What changed in this filing

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Item 2. Management’s Discussion and Analysis

2022-11-02 compared with 2022-08-08 · 148 added, 155 removed, 328 unchanged (48% of the section changed)

20 unchanged sentences

In the study, patients are randomized to either treatment with AMT-130 or to an imitation surgical procedure.

The treated patients have received a single administration of AMT-130 using MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).The trial consists of a blinded 12-month period followed by unblinded long-term follow-up for five years.

The treated patients have received a single administration of AMT-130 using MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).

The trial consists of a blinded 12-month period followed by unblinded long-term follow-up for five years.

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

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

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

A third cohort, which will include up to 18 additional randomized patients receiving the higher dose, will explore the use of alternative stereotactic navigation systems to simplify placement of catheters for infusions of AMT-130.

15 unchanged sentences

The reactions in these three patients each have been classified as suspected unexpected serious adverse reactions (“SUSARs”) and reported to the appropriate regulatory agencies in the U.S.

Following a review of these SUSARs by the independent Data Safety Monitoring Board for the clinical trial (the “DSMB”), the DSMB did not believe that such reactions represented dose limiting toxicities, but did recommend postponement of further enrollment and dosing of high dose patients and postponement of further site activation until the DSMB conducts a comprehensive safety review.

We are following the DSMB’s recommendations and expect to hold the safety review in the third quarter or early fourth quarter of 2022.

While the DSMB recommended postponement of dosing of patients with the AMT-130 high dose, it also stated that eligible trial participants that did not initially receive treatment in the U.S.

low dose cohort could cross over to receive a low dose intervention.

The Company expects that such control patients from the U.S.

low dose cohort will cross over to receive the low dose intervention during the third quarter of 2022.

Phase I/II clinical trial, all 26 patients in the first two dose cohorts have been enrolled.

In the European Phase Ib/II study, the six patient lower dose cohort is fully enrolled.

Four of nine patients in the European clinical trial have been enrolled in the higher dose cohort.

To date, a total of 36 patients have been enrolled across the two clinical trials including 10 control patients and 26 patients treated with AMT-130, of which 14 patients received the higher dose and 12 patients received the low dose.

Following a review of these SUSARs by the independent Data Safety Monitoring Board for the clinical trial (the “DSMB”) in July 2022, the DSMB recommended postponement of further enrollment and dosing of high-dose patients and postponement of further site activation until the DSMB had conducted a comprehensive safety investigation.

We completed a comprehensive safety investigation in October 2022.

The DSMB recommended resuming treatment at the higher dose of AMT-130 for the remaining five European patients and any patients in the U.S.

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

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

We have added additional risk mitigation procedures including closer patient monitoring during the first two weeks after the administration of AMT-130 and a seven-day, post-surgical in-person visit.

The DSMB recommended that the use of immunosuppression remain at the discretion of the treating physician.

We plan to resume patient dosing in the open-label European study in the fourth quarter of 2022 and complete enrollment of the remaining five of the nine patient higher-dose cohort in the first half of 2023.

CSL Behring collaboration

1 unchanged sentence

The transaction became fully effective on May 6, 2021, one day after the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 expired (“Closing”).

In accordance with the CSL Behring agreement, CSL Behring is solely responsible for all regulatory activities, including any filings and agency interactions associated with etranacogene dezaparvovec and the companion diagnostic test for neutralizing antibodies to AAV5.

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

2 unchanged sentences

On May 24, 2022, we announced that the FDA had accepted for review the Biologics License Application (“BLA”).

As of June 30, 2022, we received the $55.0 million owed to us by CSL Behring related to the global regulatory submissions.

In March and April 2022, we received the $55.0 million owed to us by CSL Behring related to the global regulatory submissions.

In July 2022, CSL Behring was notified by the Committee for Advanced Therapies (“CAT”) in Europe that they will be unable to complete their review in accordance with the accelerated assessment timetable and will now switch to a standard review procedures.

