like in 2020 ($3.4 million) offset by various increases, including increases in professional fees as a result of expanding the organization and to support the cGMP validation of our Lexington facility;

● We incurred $7.1 million of expenses for the year ended December 31, 2022 related to an increase in the fair value of contingent consideration associated with the Corlieve Transaction, compared to $6.7 million and nil for the same periods in 2021 and 2020.

The decrease in costs in 2023 of $10.4 million, compared to 2022, is due to a decrease in contractual license expenses and a reduction in consultant-related expenses.

For the year ended December 31, 2023 we incurred $3.1 million in contractual license expenses upon the EMA approval of HEMGENIX® in February 2023 while for the year ended December 31, 2022, we incurred $7.0 million of contractual license expense upon FDA approval of HEMGENIX ® and $1.1 million contractual license expense for a valid patent claim granted within the EU.

The increase in 2022 compared to 2021 is primarily related to the contractual license expenses incurred in 2022.

Selling, general and administrative expenses

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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 etranacogene dezaparvovec and advisory fees related to obtaining the CSL Behring Agreement.

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.

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.

● We incurred $24.8 million in personnel and contractor expenses in 2023 compared to $21.1 million in 2022 and $16.0 million in 2021.

The increase in 2022 of $5.0 million, compared to 2021, and the increase in 2021 compared to 2020 of $2.4 million was primarily driven by an increase in personnel and contractor related expenses to support our growth;

The increase in 2023 of $3.7 million, compared to 2022, and the increase in 2022 of $5.1 million, compared to 2021 was primarily related to the hiring of personnel and contractors to support our business operations.

In 2023, we also incurred $0.4 million in severance expense related to the Reorganization;

● We incurred $17.4 million of share-based compensation expenses in 2023 compared to $15.5 million in 2022 and $12.8 million in 2021.

The increase in 2023 compared to 2022 of $1.8 million was primarily related to an increase in awards granted and from recognizing compensation cost for performance share units granted in December 2021 that were deemed probable in the year ended December 31, 2023 offset by a decrease in expense related to a decrease in the fair value of awards granted.

The increase in 2022 compared to 2021 of $2.7 million was primarily related to the increase in awards granted, including those to newly recruited personnel as well as an increase in expense related to performance share units that were deemed probable;

The increase in 2021 compared to 2020 of $3.0 million was also primarily driven by the increase in awards granted including those to newly recruited personnel;

● We incurred $11.7 million in professional fees in 2023 compared to $7.1 million in 2022 and $9.4 million in 2021.

We regularly incur accounting, audit and legal fees associated with operating as a public company.

Additionally, in the years ended December 31, 2021 and December 31, 2020, we incurred professional fees in relation to our licensing transaction with CSL Behring and our acquisition of Corlieve;

● We incurred $1.0 million, $5.1 million and nil in financial advisory fees in relation to our licensing transaction with CSL Behring in the years ended December 31, 2022, December 31, 2021 and December 31, 2020.

These fees are calculated as a percentage of license revenue recognized under the CSL Behring Agreement.

In the year ended December 31, 2023, we incurred additional costs in professional fees related to our global licensing agreement with Apic Bio, entering into the Royalty Purchase Agreement, and other corporate initiatives.

Additionally, in the year ended December 31, 2021 we incurred professional fees in relation to our licensing transaction with CSL Behring and our acquisition of uniQure France SAS;

● We incurred $3.8 million, $1.0 million and $5.1 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the years ended December 31, 2023, December 31, 2022 and December 31, 2021;

● We incurred $3.8 million in intellectual property fees including registration and professional fees in the year ended December 31, 2023 compared to $1.5 million in 2022 and $2.4 million in 2021.

The increase in 2023 compared to 2022 of $2.3 million is mainly related to an increase in professional fees.

The decrease in 2022 compared to 2021 of $0.9 million related to a decrease in intellectual property license fees and registration costs;

● We incurred $11.6 million in other operating expenses in 2023 compared to $7.9 million in 2022 and $8.2 million in 2021.

The increase in 2023 compared 2022 of $3.7 million was primarily a result of an increase in information technology expenses.

Other items, net

We recognized $0.3 million, $3.0 million and nil in other income in relation to the equity stake received in VectorY B.V.

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 for the years ended December 31, 2022, 2021 and 2020, respectively.

entered into in April 2021.

We recognized nil in other income of employee retention credit under the U.S.

CARES Act in the year ended December 31, 2022, compared to $2.6 million and nil such income for the same periods in 2021 and 2020, respectively.

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.

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.

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Interest expense

Foreign currency gains / (losses), net

Foreign currency (losses) / gains, net

Other non-operating gains / (losses)

Total non-operating income, net

Total non-operating (expense) / income, net

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.

Interest income decreased by $0.7 million in 2021 compared to 2020 due to a changes in market interest rates during 2021.

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.

Our interest expense in 2021 primarily increased by $3.6 million compared to 2020 due to the additional $35.0 million we drew down on our loan facility from Hercules in January 2021.

We hold monetary items and enter into transactions in foreign currencies, predominantly in euros and U.S.

