Item 2. Management’s Discussion and Analysis
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”). Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S. GAAP”) and unless otherwise indicated are presented in U.S. dollars.
Overview
We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, amyotrophic lateral sclerosis (“ALS”), refractory mesial temporal lobe epilepsy (“MTLE”) and Fabry disease.
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. (“US study”) as well as an open-label Phase Ib/II study in Europe with the same early-manifest criteria for Huntington’s disease as the U.S. study (“European study”). We completed the enrollment of all 26 patients in the first two cohorts of our US study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023.
In November 2023, we initiated patient dosing in a third cohort of up to 12 patients to further investigate both doses of AMT-130 in combination with perioperative immunosuppression. Patient enrollment is ongoing in this cohort with a focus on evaluating near-term safety and tolerability.
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:
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● 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.
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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 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. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept (“POC”) data.
Fabry disease program (AMT-191)
AMT-191 is our gene therapy candidate for the treatment of Fabry disease. AMT-191 comprises of an AAV5 capsid that incorporates the α-galactosidase A (“GLA”) transgene and a proprietary, highly potent, liver-specific promoter. In November 2023 we announced that the FDA had cleared the IND application for AMT-191.
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We are initiating a first-in-human Phase I/IIa clinical trial that will be conducted in the United States. 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.
Amyotrophic Lateral Sclerosis (AMT-162)
AMT-162 is our gene therapy candidate for a one-time, intrathecally administered investigational gene therapy for ALS caused by mutations in superoxide dismutase 1 (“SOD1”), a rapidly progressing, rare motor neuron disease that leads to loss of everyday functions and is uniformly fatal. Mutations in the SOD1 gene of ALS account for approximately one-fifth of all inherited forms of this fatal disease. AMT-162 is comprised of a recombinant AAVrh10 vector that expresses a miRNA designed to knock down the expression of SOD1 with the goal of slowing down or potentially reversing the progression of ALS in patients with SOD1 mutations. The FDA has cleared the IND application for AMT-162 and has granted Orphan Drug and Fast Track designation.
We are initiating a first-in-human Phase I/II clinical trial that will be a U.S.-based, multi-center, open-label trial consisting of three cohorts with up to four patients each receiving a one-time intrathecal infusion with immunosuppression. Safety, tolerability and early signs of efficacy will be evaluated in the study.
Financial Overview
Key components of our results of operations include the following:
Three months ended June 30,
Six months ended June 30,
2024
2023
2024
2023
(in thousands)
(in thousands)
Total revenues
$
11,126
$
2,422
$
19,611
$
7,747
Cost of license revenues
(234)
—
(384)
—
Cost of contract manufacturing revenues
(7,227)
(1,352)
(16,303)
(3,787)
Research and development expenses
(33,655)
(46,036)
(74,347)
(106,845)
Selling, general and administrative expenses
(15,767)
(21,181)
(29,704)
(39,029)
Net loss
(56,299)
(68,474)
(121,917)
(145,701)
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.
Critical Accounting Policies and Estimates
In preparing our consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the “SEC”) we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. A summary of our critical accounting policies as well as a discussion of our critical accounting estimates are presented in our Annual Report . There were no material changes to our critical accounting policies during the six months ended June 30, 2024 with the exception of determining the accounting treatment of the Lexington Transaction.
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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
We entered into a development and commercial supply agreement with CSL Behring in June 2020. We recognize the cost to manufacture HEMGENIX® under such agreement as cost of contract manufacturing.
Research and development expenses
We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:
● employee-related expenses, including salaries, benefits, travel and share-based compensation expense;
● costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;
● costs incurred to conduct consistency and comparability studies;
● costs incurred for the development and improvement of our manufacturing processes and methods;
● costs associated with research activities for enabling technology platforms, such as next-generation vectors, promoters and re-administration of gene therapies;
● 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;
● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and
● changes in the fair value of liabilities recorded in relation to our acquisition of uniQure France SAS.
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Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:
● the scope, rate of progress and expense of our research and development activities;
● our ability to successfully manufacture and scale-up production;
● clinical trial protocols, speed of enrollment and resulting data;
● the effectiveness and safety of our product candidates; and
● the timing of regulatory approvals.
A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidate.
Selling, general and administrative expenses
Our general and administrative expenses consist principally of employee, office, consulting, legal and other professional and administrative expenses. We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.
Other items, net
Our other income primarily consists of payments received to subsidize our research and development efforts and income from the subleasing of our Amsterdam facility.
Our other expense primarily consists of expenses we incur in relation to our subleasing income.