In the United States, the BLA remains under Priority Review at this time.

In the United States, the BLA remains under Priority Review at this time having Breakthrough Designation Status.

In July 2022, following a comprehensive multi-day facility inspection, the EMA notified us that Good Manufacturing Practice (“GMP”) certification can be issued for our Lexington manufacturing site to produce commercial supply of etranacogene dezaparvovec.

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

In August 2022, the GMP certification for the Amsterdam facility was amended to include release testing of the Product in the European Union following inspection by the Inspectie Gezondheidszorg en Jeugd (“IGJ”).

On September 6, 2022 as discussed above, CSL Behring notified us of its intent to transfer manufacturing technology related to the Product to a third-party contract manufacturer designated by CSL Behring and to retain the Company as a source for manufacturing.

Preclinical programs

In May 2022, we presented certain preclinical findings on our gene therapy candidates for refractory temporal lobe epilepsy, Fabry disease, Parkinson’s disease, Amyotrophic lateral sclerosis, and Alzheimer’s disease at the American Society of Gene and Cell Therapy Hybrid Congress.

In July and August 2022, respectively, we initiated Investigational New Drug-enabling (“IND-enabling”), Good Laboratory Practice (“GLP”) toxicology studies in non-human primates for our gene therapy candidates in refractory temporal lobe epilepsy and Fabry disease.

Covid pandemic

1 unchanged sentence

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

a) Workplace and employees

Throughout the pandemic, we have been implementing measures to address the impact of Covid on our business.

We have implemented a series of protocols governing the operations of our Lexington facility to comply with the requirements of the various orders and guidance from the Commonwealth of Massachusetts and other related orders, guidance, laws, and regulations.

We continue to monitor local government rules and recommendations and office protocols will be aligned with these rules and recommendations.

Effective June 9, 2022, we strongly encourage all vaccinated employees to wear facemasks in the Lexington facility, particularly for in-person meetings and when social distancing is not possible.

The Covid-19 Protocol in the Lexington facility continues to require unvaccinated employees to wear facemasks and apply social distancing at the office.

All non-essential employees in the Lexington facility are allowed to return to the office, but have the option to work from home or from another remote location through at least August 31, 2022.

Effective February 28, 2022, the Amsterdam facility is open to all employees and visitors, in line with the most updated Dutch government measures.

We will continue to comply with additional Dutch measures, which amongst others is recommended for employers to make agreements with employees that allow working from home to continue to the extent applicable.

b) Business impact

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

operations and overall financial performance remain uncertain.

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

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

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

10 unchanged sentences

On November 17, 2021, the Patent Trials and Appeals Board (“PTAB”) issued decisions granting institution on all three IPR proceedings.

The parties completed briefing of the substantive issues in July 2022 and a hearing is planned before the PTAB in August 2022.

The parties completed briefing of the substantive issues in July 2022 and a hearing was held before the PTAB in August 2022.

We expect a decision from the PTAB on or about November 2022.

Financial Overview

Key components of our results of operations include the following:

Three months ended June 30,

Six months ended June 30,

Three months ended September 30,

Nine months ended September 30,

(in thousands)

6 unchanged sentences

Net (loss) / income

As of June 30, 2022 and December 31, 2021, we had cash and cash equivalents of $500.5 million and $556.3 million, respectively.

We had a net loss of $39.1 million and $85.7 in the three and six months ended June 30, 2022, respectively, compared to net income of $399.5 and $357.9 million for the same periods in 2021.

As of June 30, 2022 and December 31, 2021, we had accumulated deficits of $540.9 million and $455.1 million, respectively.

As of September 30, 2022 and December 31, 2021, we had cash and cash equivalents of $440.3 million and $556.3 million, respectively.

We had a net loss of $47.9 million and $133.6 in the three and nine months ended September 30, 2022, respectively, compared to net loss of $36.5 million and net income of $321.4 million for the same periods in 2021.