We recognize foreign exchange results related to changes in these foreign currencies.

In 2022, we recognized a net foreign currency gain of $23.2 million related to our borrowings from Hercules 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 $29.7 million in 2021 and a net loss of $13.6 million in 2020.

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 derivative financial liability related to the change of control payment (“CoC-payment”) compared to a net loss of $0.2 million in 2021 and a net gain of $0.5 million in 2020.

We recorded the 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 in 2021 of $0.2 million in 2021 for the increase in the fair market value of the derivative financial liability related to the CoC-payment.

We recorded a net gain $0.5 million in 2020 within Other non-operating gains / (losses) related to the net impact of terminating the BMS warrants and recognizing a derivative financial liability for the CoC-payment.

We recognized $1.5 million of deferred tax income in 2022, compared to $3.2 million of deferred tax expense in 2021 and $16.4 million of deferred tax income in 2020.

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.

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.

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.

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 as well as deferred tax expense resulting from the release of valuation allowance for the tax benefit of share issuance costs within the Netherlands.

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.

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.

Deferred tax income recorded in 2020 results from the release of the valuation allowance recorded for our net deferred tax assets by our U.S.

Financial Position, Liquidity and Capital Resources

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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.

We believe that our cash and cash equivalents and investment securities will fund our operations into 2025 assuming the achievement of $100.0 million of first commercial sale milestone in the U.S.

and into the first half of 2025 if the $75.0 million first commercial sale milestone in any of the five contractually defined European countries would be achieved prior to July 2, 2023 under the CSL Behring Agreement.

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:

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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 December 2025.

We are contractually required to repay the $100.0 million in full in January 2027.

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.

Commitments related to Corlieve acquisition (nominal amounts)

In relation to the Corlieve Transaction, 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 SPA.

As of December 31, 2022, our commitment amounts include up to $42.8 million in potential milestone payments through Phase I/II development and $171.3 million in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the U.S.

Commitments related to acquisition of uniQure France SAS (nominal amounts)

In relation to our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractual milestones.

The commitments include payments related to post-acquisition services that we agreed to as part of the transaction.

In September 2023, we made a payment of EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS following the FDA’s clearance of the IND application for AMT-260.

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, 2022, we expect these obligations will become payable between 2023 and 2031.

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.

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We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our License Sale to CSL Behring.

We also owe payments to a financial advisor related to any payments we will collect under the CSL Behring Agreement.

We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.

The table below summarizes our consolidated cash flow data for the years ended December 31:

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Cash, cash equivalents and restricted cash at the beginning of the period

Net cash (used in) / generated from operating activities

Net cash used in operating activities

Net cash used in investing activities

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We continue to incur losses in the current period.

We recorded a net loss of $126.8 million for the year ended December 31, 2022, and net income of $329.6 million in 2021, and a net loss of $125.0 million in 2020.

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.

Sources of liquidity

From our first institutional venture capital financing in 2006 through May 2021, we funded our operations primarily through private and public placements of equity securities and convertible and other debt securities as well as payments from our collaboration partners.

In May 2021, we received a $462.4 million cash payment due from CSL Behring upon Closing.

During 2022 we collected the $55.0 million related to CSL Behring’s global regulatory submissions for etranacogene dezaparvovec in March and April 2022 and are eligible to receive additional milestone payments, as well as royalties on net sales, from CSL Behring.

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 may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.

We will pay 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.

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®.

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 did not issue in ordinary shares under the Sales Agreement for the 12 month period ended December 31, 2022.

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.

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.

We are subject to certain covenants under our 2021 Restated 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 2021 Restated Facility may limit our ability to obtain debt financing.

The 2021 Restated Facility permits us to issue up to $500.0 million of convertible debt as well as to enter into a transaction to sell the royalties under the CSL Behring agreement subject to certain conditions .

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.

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.

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.

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.

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Adjustments to reconcile net (loss) /income to net cash (used in) / generated from operating activities:

Depreciation and amortization

Share-based compensation expenses

Deferred tax (income) / expense

Change in fair value of contingent consideration and derivative financial instruments, net

Depreciation, amortization and impairment

Amortization of premium/discount on investment securities

Share-based compensation expense

Royalty financing agreement interest expense

Deferred tax expense / (income)

Change in fair value of contingent consideration and derivative financial instrument, net

Unrealized foreign exchange (gains) / losses, net

Change in deferred revenue

Other non-cash items, net

Other items, net

Changes in operating assets and liabilities:

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Accrued expenses, other liabilities, and operating leases

Contingent consideration milestone payment

Net cash (used in) / generated from operating activities

Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the premium/discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $26.9 million of interest expense related to the royalty financing agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.

Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $81.1 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $1.3 million.

There was a net decrease in contract assets of $100.0 million related to the collection of the $100.0 million milestone due from CSL Behring in July 2023 .

There was an increase in inventory balances of $6.7 million.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $10.8 million, primarily related to a decrease of $4.2 million in accounts payable and a decrease of $6.6 million related to various accruals.

Net cash used in operating activities also includes a payment for a contingent consideration milestone of $1.9 million.