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Results of Operations
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,
2024
2023
2024 vs 2023
(in thousands)
Total revenues
$
11,126
$
2,422
$
8,704
Operating expenses:
Cost of license revenues
(234)
—
(234)
Cost of contract manufacturing
(7,227)
(1,352)
(5,875)
Research and development expenses
(33,655)
(46,036)
12,381
Selling, general and administrative expenses
(15,767)
(21,181)
5,414
Total operating expenses
(56,883)
(68,569)
11,686
Other income
1,983
1,302
681
Other expense
(236)
(229)
(7)
Loss from operations
(44,010)
(65,074)
21,064
Other non-operating items, net
(11,341)
(3,237)
(8,104)
Net loss before income tax expense
$
(55,351)
$
(68,311)
$
12,960
Income tax expense
(948)
(163)
(785)
Net loss
$
(56,299)
$
(68,474)
$
12,175
Revenues
Our revenues for the three months ended June 30, 2024 and 2023 were as follows:
Three months ended June 30,
2024
2023
2024 vs 2023
(in thousands)
License revenues
$
1,869
$
793
$
1,076
Contract manufacturing revenues
2,124
1,310
814
Collaboration revenues
7,133
319
6,814
Total revenues
$
11,126
$
2,422
$
8,704
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.
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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
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,
2024
2023
2024 vs 2023
(in thousands)
Huntington's disease (AMT-130)
$
2,894
$
4,243
$
(1,349)
Temporal lobe epilepsy (AMT-260)
1,998
2,486
(488)
Amyotrophic lateral sclerosis (AMT-162)
1,652
587
1,065
Fabry disease (AMT-191)
1,455
400
1,055
Programs in preclinical development and platform related expenses
898
2,257
(1,359)
Etranacogene dezaparvovec (AMT-060/061)
—
(2,003)
2,003
Total direct research and development expenses
$
8,897
$
7,970
$
927
Employee and contractor-related expenses
13,035
17,760
(4,725)
Facility expenses
5,976
7,676
(1,700)
Share-based compensation expense
2,856
4,732
(1,876)
Other expenses
2,159
1,786
373
Disposables
1,420
5,875
(4,455)
Severance costs
1,098
—
1,098
Fair value changes related to contingent consideration
(1,786)
237
(2,023)
Total other research and development expenses
$
24,758
$
38,066
$
(13,308)
Total research and development expenses
$
33,655
$
46,036
$
(12,381)
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.
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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; and
● 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;
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● 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; and
● 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,
2024
2023
2024 vs 2023
(in thousands)
Interest income
$
5,805
$
3,229
$
2,576
Interest expense
(16,157)
(6,840)
(9,317)
Foreign currency (losses) / gains, net
(989)
374
(1,363)
Total non-operating expense, net
$
(11,341)
$
(3,237)
$
(8,104)
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. dollars. 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.
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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,
2024
2023
2024 vs 2023
(in thousands)
Total revenues
$
19,611
$
7,747
$
11,864
Operating expenses:
Cost of license revenues
(384)
—
(384)
Cost of contract manufacturing
(16,303)
(3,787)
(12,516)
Research and development expenses
(74,347)
(106,845)
32,498
Selling, general and administrative expenses
(29,704)
(39,029)
9,325
Total operating expenses
(120,738)
(149,661)
28,923
Other income
3,359
3,113
246
Other expense
(470)
(445)
(25)
Loss from operations
(98,238)
(139,246)
41,008
Non-operating expense, net
(22,075)
(7,499)
(14,576)
Loss before income tax benefit
$
(120,313)
$
(146,745)
26,432
Income tax (expense) / benefit
(1,604)
1,044
(2,648)
Net loss
$
(121,917)
$
(145,701)
$
23,784
Revenue
Our revenue for the six months ended June 30, 2024 and 2023 was as follows:
Six months ended June 30,
2024
2023
2024 vs 2023
(in thousands)
License revenues
$
3,071
$
793
$
2,278
Contract manufacturing revenues
6,114
6,247
(133)
Collaboration revenues
10,426
707
9,719
Total revenues
$
19,611
$
7,747
$
11,864
License revenues
We recognize license revenues from CSL Behring, related to royalty payments owed on HEMGENIX® sales, when earned. 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
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 $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. Collaboration revenue related to these contracted services is recognized when the performance obligations are satisfied.
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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 $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 expense
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.
Six months ended June 30,
2024
2023
2024 vs 2023
(in thousands)
Huntington's disease (AMT-130)
5,320
7,976
(2,656)
Temporal lobe epilepsy (AMT-260)
4,252
7,308
(3,056)
Amyotrophic lateral sclerosis (AMT-162)
$
3,629
$
10,628
$
(6,999)
Fabry disease (AMT-191)
2,825
1,488
1,337
Programs in preclinical development and platform related expenses
1,347
4,142
(2,795)
Etranacogene dezaparvovec (AMT-060/061)
—
(1,336)
1,336
Total direct research and development expenses
$
17,373
$
30,206
$
(12,833)
Employee and contractor-related expenses
29,958
36,463
(6,505)
Facility expenses
13,071
14,462
(1,391)
Share-based compensation expense
6,281
9,037
(2,756)
Other expenses
4,846
5,684
(838)
Disposables
3,341
9,781
(6,440)
Severance costs
1,098
—
1,098
Fair value changes related to contingent consideration
(1,621)
1,212
(2,833)
Total other research and development expenses
$
56,974
$
76,639
$
(19,665)
Total research and development expenses
$
74,347
$
106,845
$
(32,498)
Direct research and development expenses
Huntington’s disease (AMT-130)
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. 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 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.