As of September 30, 2022 and December 31, 2021, we had accumulated deficits of $588.7 million and $455.1 million, respectively.

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

8 unchanged sentences

In preparing our consolidated financial statements in accordance with U.S.

GAAP and pursuant to the rules and regulations promulgated by 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.

GAAP and pursuant to the rules

and regulations promulgated by 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.

2 unchanged sentences

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, 2022 or reasonably possible changes of our critical accounting estimates as of June 30, 2022 that could have had a material impact on our results of operations for the three and six-month period ended June 30, 2022.

There were no material changes to our critical accounting policies during the nine months ended September 30, 2022 or reasonably possible changes of our critical accounting estimates as of September 30, 2022 that could have had a material impact on our results of operations for the three and nine-month period ended September 30, 2022.

Cost of contract revenues

We expense contract fulfillment costs associated with license revenue recognized under the CSL Behring Agreement as cost of contract revenues.

We expense contract fulfillment costs associated with the sale of the exclusive global rights to the Product recognized under the CSL Behring Agreement as cost of contract revenues.

Cost of contract manufacturing

28 unchanged sentences

We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.

Our selling costs in the three and six months ended June 30, 2021 included employee expenses, as well as professional fees related to the preparation of a commercial launch of etranacogene dezaparvovec and advisory fees related to obtaining the CSL Behring Agreement.

Our selling costs in the prior year included employee expenses as well as professional fees related to the preparation of a commercial launch of etranacogene dezaparvovec and advisory fees related to obtaining the CSL Behring Agreement.

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.

For the three and six months ended June 30, 2021, other income also consisted of the recognition of the equity stake received in VectorY B.V., as well as an employee retention credit under the U.S.

Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”).

For the three and nine months ended September 30, 2021, other income consisted of an employee retention credit under the U.S.

Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”), and for the nine months ended September 30, 2021, it also included the recognition of the equity stake received in VectorY B.V.

Our other expense primarily consists of expenses we incur in relation to our subleasing income.

Results of Operations

Comparison of the three months ended June 30, 2022 and 2021

The following table presents a comparison of our results of operations for the three months ended June 30, 2022 and 2021.

Three months ended June 30,

Comparison of the three months ended September 30, 2022 and 2021

The following table presents a comparison of our results of operations for the three months ended September 30, 2022 and 2021.

Three months ended September 30,

(in thousands)

1 unchanged sentence

Operating expenses:

Cost of contract revenues

Cost of contract manufacturing

3 unchanged sentences

Other expense

(Loss) / income from operations

Loss from operations

Other non-operating items, net

Net (loss) / income before income tax benefit / (expense)

Net loss before income tax expense

Income tax benefit / (expense)

Net (loss) / income

Our revenue for the three months ended June 30, 2022 and 2021 was as follows:

Three months ended June 30,

Our revenue for the three months ended September 30, 2022 and 2021 was as follows:

Three months ended September 30,

(in thousands)

License revenue

Collaboration revenue

Total revenues

We did not recognize any license revenue related to the CSL Behring Agreement in the three months ended June 30, 2022.

In the three months ended June 30, 2021, we recognized $462.4 million in license revenue related to the sale of the exclusive global rights to the Product (“License Sale”) on Closing of the CSL Behring Agreement in May 2021.

We recogniz ed nil collaboration revenue in the three months ended June 30, 2022, compared to $0.4 million for the same period in 2021.

We recogniz ed $0.7 million collaboration revenue in the three months ended September 30, 2022, compared to $0.5 million for the same period in 2021 from the CSL Behring Agreement.

Since May 6, 2021, we recognize collaboration revenue related to services we provide in accordance with the CSL Behring Agreement.

We recognized $0.5 million collaboration revenue in the three months ended June 30, 2022 compared to $1.1 million for the three months ended June 30, 2021.

Cost of contract revenues

We did not recognize any cost of contract revenues for the three months ended June 30, 2022 compared to $23.2 million for the three months ended June 30, 2021.

