What changed in this filing

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Item 2. Management’s Discussion and Analysis

2024-08-01 compared with 2024-05-07 · 302 added, 107 removed, 216 unchanged (65% of the section changed)

Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (“MD&A”) is intended to help the reader understand our results of operations and financial condition.

This MD&A is provided as a supplement to, and should be read in conjunction with, our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”) .

The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”).

Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S.

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We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, amyotrophic lateral sclerosis (“ALS”), refractory mesial temporal lobe epilepsy (“MTLE”) and Fabry disease.

Our internally developed HEMGENIX®, a gene therapy for the treatment of hemophilia B, has been approved for commercialization by the United States Food and Drug Administration (the “FDA”) in November 2022 and the European Medicines Agency (“EMA”) in February 2023.

The approval of HEMGENIX® follows more than a decade of research and clinical development, represents a major milestone in the field of gene therapy and ushers in a new treatment approach for patients living with hemophilia B.

We license HEMGENIX® to CSL Behring LLC (“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 of the product, a portion of which we sold to a royalty acquisition company in 2023 in exchange for up-front cash.

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

We produce our adeno-associated virus-based gene therapies in our own facilities with a proprietary, current good manufacturing practices (“GMP”) -compliant manufacturing process.

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

Recent Product Candidate Developments

Huntington’s disease program (AMT-130)

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a miRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

Business Developments

Huntington’s disease program (AMT-130) u pdated interim clinical data

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a micro ribonucleic acid (“miRNA”) specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

We are currently conducting a multi-center randomized, controlled, and blinded Phase I/II clinical trial for AMT-130 in the U.S.

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Patient enrollment is ongoing in this cohort with a focus on evaluating near-term safety and tolerability.

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 US study as well as the 13 patients enrolled in the European study.

We plan to initiate interactions with the FDA in the second quarter of 2024.

The goal of these interactions is to define the future clinical and regulatory pathway for AMT-130 and is expected to include discussions of the data from the ongoing Phase I/II trials and the potential to leverage robust natural history comparators and long-term clinical data from the Phase I/II studies.

On June 3, 2024, we announced that the FDA granted Regenerative Medicine Advanced Therapy (“RMAT”) designation for AMT-130 for the treatment of Huntington’s disease.

The FDA granted RMAT designation based on the potential of AMT-130 to address the major unmet medical need among patients with Huntington’s disease.

The designation follows the FDA’s review of interim Phase I/II clinical data for AMT-130 announced in December 2023 and is based on an analysis comparing these 24-month clinical data to a non-concurrent criteria-matched natural history cohort.

On July 9, 2024, we announced updated interim clinical data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing U.S.

and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease.

The data included a statistical analysis of clinical outcomes for the 21 treated patients at 24 months compared to an expanded, propensity-weighted external control (n=154) developed in collaboration with the Cure Huntington’s Disease Initiative (CHDI) using data from the TRACK-HD, TRACK-ON and PREDICT-HD natural history studies.

The external control includes patients that met the Phase I/II clinical trial eligibility criteria, and whose data contributions were statistically weighted using propensity scoring to closely match the baseline characteristics of patients treated with AMT-130.

Disease-related outcomes for these well-balanced cohorts were then compared after 24 months follow-up and are summarized below:

● A statistically significant, dose-dependent, slowing in disease progression measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) was observed through 24 months in patients receiving the high dose of AMT-130.

o At 24 months, the mean change in cUHDRS for patients receiving the high-dose of AMT-130 was -0.2 compared to -1.0 for patients in the propensity score-weighted external control, representing an 80% slowing of disease progression (p=0.007).

o At 24 months, the mean change in cUDHRS for patients receiving the low-dose of AMT-130 was -0.7 compared to -1.0 for patients in the propensity score-weighted external control, representing a 30% slowing of disease progression (p=0.21).

o cUHDRS has been demonstrated to be the most sensitive measurement of clinical progression in Huntington’s disease patients.

● Trends in measurements of motor and cognitive function showed near-baseline stability throughout the 24 months of follow-up in patients receiving the high dose of AMT-130.

● A statistically significant reduction of NfL in cerebrospinal fluid (CSF) was observed in patients treated with AMT-130.

o Patients treated with AMT-130 had a mean reduction in CSF NfL of 11% compared to baseline (p=0.02) at 24 months.

o Mean CSF NfL levels for both high and low doses were below baseline at 24 months.

o CSF NfL is a biomarker of neurodegeneration that has been shown to be associated with the clinical severity of Huntington’s disease.

An independent natural history study demonstrated a 26% increase in CSF NfL at 24 months in patients with early manifest Huntington’s disease (n=19).

Based on data observed to date, AMT-130 remains generally well-tolerated, with a manageable safety profile at both doses.

There were no new AMT-130-related serious adverse events reported.

We intend to hold a Type B, multi-disciplinary RMAT meeting with U.S.

Food and Drug Administration (FDA) to present these updated data and discuss potential clinical development pathways.

Sale of commercial manufacturing activities

On June 29, 2024, we and our affiliates, uniQure Inc.

and uniQure B.V., entered into various agreements with Genezen Holdings Inc.

and its affiliate Genezen MA, Inc.

(together “Genezen”) to sell our commercial manufacturing activities located in Lexington, MA (the “Lexington Facility”) (the “Lexington Transaction”).

The transaction closed on July 22, 2024 (the “Closing”).