Net cash used in operating activities was $145.1 million for the year ended December 31, 2022, and consisted of a net loss of $126.8 million adjusted for non-cash items, including depreciation and amortization expense of $8.5 million, share-based compensation expense of $34.2 million, changes in the fair value of contingent consideration and the derivative financial liability of $4.3 million, unrealized foreign exchange gains of $22.1 million and a change in deferred taxes of $1.5 million.

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The net increase related to $100.0 million recognized as a contract asset in the current period and collection of $55.0 million of the contract asset related to the CSL milestones of $55.0 million in March 2022 and April 2022.

There was an increase in inventories of $6.9 million related to the production of HEMGENIX TM under the CSL Behring Agreement.

There was an increase in inventories of $6.9 million related to the production of HEMGENIX ® under the CSL Behring Agreement.

These changes also relate to a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $12.6 million, primarily related to an increase in accounts payable.

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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.

Net cash used in operating activities was $134.8 million for the annual period ended December 31, 2020, and consisted of a net loss of $125.0 million adjusted for non-cash items, including depreciation and amortization expense of $10.6 million, share-based compensation expense of $21.8 million, fair value gain of derivative financial instruments of $0.5 million, unrealized foreign exchange loss of $14.7 million, a change in deferred tax income of $16.4 million and a decrease in unamortized deferred revenue of $33.6 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $6.5 million.

These changes primarily related to a net increase in accounts receivable and accrued income, prepaid expenses, and other current assets of $7.0 million and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $0.5 million.

Net cash used in investing activities

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Investment in investment securities

Proceeds from maturity of investment securities

Build out of Amsterdam site

Build out of Lexington site

Acquisition of Corlieve, net of cash acquired

Acquisition of licenses, patents, and other rights

Total investments

In 2022, we invested $163.1 million of our cash on hand into euro and dollar denominated government bonds.

We made no such investments in 2021 and 2020.

Acquisition of uniQure France SAS, net of cash acquired

Net cash used in investing activities

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.

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 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.

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.

In 2023, we invested $3.8 million in our facility in Lexington compared to $5.8 million in 2022 and $5.0 million in 2021.

Our investments in 2022 increased as a result of the two new Lexington sites, which commenced in 2022.

We paid EUR 1.8 million ($1.9 million) to acquire the remaining outstanding shares of Corlieve in February, July and September 2022.

We paid EUR 42.1 million ($49.9 million), net of EUR 2.8 million ($3.3 million) of cash acquired, during the year ended December 31, 2021 to acquire 97.7% of the outstanding ordinary shares of Corlieve on July 30, 2021.

Our investments in 2023 are a result of improvements made to the lease facility and investment in office and laboratory equipment.

We paid EUR 1.8 million ($1.9 million) to acquire the remaining outstanding shares of uniQure France SAS in February, July and September 2022.

Net cash generated from financing activities

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Cash flows from financing activities

Proceeds from Royalty Financing Agreement, net of debt issuance costs

Contingent consideration milestone payment

Proceeds from issuance of shares related to employee stock option and purchase plans

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Proceeds from issuance of ordinary shares, net of issuance costs

Repayment of debt assumed through Corlieve Transaction

Repayment of debt assumed through the acquisition of uniQure France SAS

Net cash generated from financing activities

In June 2023, we received $370.1 million net proceeds from the Royalty Financing Agreement.

In September 2023, following the FDA’s clearance of the IND application for AMT-260, we made a payment of $10.6 million to the former shareholders of uniQure France SAS based on contractually defined milestones.

$9.6 million of this payment related to a contingent consideration of which $7.6 million was classified as cash flows from financing activities and $1.9 million was classified as a net cash flow used in operating activities.

In 2023, we received $0.3 million from the exercise of options to purchase ordinary shares issued in accordance with our share incentive plans, compared to $1.4 million in 2022 and $2.8 million in 2021.

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No such proceeds were received in 2023 or 2022.

Upon the acquisition of Corlieve, Corlieve held loans with an outstanding amount equal to EUR 1.4 million ($1.6 million).

Upon the acquisition of uniQure France SAS, uniQure France SAS held loans with an outstanding amount equal to EUR 1.4 million ($1.6 million).

During the year ended December 31, 2021, the loans were repaid in their entirety.

Funding requirements

We believe that our cash and cash equivalents and investment securities will fund our operations into 2025 assuming the achievement of $100.0 million of first commercial sale milestone in the U.S.

Our future capital requirements will depend on many factors, including but not limited to:

● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement;

● earnout payments we might owe the former shareholders of Corlieve, which are subject to the achievement of specific development and regulatory milestones;

● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones;

● the scope, timing, results, and costs of our current and planned clinical trials, including those for AMT-130 in Huntington’s disease;

● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements;

● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;

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● the costs associated with maintaining quality compliance and optimizing our manufacturing processes, including the operating costs associated with our Lexington, Massachusetts manufacturing facility.

● the costs associated with increasing the scale and capacity of our manufacturing capabilities.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 7. Management’s Discussion and Analysis