The cost recorded in 2021 included $16.7 million of expenses related to payments owed to our licensors on Closing as well as $6.5 million of other external expenses incurred to fulfill the License Sale to CSL Behring.

We recognized $0.8 million collaboration revenue in the three months ended September 30, 2022 compared to $1.5 million for the three months ended September 30, 2021.

Cost of contract manufacturing

We incurred $0.8 million of cost of contract manufacturing related to the manufacture of the Product in the three months ended June 30, 2022, which we did not capitalize as inventory in accordance with our accounting policies.

We incurred $0.9 million of cost of contract manufacturing related to the manufacture of the Product in the three months ended September 30, 2022, which we did not capitalize as inventory in accordance with our accounting policies, compared to nil cost of contract manufacturing in the three months ended September 30, 2021.

Research and development expenses

Research and development expenses for the three months ended June 30, 2022 were $46.2 million, compared to $32.7 million for the same period in 2021.

Research and development expenses for the three months ended September 30, 2022 were $48.1 million, compared to $36.4 million for the same period in 2021.

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,

Three months ended September 30,

(in thousands)

Temporal lobe epilepsy (AMT-260)

Huntington's disease (AMT-130)

Programs in preclinical development and platform related expenses

Temporal lobe epilepsy (AMT-260)

Fabry disease (AMT-190)

Etranacogene dezaparvovec (AMT-060/061)

Other programs in preclinical development and platform related expenses

Total direct research and development expenses

1 unchanged sentence

Facility expenses

Fair value changes related to contingent consideration

Share-based compensation expense

Other expenses

Fair value changes related to contingent consideration

Total other research and development expenses

1 unchanged sentence

Direct research and development expenses

Temporal lobe epilepsy (AMT-260)

In the three months ended June 30, 2022, and June 30, 2021, we incurred $6.3 million and nil, respectively for the preclinical development of temporal lobe epilepsy, which we acquired from Corlieve Therapeutics SAS (“Corlieve”) on July 30, 2021.

Huntington disease (AMT-130)

In the three months ended June 30, 2022 and June 30, 2021, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States.

Expenses incurred for the three months ended June 30, 2022 also included costs related to our Phase Ib/II clinical trial in Europe .

Preclinical programs & platform development

In the three months ended June 30, 2022, we incurred $2.2 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of Fabry disease (AMT-191), as well as various other research programs and technology innovation projects.

In the three months ended June 30, 2021, we incurred $2.4 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of SCA3 (AMT-150) and Fabry disease (AMT-190), as well as various other research programs and technology innovation projects.

In the three months ended September 30, 2022, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States and in Europe.

In the three months ended September 30, 2021, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States.

Temporal lobe epilepsy (AMT-260)

In the three months ended September 30, 2022 and September 30, 2021, we incurred $2.5 million and $0.1 million, respectively, for the preclinical development of temporal lobe epilepsy, which we acquired from Corlieve Therapeutics SAS (“Corlieve”) on July 30, 2021.

Fabry disease (AMT-190)

In the three months ended September 30, 2022 and September 30, 2021, we incurred $0.5 million and $0.1 million of costs, respectively, primarily related to our preclinical activities for the treatment of Fabry disease (AMT-190).

Hemophilia B (AMT-060/061)

In the three months ended June 30, 2022 and June 30, 2021, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial and Manufacturing Development.

In the three months ended September 30, 2022 and September 30, 2021, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial and Manufacturing Development.

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

After the Closing, CSL Behring is responsible for the clinical and regulatory activities and commercialization of the Product, with the Company managing the existing trials on behalf of CSL Behring.

Direct research and development expenses related to clinical development incurred in the three months ended June 30, 2022 are presented net of reimbursements due from CSL Behring.

Direct research and development expenses related to clinical development incurred in the three months ended September 30, 2022 and September 30, 2021 are presented net of reimbursements due from CSL Behring.

I n the same periods in 2022 and 2021, we also incurred costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec.