As consideration, we received (i) shares of newly issued Series C preferred stock of Genezen Holdings Inc.

valued at $12.5 million, which are convertible into common stock and will accrue an 8.0% per annum cumulative dividend, and (ii) a convertible promissory note with a nominal amount of $12.5 million, bearing interest at 8.0% per annum and maturing 63 months following the date of issuance (the “Consideration”).

Our Chief Executive Officer, Matthew Kapusta will join the board of directors of Genezen in connection with the closing of the transaction.

We entered into an asset purchase agreement (“APA”) with Genezen.

Pursuant to the APA, Genezen agreed to acquire the manufacturing equipment and related manufacturing operations along with certain other assets associated with the Lexington Facility.

We, Genezen and the landlord of our Lexington Facility entered into an agreement for us to assign and Genezen to assume the existing lease agreement between us and the landlord.

Genezen extended offers of employment to a significant majority of our employees located at the Lexington Facility, with the remaining employees terminated effective August 30, 2024.

Concurrent with entering into the APA, we entered into a commercial supply agreement (“CSA”) with Genezen.

Pursuant to the terms of the CSA, we subcontracted the manufacturing of HEMGENIX® to Genezen.

The CSA includes a minimum term of three years and minimum purchase commitments of HEMGENIX® commercial supplies of $43.3 million over the first three-years, unless certain contractual provisions are triggered.

We will continue to sell these HEMGENIX® commercial supplies to CSL Behring.

Additionally, we entered into a development and other manufacturing services agreement (“DMSA”) with Genezen.

Pursuant to the terms of the DMSA, we have preferred customer status to receive manufacturing and development services to support our investigational gene therapy programs and other services related to the manufacture of HEMGENIX® under the CSA.

The DMSA has a minimum term of three years and requires us to purchase services for a total minimum of $14.0 million.

The CSA and DMSA became effective at Closing.

As a result of the Lexington Transaction, the Chief Operating Officer role was eliminated, and Pierre Caloz, will depart the company, continuing to lend his expertise in an advisory capacity.

In connection with Mr.

Caloz’s departure, the Board of Directors of the Company have appointed Amin Abujoub, Ph.D., our current Chief Quality Officer, to serve as the Company’s Chief Technical Operations Officer, effective as of July 22, 2024.

Hercules Loan Repayment and Amendment

On June 28, 2024 we and Hercules amended the 2023 Amended Facility (“2024 Amended Facility”).

On July 19, 2024, in connection with the closing of the Lexington Transaction, we prepaid $50.0 million of the total $100.0 million principal outstanding.

The remaining $50.0 million principal outstanding will need to be repaid on January 5, 2027 (the “Maturity Date”).

Results of the business review

On July 23, 2024, we announced the closing of the sale of its Lexington Facility to Genezen and on August 1, 2024, we announced an organizational restructuring.

These actions were the outcome of a recently completed, comprehensive review of our operations with the goals of conserving capital and streamlining the organization.

As part of these changes, we expect to eliminate approximately 300 positions or 65% of our workforce.

We estimate that we will incur costs in the range of $6.5 million to $7.5 million in connection with the restructuring, consisting primarily of cash expenditures related to employee severance costs.

These costs are subject to assumptions, including local law requirements, and actual expenses may differ materially from the estimates discussed above.

The restructuring is subject to the review by our Amsterdam-based works council, which is expected to be completed in the third quarter of 2024.

We expect the restructuring to be substantially completed by the end of 2024.

Recent Developments of other Product Candidates

Temporal lobe epilepsy program (AMT-260)

In August 2023, the FDA cleared the IND application for AMT-260, our gene therapy candidate for refractory MTLE.

AMT-260 comprises an AAV9 vector that locally delivers two engineered micro ribonucleic acids (“miRNAs”) designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE.

AMT-260 comprises an AAV9 vector that locally delivers two engineered miRNAs designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE.

We are initiating a Phase I/IIa clinical trial that will be conducted in the United States and consist of two parts.

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The multicenter, open-label clinical trial consists of two dose-escalating cohorts of up to three adult male patients each to assess safety, tolerability, and early signs of efficacy of AMT-191 in patients with Fabry disease.

Three patients will be dosed in the initial dose.

If no dose-limiting toxicology is identified, the dose will be escalated.

If dose-limiting toxicology occurs in one of the three initial patients, three additional patients will be enrolled at the same dose level.

If no additional patients in the cohort experience a dose-limiting toxicology, the dose will be escalated.

Assessments will be made at three- and six-months post-treatment.

Amyotrophic Lateral Sclerosis (AMT-162)

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Key components of our results of operations include the following:

Three months ended March 31,

Three months ended June 30,

Six months ended June 30,

(in thousands)

Total revenues

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Selling, general and administrative expenses

As of March 31, 2024 and December 31, 2023, we had cash and cash equivalents and investment securities of $555.7 million and $617.9 million, respectively.

We had a net loss of $65.6 million in the three months ended March 31, 2024, compared to net loss of $77.2 million for the same period in 2023.

As of March 31, 2024 and December 31, 2023, we had accumulated deficits of $956.0 million and $890.4 million, respectively.

As of June 30, 2024 and December 31, 2023, we had cash and cash equivalents and investment securities of $524.4 million and $617.9 million, respectively.

We had a net loss of $56.3 million and $121.9 million in the three and six months ended June 30, 2024, respectively, compared to a net loss of $68.5 million and $145.7 million for the same period in 2023.