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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 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 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 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 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 $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 $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; and
● 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 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;
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● 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 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; and
● 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 $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 six months ended June 30, 2024 and 2023 were as follows:
Six months ended June 30,
2024
2023
2024 vs 2023
(in thousands)
Interest income
$
12,313
$
4,898
$
7,415
Interest expense
(32,254)
(10,402)
(21,852)
Foreign currency losses, net
(2,134)
(1,995)
(139)
Total non-operating expense, net
$
(22,075)
$
(7,499)
$
(14,576)
We recognize interest income associated with our cash and cash equivalents and investment securities. 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. dollars. 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 $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 $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 .
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Financial Position, Liquidity and Capital Resources
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, we believe that our cash and cash equivalents and investment securities will fund our operations through the end of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. We expect that we will require additional funding if we decide to advance AMT-130 for our Huntington’s disease gene therapy program or any of our other product candidates into late-stage clinical development. Our material cash requirements include the following contractual and other obligations:
Debt
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. 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.
Leases
We entered into lease arrangements for facilities, including corporate, manufacturing and office space. 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)
In relation to our acquisition of uniQure France SAS in 2021, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractual milestones. The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. In September 2023, we made a payment of EUR 10.0 million ($10.6 million) to the former shareholders of uniQure France SAS following the FDA’s clearance of the IND application for AMT-260. As of 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 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.
Commitments related to licensors and financial advisors
We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our sale of the exclusive global rights of HEMGENIX® to CSL Behring. We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.
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The table below summarizes our consolidated cash flow data for the six months ended:
Six months ended June 30,
2024
2023
(in thousands)
Cash, cash equivalents and restricted cash at the beginning of the period
$
244,544
$
231,173
Net cash used in operating activities
(93,286)
(135,299)
Net cash generated from investing activities
141,922
48,818
Net cash generated from financing activities
50
370,268
Foreign exchange impact
(2,191)
1,812
Cash, cash equivalents and restricted cash at the end of period
$
291,039
$
516,772
We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement. We continue to incur losses in the current period. We recorded a net loss of $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
From our first institutional venture capital financing in 2006 through the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners as well as from selling a portion of royalties due from our collaboration partner CSL Behring. We have collected $100.0 million in July 2023 related to the first sale milestone of HEMGENIX® in the U.S., and are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from royalty financing) on net sales of HEMGENIX®.
On May 12, 2023 we and Hercules amended the 2021 Restated Facility. The 2023 Amended Facility extended the maturity date and interest-only period from December 1, 2025 to January 5, 2027. 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. Under the 2024 Amended Facility, we owe a back-end fee of $2.4 million on December 1, 2025 and a back-end fee of $0.6 million on the maturity date.
We are subject to certain covenants under the 2024 Amended Facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business. In addition, our pledge of assets as collateral to secure our obligations under the 2024 Amended Facility may limit our ability to obtain debt financing. The 2024 Amended Facility permits us to issue up to $500.0 million of convertible debt.
To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all. If financing is not available when needed, including through debt or equity financing, or is available only on unfavorable terms, we may be unable to meet our cash needs. If we raise additional funds through collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.
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Net cash used in operating activities
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 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 $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 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 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,
2024
2023
(in thousands)
Proceeds from maturity of investment securities
$
297,806
$
52,234
Investment in investment securities
(152,936)
—
Capital expenditures - Amsterdam site
(1,532)
(1,294)
Capital expenditures - Lexington site
(1,416)
(2,122)
Net cash generated from investing activities
$
141,922
$
48,818
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.
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Net cash generated from financing activities
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,
2024
2023
(in thousands)
Cash flows from financing activities
Proceeds from Royalty Financing Agreement, net of debt issuance costs
$
-
$
370,062
Proceeds from issuance of ordinary shares related to employee stock option and purchase plans
50
206
Net cash generated from financing activities
$
50
$
370,268
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, 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 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;
● the extent to which we acquire or in-license other businesses, products, product candidates or technologies;
● the amount and timing of revenue, if any, we receive from manufacturing products for CSL Behring;
● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates;
● the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates;
● the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies;
● the cost, timing and outcome of regulatory reviews associated with our product candidates;
● our ability to enter into collaboration arrangements in the future; and
● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims.
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