2 unchanged sentences

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

Preclinical programs & platform development

In the three months ended September 30, 2022, we incurred $0.9 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of various other research programs and technology innovation projects.

In the three months ended September 30, 2021, we incurred $2.2 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of SCA3 (AMT-150), as well as various other research programs and technology innovation projects.

Other research & development expenses

● We incurred $15.1 million in personnel and contractor related expenses in the three months ended June 30, 2022, compared to $13.1 million for the same period in 2021.

● We incurred $15.6 million in personnel and contractor related expenses in the three months ended September 30, 2022, compared to $14.6 million for the same period in 2021.

The increase was primarily a result of the recruitment of personnel to support the development of our product candidates;

● We incurred $5.4 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended June 30, 2022, compared to $4.4 million in the same period in 2021 as a result of investments we made to expand our facilities;

● We incurred $4.5 million in disposable costs compared to $3.6 million for the same period in 2021;

● We incurred $4.3 million in share-based compensation expenses in the three months ended June 30, 2022, compared to $3.3 million for the same period in 2021.

● We incurred $6.5 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended September 30, 2022, compared to $4.8 million in the same period in 2021 as a result of investments we made to expand our facilities;

● We incurred $6.0 million in disposable costs compared to $4.8 million for the same period in 2021 as we continue to expand our activities to support the development of our product candidates;

● We incurred $5.5 million of expenses in the three months ended September 30, 2022 related to an increase in the fair value of contingent consideration associated with the acquisition of Corlieve Therapeutics SAS (“Corlieve”), compared to $0.4 million in the same period in 2021;

● We incurred $3.7 million in share-based compensation expenses in the three months ended September 30, 2022, compared to $3.2 million for the same period in 2021.

The increase was primarily a result of an increase in awards granted, including those to newly recruited personnel;

● We incurred $2.1 million of other expenses for the three months ended June 30, 2022, compared to $1.2 million for the same period in 2021.

● We incurred $2.2 million of other expenses for the three months ended September 30, 2022, compared to $1.3 million for the same period in 2021.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended June 30, 2022 were $12.5 million, compared to $17.3 million for the same period in 2021.

● We incurred $5.2 million in personnel and contractor related expenses in the three months ended June 30, 2022, compared to $4.2 million in the same period in 2021;

● We incurred $3.4 million in share-based compensation expenses in the three months ended June 30, 2022, compared to $3.7 million in the same period in 2021;

● We incurred $1.5 million in professional fees in the three months ended June 30, 2022, compared to $2.0 million in the same period in 2021.

Selling, general and administrative expenses for the three months ended September 30, 2022 were $13.3 million, compared to $12.0 million for the same period in 2021.

● We incurred $5.2 million in personnel and contractor related expenses in the three months ended September 30, 2022, compared to $3.8 million in the same period in 2021;

● We incurred $3.8 million in share-based compensation expenses in the three months ended September 30, 2022, compared to $2.7 million in the same period in 2021;

● We incurred $1.6 million in professional fees in the three months ended September 30, 2022, compared to $1.5 million in the same period in 2021.

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

● We incurred nil financial advisory fees in relation to our licensing transaction with CSL Behring in the three months ended June 30, 2022, compared to $4.5 million in the same period in 2021.

Other items, net

In the three months ended June 30, 2022, we recognized $0.3 million in other income in relation to the equity stake received in VectorY B.V.

in conjunction with a settlement agreement entered into in April 2021 compared to $3.0 million such income for the same period in 2021.

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

CARES Act in the three months ended June 30, 2022, compared to $1.3 million for the same period in 2021.

We recognized $2.6 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, 2022, compared to $3.0 million for the same period in 2021.

CARES Act in the three months ended September 30, 2022, compared to $1.3 million for the same period in 2021.

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 September 30, 2022, compared to $0.0 million for the same period in 2021.

Other income also includes income from subleasing of a portion of our Amsterdam facility.