As of June 30, 2024 and December 31, 2023, we had accumulated deficits of $1,012.3 million and $890.4 million, respectively.

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

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A summary of our critical accounting policies as well as a discussion of our critical accounting estimates are presented in our Annual Report .

There were no material changes to our critical accounting policies during the three months ended March 31, 2024 or reasonably possible changes of our critical accounting estimates as of March 31, 2024 that could have had a material impact on our results of operations for the three ended March 31, 2024.

There were no material changes to our critical accounting policies during the six months ended June 30, 2024 with the exception of determining the accounting treatment of the Lexington Transaction.

Lexington Transaction

We entered into various agreements with Genezen on June 29, 2024 to divest our Lexington Facility in return for the Consideration.

The Lexington Transaction closed on July 22, 2024.

We determined that as of June 30, 2024 the requirements of Topic 360 to classify certain assets as assets held for sale within our consolidated Balance Sheet were met.

Accordingly, we presented tangible assets with a carrying amount of $15.3 million, the right-of-use asset related to the Lexington Facility with a carrying amount of $11.9 million, goodwill with a carrying amount of $2.5 million, as well as inventory with a net realizable value of $8.0 million within “Assets held for sale” on our consolidated Balance Sheet.

In addition, we presented $17.9 million of leasehold liabilities within “Liabilities held for sale”.

If these criteria had not been met, then we would have continued to present these items respectively within “Property, plant and equipment, net of accumulated depreciation”, “Inventories, net”, “Operating lease right-of-use assets”, “Current portion of operating lease liabilities”, and “Operating lease liabilities, net of current portion” within the consolidated balance sheets.

We determined that the closing conditions were contingent on several conditions that were not met as of June 30, 2024.

Therefore, we did not account for the result of the divestment in the three- and six-month period ended June 30, 2024.

Cost of contract manufacturing

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Results of Operations

Comparison of the three months ended March 31, 2024 and 2023

The following table presents a comparison of our results of operations for the three months ended March 31, 2024 and 2023.

Three months ended March 31,

Comparison of the three months ended June 30, 2024 and 2023

The following table presents a comparison of our results of operations for the three months ended June 30, 2024 and 2023.

Three months ended June 30,

(in thousands)

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Other non-operating items, net

Net loss before income tax (expense) / benefit

Net loss before income tax expense

Income tax expense

Our revenues for the three months ended June 30, 2024 and 2023 were as follows:

Three months ended June 30,

(in thousands)

License revenues

Contract manufacturing revenues

Collaboration revenues

Total revenues

License revenues

We recognize license revenues from CSL Behring, related to royalty payments owed on HEMGENIX® sales, when earned.

For the three months ended June 30, 2024, we recognized $1.9 million of license revenues, compared to $0.8 million for the same period in 2023.

Contract manufacturing revenues

We recognize contract manufacturing revenues related to contract manufacturing HEMGENIX® for CSL Behring.

Contract manufacturing revenues is realized when earned upon sales of HEMGENIX® drug product to CSL Behring.

We recognized $2.1 million contract manufacturing revenues in the three months ended June 30, 2024 , compared to $1.3 million for the same period in 2023.

Collaboration revenues

We provide services to CSL Behring in accordance with the CSL Behring Agreement.

Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.

For the three months ended June 30, 2024 and 2023 we recognized $7.1 million and $0.3 million of collaboration revenue for CSL Behring, respectively.

The increase in collaboration revenue of $6.8 million in the three months ended June 30, 2024 compared to the same period in 2023 was primarily related to additional development and other services provided to CSL Behring in relation to the CSL Behring Agreement.

Cost of contract manufacturing

We incurred $7.2 million of cost of contract manufacturing related to the manufacture of HEMGENIX® in the three months ended June 30, 2024, compared to $1.4 million cost of contract manufacturing in the three months ended June 30, 2023.

The increase in cost of $5.9 million in 2024 is primarily related to expensing costs not expected to be recovered from selling HEMGENIX® under the terms of our development and commercial supply agreement with CSL Behring.

R&D expenses for the three months ended June 30, 2024 were $33.7 million, compared to $46.0 million for the same period in 2023.

Other research and development expenses are separately classified in the table below.

These other expenses are not allocated as they are deployed across multiple projects under development.

Three months ended June 30,

(in thousands)

Huntington's disease (AMT-130)

Temporal lobe epilepsy (AMT-260)

Amyotrophic lateral sclerosis (AMT-162)

Fabry disease (AMT-191)

Programs in preclinical development and platform related expenses

Etranacogene dezaparvovec (AMT-060/061)

Total direct research and development expenses

Employee and contractor-related expenses

Facility expenses

Share-based compensation expense

Other expenses

Severance costs

Fair value changes related to contingent consideration

Total other research and development expenses

Total research and development expenses

Direct research and development expenses

Huntington’s disease (AMT-130)

In the three months ended June 30, 2024 and June 30, 2023, we incurred costs of $2.9 million and $4.2 million respectively.

Our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trials in the U.S.

and in Europe.

We completed enrollment of the first two cohorts in the U.S.

in March 2022 and in Europe in June 2023.

This resulted in a $1.3 million reduction of costs in 2024 compared to the prior year as we typically incur higher costs during the first six months of patient follow-up compared to the period thereafter.

Temporal lobe epilepsy (AMT-260)

In the three months ended June 30, 2024 and June 30, 2023, we incurred costs of $2.0 million and $2.5 million respectively, for the development of AMT-260.

In August 2023, the FDA cleared our IND application, and we continue to incur costs for the preparation of a Phase I clinical trial.

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.

In the three months ended June 30, 2024, we incurred $1.6 million of costs to initiate a Phase I/II clinical trial in 2024.

In the three months ended June 30, 2023 we incurred $0.6 million of expenses related to our preclinical activities.

Fabry disease (AMT-191)

In the three months ended June 30, 2024 and June 30, 2023, we incurred costs of $1.5 million and $0.4 million, respectively, related to our development of AMT-191.

In November 2023, the FDA cleared the IND application, and we started incurring additional costs for the Phase I/II clinical trial preparation.

Preclinical programs and platform development

In the three months ended June 30, 2024 and June 30, 2023, we incurred $0.9 million and $2.3 million of costs, respectively, primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Etranacogene dezaparvovec (AMT-060/061)

We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the three months ended June 30, 2024 and June 30, 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

Other research & development expenses

● We incurred $13.0 million in personnel and contractor-related expenses in the three months ended June 30, 2024 , compared to $17.8 million for the same period in 2023.

The decrease was primarily related to the Lexington facility being primarily dedicated to contract manufacturing during the three months ended June 30, 2024 compared to the facility having been dedicated to research and development activities in the prior period;

● We incurred $6.0 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the three months ended June 30, 2024 , compared to $7.7 million in the same period in 2023;

● We incurred $2.9 million in share-based compensation expenses in the three months ended June 30, 2024 , compared to $4.7 million for the same period in 2023.

The decrease was primarily a result of the reduction in expense from the restructuring that occurred in October 2023 and a decrease in the fair value of the awards granted;

● We incurred $2.2 million of other expenses for the three months ended June 30, 2024 , compared to $1.8 million for the same period in 2023;

● We incurred $1.4 million in disposable costs in the three months ended June 30, 2024 , compared to $5.9 million for the same period in 2023.

The decrease was primarily a result of the reduction in expense from the restructuring that occurred in October 2023;

● We included $1.1 million of severance costs related to the Lexington Transaction in the three months ended June 30, 2024 with no such cost in the same period in 2023;

● We recognized a $1.8 million gain in the three months ended June 30, 2024 related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $0.2 million increase in fair value for the same period in 2023.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended June 30, 2024 were $15.8 million, compared to $21.2 million for the same period in 2023.

● We incurred $5.7 million in personnel and contractor-related expenses in the three months ended June 30, 2024 , compared to $6.4 million in the same period in 2023.

The decrease was primarily related to the reduction in personnel and contractors as a result of the restructuring that occurred in October 2023;

● We incurred $4.1 million in professional fees in the three months ended June 30, 2024 , compared to $2.5 million in the same period in 2023.

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

The increase from the prior period includes an increase in professional fees related to the Lexington Transaction;

● We incurred $3.3 million in share-based compensation expenses in the three months ended June 30, 2024 , compared to $4.0 million in the same period in 2023;

● We incurred $1.6 million in other expenses in the three months ended June 30 , 2024, compared to $3.2 million in the same period in 2023.

The $1.6 million decrease was primarily related to a decrease in intellectual property fees as well as a reduction of expenses for information technology;

● We incurred $0.3 million of severance costs related to the Lexington Transaction in the three months ended June 30, 2024 with no such cost in the same period in 2023;

● We incurred nil in financial advisory fees in relation to our licensing transaction with CSL Behring in the three months ended June 30, 2024, compared to $3.8 million in 2023.

Other items, net

We recognized $1.6 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the three months ended June 30, 2024, compared to $1.2 million for the same period in 2023.

Other items, net for the periods presented primarily related to income from the subleasing of our Amsterdam facility and expenses we incur in relation to the subleasing facility.

Other non-operating items, net

Our other non-operating items, net, for the three months ended June 30, 2024 and 2023 were as follows:

Three months ended June 30,

(in thousands)

Interest income

Interest expense

Foreign currency (losses) / gains, net

Total non-operating expense, net

We recognize interest income associated with our cash and cash equivalents and investment securities.

We recognized $5.8 million in interest income in the three months ended June 30, 2024, compared to $3.2 million in the same period in the prior year.

Our interest income increased by $2.6 million due to the interest income earned on investment securities as well as cash on hand during the three months ended June 30, 2024.

We recognized $16.2 million in interest expense for the three months ended June 30, 2024 and $6.8 million for the three months ended June 30, 2023.

Our interest expense in 2024 increased due to an increase of $9.3 million in non-cash interest expense related to the Royalty Financing Agreement that we entered into in May 2023.

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.

We recognized a net foreign currency loss, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $1.0 million during the three months ended June 30, 2024, compared to a net gain of $0.4 million during the same period in 2023.

Income tax benefit

We recognized $0.9 million of deferred tax expense in the three months ended June 30, 2024 , and $0.2 million of deferred tax expense for the same period in 2023.

Comparison of the six months ended June 30, 2024 and 2023

The following table presents a comparison of our results of operations for the six months ended June 30, 2024 and 2023:

Six months ended June 30,

(in thousands)

Total revenues

Operating expenses:

Cost of license revenues

Cost of contract manufacturing

Research and development expenses

Selling, general and administrative expenses

Total operating expenses

Other expense

Loss from operations

Non-operating expense, net

Loss before income tax benefit

Income tax (expense) / benefit

Our revenues for the three months ended March 31, 2024 and 2023 were as follows:

Three months ended March 31,

Our revenue for the six months ended June 30, 2024 and 2023 was as follows:

Six months ended June 30,

(in thousands)

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We recognize license revenues from CSL Behring, related to royalty payments owed on HEMGENIX® sales, when earned.

For the three months ended March 31, 2024, we recognized $1.2 million of license revenues (nil for the same period in 2023).

For the six months ended June 30, 2024, we recognized $3.1 million of license revenues, compared to $0.8 million for the same period in 2023.

Contract manufacturing revenues

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Contract manufacturing revenues is realized when earned upon sales of HEMGENIX® drug product to CSL Behring.

We recognized $4.0 million contract manufacturing revenues in the three months ended March 31, 2024 , compared to $4.9 million for the same period in 2023.

We recognized $6.1 million contract manufacturing revenues in the six months ended June 30, 2024 , compared to $6.2 million for the same period in 2023.

Collaboration revenues

We provide services to CSL Behring in accordance with the CSL Behring Agreement.

For the three months ended March 31, 2024 and 2023 we recognized $3.3 million and $0.4 million of collaboration revenue for CSL Behring, respectively.

Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.

For the six months ended June 30, 2024 and 2023 we recognized $10.4 million and $0.7 million of collaboration revenue for CSL Behring, respectively.

The increase in collaboration revenue of $9.7 million in the six months ended June 30, 2024 compared to the same period in 2023 was primarily related to additional development and other services provided to CSL Behring in relation to the CSL Behring Agreement.

Cost of contract manufacturing

We incurred $9.1 million of cost of contract manufacturing related to the manufacture of HEMGENIX® in the three months ended March 31, 2024, compared to $2.4 million cost of contract manufacturing in the three months ended March 31, 2023.

The increase in cost of $6.7 million in 2024 is primarily related to expensing costs previously capitalized as work in progress, including a write-down to lower net realizable value.

R&D expenses for the three months ended March 31, 2024 were $40.7 million, compared to $60.8 million for the same period in 2023.

We incurred $16.3 million of cost of contract manufacturing related to the manufacture of HEMGENIX® in the six months ended June 30, 2024, compared to $3.8 million cost of contract manufacturing in the six months ended June 30, 2023.

The increase in cost of $12.5 million in 2024 is primarily related to expensing costs not expected to be recovered from selling HEMGENIX® under the terms of our development and commercial supply agreement with CSL Behring.

R&D expenses for the six months ended June 30, 2024 were $74.3 million, compared to $106.8 million for the same period in 2023.

Other research and development expenses are separately classified in the table below.

These other expenses are not allocated as they are deployed across multiple projects under development.

Three months ended March 31,

Six months ended June 30,

(in thousands)

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Other expenses

Severance costs

Fair value changes related to contingent consideration

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Huntington’s disease (AMT-130)

In the three months ended March 31, 2024 and March 31, 2023, we incurred costs of $2.4 million and $3.7 million respectively.

In the six months ended June 30, 2024 and June 30, 2023, we incurred costs of $5.3 million and $8.0 million respectively.

Our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trials in the U.S.

and in Europe.

In the prior year, we had enrollment for the European clinical trial.

We completed enrollment of the first two cohorts in the U.S.

in March 2022 and in Europe in June 2023.

This resulted in a $2.7 million reduction of cost in 2024 compared to the prior year as we typically incur higher costs per patient during the first six months of enrollment compared to the follow-up period thereafter.

Temporal lobe epilepsy (AMT-260)

In the three months ended March 31, 2024 and March 31, 2023, we incurred costs of $2.3 million and $4.3 million respectively, for the development of AMT-260.

In August 2023, the FDA cleared our IND application, and we started incurring costs for the preparation of a Phase I clinical trial.

In the six months ended June 30, 2024 and June 30, 2023, we incurred costs of $4.3 million and $7.3 million respectively, for the development of AMT-260.

In August 2023, the FDA cleared our IND application, and we continue to incur costs for the preparation of a Phase I clinical trial.

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.

In the three months ended March 31, 2024, we incurred $2.0 million of costs to initiate a Phase I/II clinical trial in 2024.

In the three months ended March 31, 2023 we incurred $10.0 million of expenses related to the transaction.

In the six months ended June 30, 2024, we incurred $3.6 million of costs to initiate a Phase I/II clinical trial in 2024.

In the six months ended June 30, 2023 we incurred $10.6 million of expenses related to the transaction.

Fabry disease (AMT-191)

In the three months ended March 31, 2024 and March 31, 2023, we incurred costs of $1.3 million and $1.1 million, respectively, related to our development of AMT-191.

In the six months ended June 30, 2024 and June 30, 2023, we incurred costs of $2.8 million and $1.5 million, respectively, related to our development of AMT-191.

In November 2023, the FDA cleared the IND application, and we started incurring additional costs for the Phase I/II clinical trial preparation.

Preclinical programs & platform development

In the three months ended March 31, 2024 and March 31, 2023, we incurred $0.4 million and $1.9 million of costs, respectively, primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

In the six months ended June 30, 2024 and June 30, 2023, 2023, we incurred $1.3 million and $4.1 million of costs, respectively, primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

Etranacogene dezaparvovec (AMT-060/061)

We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the three months ended March 31, 2024 and March 31, 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the six months ended June 30, 2024 and June 30, 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition.

Other research & development expenses

● We incurred $16.9 million in personnel and contractor-related expenses in the three months ended March 31, 2024 , compared to $18.7 million for the same period in 2023.

The decrease was primarily related to the reduction in personnel and contractors as a result of the restructuring that occurred in October 2023;

● We incurred $7.1 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the three months ended March 31, 2024 , compared to $6.8 million in the same period in 2023;

● We incurred $3.4 million in share-based compensation expenses in the three months ended March 31, 2024 , compared to $4.3 million for the same period in 2023.

The decrease was primarily a result of the reduction in expense from the restructuring that occurred in October 2023;

● We incurred $2.7 million of other expenses for the three months ended March 31, 2024 , compared to $4.4 million for the same period in 2023;

● We incurred $1.9 million in disposable costs in the three months ended March 31, 2024 , compared to $3.9 million for the same period in 2023.

● We incurred $30.0 million in personnel and contractor-related expenses in the six months ended June 30, 2024 , compared to $36.5 million for the same period in 2023.

The decrease was primarily related to the Lexington facility being primarily dedicated to contract manufacturing during the six months ended June 30, 2024 compared to the facility having been dedicated to research and development activities in the prior period;

● We incurred $13.1 million in operating expenses and depreciation expenses related to our rented facilities in Amsterdam and Lexington, Massachusetts in the six months ended June 30, 2024 , compared to $14.5 million in the same period in 2023;

● We incurred $6.3 million in share-based compensation expenses in the six months ended June 30, 2024 , compared to $9.0 million for the same period in 2023.

The decrease was primarily a result of the reduction in expense from the restructuring that occurred in October 2023, and a decrease in the fair value of awards granted;

● We incurred $4.8 million of other expenses for the six months ended June 30, 2024 , compared to $5.7 million for the same period in 2023;

● We incurred $3.3 million in disposable costs in the six months ended June 30, 2024 , compared to $9.8 million for the same period in 2023.

The decrease was primarily a result of the reduction in expense from the restructuring that occurred in October 2023;

● We incurred $0.2 million of expenses in the three months ended March 31, 2024 related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $1.0 million increase in fair value for the same period in 2023.

● We incurred $1.1 million of severance costs related to the Lexington Transaction in the six months ended June 30, 2024 with no such cost in the same period in 2023;

● We recognized a $1.6 million gain in the six months ended June 30, 2024 related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to $1.2 million loss related to an increase in fair value for the same period in 2023.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended March 31, 2024 were $13.9 million, compared to $17.8 million for the same period in 2023.

● We incurred $6.2 million in personnel and contractor-related expenses in the three months ended March 31, 2024 , compared to $5.9 million in the same period in 2023;

● We incurred $3.4 million in share-based compensation expenses in the three months ended March 31, 2024 , compared to $3.7 million in the same period in 2023;

● We incurred $2.0 million in professional fees in the three months ended March 31, 2024 , compared to $3.2 million in the same period in 2023.

Selling, general and administrative expenses for the six months ended June 30, 2024 were $29.7 million, compared to $39.0 million for the same period in 2023.

● We incurred $11.9 million in personnel and contractor-related expenses in the six months ended June 30, 2024 , compared to $12.4 million in the same period in 2023.

The decrease was primarily related to the reduction in personnel and contractors as a result of the restructuring that occurred in October 2023;

● We incurred $6.8 million in share-based compensation expenses in the six months ended June 30, 2024 , compared to $7.8 million in the same period in 2023.

The decrease was primarily related to a decrease in the fair value of awards granted;

● We incurred $6.1 million in professional fees in the six months ended June 30, 2024 , compared to $5.7 million in the same period in 2023.

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

In the prior period, we incurred professional fees related to our global licensing agreement with Apic Bio;

● We incurred $2.0 million in other expenses in the three months ended March 31, 2024, compared to $4.0 million in the same period in 2023.

In 2024, we incurred professional fees related to the Lexington Transaction.

There was a partial offset as in the prior period we incurred professional fees related to our global licensing agreement with Apic Bio;

● We incurred $2.9 million in other expenses in the six months ended June 30 , 2024, compared to $6.2 million in the same period in 2023.

The $3.2 million decrease was primarily related to a decrease in intellectual property fees as well as a reduction of expenses for information technology;

● We incurred $0.3 million of severance costs related to the Lexington Transaction in the six months ended June 30, 2024 with no such cost in the same period in 2023;

● We incurred nil in financial advisory fees in relation to our licensing transaction with CSL Behring in the six months ended June 30, 2024, compared to $3.8 million in 2023.

Other items, net

We recognized $1.3 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the three months ended March 31, 2024, compared to $1.3 million for the same period in 2023.

We recognized $2.9 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the six months ended June 30, 2024, compared to $2.5 million for the same period in 2023.

Other items, net for the periods presented primarily related to income from the subleasing of our Amsterdam facility and expenses we incur in relation to the subleasing facility.

Other non-operating items, net

Our other non-operating items, net, for the three months ended March 31, 2024 and 2023 were as follows:

Three months ended March 31,

Our other non-operating items, net, for the six months ended June 30, 2024 and 2023 were as follows:

Six months ended June 30,

(in thousands)

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We recognize interest income associated with our cash and cash equivalents and investment securities.

We recognized $6.5 million in interest income in the three months ended March 31, 2024, compared to $1.7 million in the same period in the prior year.

Our interest income increased by $4.8 million due to the interest income earned on investment securities as well as cash on hand during the three months ended March 31, 2024.

We recognized $16.1 million in interest expense for the three months ended March 31, 2024 and $3.6 million for the three months ended March 31, 2023.

Our interest expense in 2024 increased due to an increase in market interest rates related to the Hercules debt as well as the recognition of $12.4 million of interest expense related to the Royalty Financing Agreement that we entered into in May 2023.

We recognized $12.3 million in interest income in the six months ended June 30, 2024, compared to $4.9 million in the same period in the prior year.

Our interest income increased by $7.4 million due to the interest income earned on investment securities as well as cash on hand during the six months ended June 30, 2024.

We recognized $32.3 million in interest expense for the six months ended June 30, 2024 and $10.4 million for the six months ended June 30, 2023.

Our interest expense in 2024 mainly increased due to an increase of $21.6 million in non-cash interest expense related to the Royalty Financing Agreement that we entered into in May 2023.

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.

We recognized a net foreign currency loss, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $1.1 million during the three months ended March 31, 2024, compared to a net loss of $2.4 million during the same period in 2023.

We recognized a net foreign currency loss, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $2.1 million during the six months ended June 30, 2024, compared to a net loss of $2.0 million during the same period in 2023.

Income tax benefit

We recognized $0.7 million of deferred tax expense in the three months ended March 31, 2024 , and $1.2 million of deferred tax benefit for the same period in 2023.

We recognized $1.6 million of deferred tax expense in the six months ended June 30, 2024 , and $1.0 million of deferred tax benefit for the same period in 2023 .

Financial Position, Liquidity and Capital Resources

As of March 31, 2024, we had cash and cash equivalents, restricted cash and investment securities of $558.9 million.

As of June 30, 2024, we had cash and cash equivalents, restricted cash and investment securities of $527.6 million.

Until such time, if ever, as we can generate substantial cash flows from successfully commercializing our proprietary product candidates, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution, and licensing arrangements.

Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs and following the Reorganization, we believe that our cash and cash equivalents and investment securities will fund our operations into second quarter of 2027.

Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe that our cash and cash equivalents and investment securities will fund our operations through the end of 2027.

We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

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Our material cash requirements include the following contractual and other obligations:

As of March 31, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc.

As of June 30, 2024, we had an outstanding loan amount owed to Hercules Capital, Inc.

(“Hercules”) for an aggregate principal amount of $100.0 million.

Future interest payments and financing fees associated with the loan total $44.2 million, with $13.4 million payable within 12 months.

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

On July 19, 2024, in connection with the closing of the Lexington Transaction, we repaid $50.0 million of the $100.0 million of principal outstanding as well as $3.1 million in end-of-term fees.

Future interest payments and financing fees associated with the loan after the $50.0 million payment in July total $24.4 million, with $10.6 million payable within 12 months.

We are contractually required to repay the remaining $50.0 million in full in January 2027.

We entered into lease arrangements for facilities, including corporate, manufacturing and office space.

As of March 31, 2024, we had fixed lease payment obligations of $50.7 million, with $8.4 million payable within 12 months.

As of June 30, 2024, we had fixed lease payment obligations of $48.5 million, with $8.5 million payable within 12 months.

Following the closing of the Lexington Transaction on July 22, 2024 we assigned our lease for the Lexington Facility to Genezen.

This will reduce our fixed lease payment obligations by $22.6 million, including $4.0 million payable within 12 months.

We continue to guarantee such payments until the end of the lease term in May 2029.

Commitments related to uniQure France SAS acquisition (nominal amounts)

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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 March 31, 2024, our remaining commitment amounts include a EUR 30.0 million ($32.4 million) milestone payment due upon treating the first patient in a Phase I/II clinical trial for AMT-260 and EUR 160.0 million ($172.7 million) in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the U.S.

As of June 30, 2024, our remaining commitment amounts include a EUR 30.0 million ($32.1 million) milestone payment due upon treating the first patient in a Phase I/II clinical trial for AMT-260 and EUR 160.0 million ($171.5 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 March 31, 2024, of $1.08/€1.00.

As of March 31, 2024, we expect these obligations will become payable between 2024 and 2031.

These payments are owed in euro and have been translated at the foreign exchange rate as of June 30, 2024, of $1.07/€1.00.

As of June 30, 2024, we expect these obligations will become payable between 2024 and 2033.

If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.

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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 three months ended:

Three months ended March 31,

The table below summarizes our consolidated cash flow data for the six months ended:

Six months ended June 30,

(in thousands)

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We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics Holding N.V.

(“AMT”) in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement.

in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement.

We continue to incur losses in the current period.

We recorded a net loss of $65.6 million in the three months ended March 31, 2024, compared to a net loss of $77.2 million during the same period in 2023.

As of March 31, 2024, we had an accumulated deficit of $956.0 million.

We recorded a net loss of $56.3 million and $121.9 million in the three and six months ended June 30, 2024, respectively, compared to a net loss of $68.5 million and $145.7 million during the same period in 2023.

As of June 30, 2024, we had an accumulated deficit of $1,012.3 million.

Sources of liquidity

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On May 12, 2023 we and Hercules amended the 2021 Restated Facility.

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 2023 Amended Facility extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027.

On June 28, 2024 we amended the 2023 Amended Facility and repaid $50.0 million of the principal outstanding at closing of the Lexington Transaction on July 22, 2024.

We are required to repay the remaining principal balance of $50.0 million on the maturity date.

The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum.

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Net cash used in operating activities

Net cash used in operating activities was $60.6 million for the three months ended March 31, 2024 and consisted of net loss of $65.6 million adjusted for non-cash items, including depreciation and amortization expense of $2.6 million, amortization of the discount on investment securities of $3.7 million, share-based compensation expense of $7.2 million, changes in the fair value of contingent consideration of $0.2 million, unrealized foreign exchange losses of $1.4 million, $12.4 million of interest expense related to the royalty financing and a change in deferred taxes of $0.7 million.

Net cash used in operating activities was $93.3 million for the six months ended June 30, 2024 and consisted of net loss of $121.9 million adjusted for non-cash items, including depreciation and amortization expense of $5.3 million, amortization of the discount on investment securities of $6.7 million, share-based compensation expense of $14.1 million, $24.8 million of interest expense related to the Royalty Financing Agreement, changes in the fair value of contingent consideration of $1.6 million, a change in deferred taxes of $1.6 million, a provision for inventory write-downs of $5.8 million and unrealized foreign exchange losses of $2.9 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $19.4 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $7.2 million.

There was a decrease in inventory balances of $2.2 million.

There was an increase in inventory balances of $2.6 million.

There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $9.5 million, primarily related to a decrease of $5.2 million from personnel related accruals.

Net cash used in operating activities was $78.3 million for the three months ended March 31, 2023 and consisted of net loss of $77.2 million adjusted for non-cash items, including depreciation and amortization expense of $2.5 million, amortization of the discount on investment securities of $0.9 million, share-based compensation expense of $8.1 million, changes in the fair value of contingent consideration of $1.0 million, unrealized foreign exchange losses of $1.2 million and a change in deferred taxes of $1.2 million.

Net cash used in operating activities was $135.3 million for the six months ended June 30, 2023 and consisted of net loss of $145.7 million adjusted for non-cash items, including depreciation and amortization expense of $5.1 million, amortization of the discount on investment securities of $1.8 million, share-based compensation expense of $17.0 million, $3.2 million of interest expense related to the Royalty Financing Agreement, a change in deferred taxes of $1.0 million, changes in the fair value of contingent consideration of $1.2 million and unrealized foreign exchange losses of $0.9 million.

Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $15.1 million.

There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $0.9 million.

There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $0.8 million.

There was an increase in inventory balances of $3.3 million.

These changes also relate to a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $12.6 million, primarily related to a decrease of $4.9 million from personnel related accruals.

Net cash generated from investing activities

In the three months ended March 31, 2024, we generated $64.4 million in our investing activities compared to generating $3.0 million for the same period in 2023.

Three months ended March 31,

In the six months ended June 30, 2024, we generated $141.9 million in our investing activities compared to generating $48.8 million for the same period in 2023.

Six months ended June 30,

(in thousands)

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Investment in investment securities

Build out of Amsterdam site

Build out of Lexington site

Capital expenditures - Amsterdam site

Capital expenditures - Lexington site

Net cash generated from investing activities

During the three months ended March 31, 2024, we received $150.1 million from the repayment of previous investments into euro and U.S.

dollar denominated government bonds ($5.3 million for three months ended March 31, 2023).

During the three months ended March 31, 2024, we invested $83.8 million of our cash on hand into euro denominated government bonds (nil for the three months ended March 31, 2023).

We invested $1.1 million and $1.3 million, respectively, into our Amsterdam, Netherlands and Lexington, Massachusetts sites during the three months ended March 31, 2024, compared to $0.7 million and $1.7 million for the same period in 2023.

During the six months ended June 30, 2024, we received $297.8 million from the repayment of previous investments into euro and U.S.

dollar denominated government bonds ($52.2 million for six months ended June 30, 2023).

During the six months ended June 30, 2024, we invested $152.9 million of our cash on hand into euro and dollar denominated government bonds (nil for the six months ended June 30, 2023).

We invested $1.5 million and $1.4 million, respectively, into our Amsterdam, Netherlands and Lexington, Massachusetts sites during the six months ended June 30, 2024, compared to $1.3 million and $2.1 million for the same period in 2023.

Net cash generated from financing activities

In the three months ended March 31, 2024, we generated nil from financing activities compared to $0.1 million for the same period in 2023.

Three months ended March 31,

In the six months ended June 30, 2024, we generated $0.1 million from financing activities compared to $370.3 million for the same period in 2023.

Six months ended June 30,

(in thousands)

Cash flows from financing activities

Proceeds from Royalty Financing Agreement, net of debt issuance costs

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

Net cash generated from financing activities

During the three months ended March 31, 2024, we received nil from the exercise of options to purchase ordinary shares in relation to our 2014 Plans, compared to $0.1 million for the same period in 2023.

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

During the six months ended June 30, 2024, we received $0.1 million from the exercise of options to purchase ordinary shares in relation to our 2014 Plans, compared to $0.2 million for the same period in 2023.

Funding requirements

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

Our future capital requirements, following the closing of the Lexington Transaction on July 22, 2024, 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 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, timing, results, and costs of our current and planned clinical trials, including those for funding late-stage clinical development of 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;

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● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims.

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

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-Q

Item 2. Management’s Discussion and Analysis