1 unchanged sentence

Other non-operating items, net

Our other non-operating items, net, for the three months ended June 30, 2022 and 2021 were as follows:

Three months ended June 30,

Our other non-operating items, net, for the three months ended September 30, 2022 and 2021 were as follows:

Three months ended September 30,

(in thousands)

2 unchanged sentences

Foreign currency gains, net

Other non-operating losses

Total other non-operating income, net

2 unchanged sentences

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

We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $19.4 million during the three months ended June 30, 2022, compared to a net gain of $6.6 million during the same period in 2021.

We recognize fair value changes related to the derivative financial liability related to a contingent payment due to BMS upon the consummation of a change of control transaction (“CoC-payment”) as described elsewhere in this Quarterly Report on Form 10-Q.

We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $14.4 million during the three months ended September 30, 2022, compared to a net gain of $10.4 million during the same period in 2021.

Income tax benefit / (expense)

We recognized $0.3 million of deferred tax benefit in the three months ended June 30, 2022, and $3.3 million of deferred tax expense for the same period in 2021.

Comparison of the six months ended June 30, 2022 and 2021

The following table presents a comparison of our results of operations for the six months ended June 30, 2022 and 2021.

Six months ended June 30,

We recognized $0.3 million of deferred tax benefit in the three months ended September 30, 2022, and $0.1 million of deferred tax expense for the same period in 2021.

Comparison of the nine months ended September 30, 2022 and 2021

The following table presents a comparison of our results of operations for the nine months ended September 30, 2022 and 2021.

Nine months ended September 30,

(in thousands)

12 unchanged sentences

Net (loss) / income

Our revenue for the six months ended June 30, 2022 and 2021 was as follows:

Six months ended June 30,

Our revenue for the nine months ended September 30, 2022 and 2021 was as follows:

Nine months ended September 30,

(in thousands)

2 unchanged sentences

Total revenues

We did not recognize any license revenue related to the CSL Behring Agreement in the six months ended June 30, 2022.

In the six months ended June 30, 2021, we recognized $462.4 million in license revenue related to the License Sale on Closing of the CSL Behring Agreement in May 2021.

We recognized $1.4 million collaboration revenue in the six months ended June 30, 2022, compared to $0.4 million for the same period in 2021.

We did not recognize any license revenue related to the CSL Behring Agreement in the nine months ended September 30, 2022.

In the nine months ended September 30, 2021, we recognized $462.4 million in license revenue related to the sale of the exclusive global rights to the Product (“License Sale”) on Closing of the CSL Behring Agreement in May 2021.

We recognized $2.1 million collaboration revenue in the nine months ended September 30, 2022, compared to $0.9 million for the same period in 2021.

Since May 6, 2021, we recognize collaboration revenue related to services we provide in accordance with the CSL Behring Agreement.

We recognized $0.8 million collaboration revenue in the six months ended June 30, 2022 compared to $1.5 million for the six months ended June 30, 2021.

We recognized $1.6 million collaboration revenue in the nine months ended September 30, 2022 compared to $3.0 million for the nine months ended September 30, 2021.

Cost of contract revenues

We did not recognize any cost of contract revenues for the six months ended June 30, 2022 compared to $23.2 million for the six months ended June 30, 2021.

We did not recognize any cost of contract revenues for the nine months ended September 30, 2022 compared to $23.2 million for the nine months ended September 30, 2021.

Cost of contract manufacturing

We incurred $0.8 million of cost of contract manufacturing related to the manufacture of the Product in the six months ended June 30, 2022, which we did not capitalize as inventory in accordance with our accounting policies.

We incurred $1.7 million of cost of contract manufacturing related to the manufacture of the Product in the nine months ended September 30, 2022, which we did not capitalize as inventory in accordance with our accounting policies, compared to nil cost of contract manufacturing in the nine months ended September 30, 2021.

Research and development expenses

Research and development expenses for the six months ended June 30, 2022 were $91.2 million, compared to $64.8 million for the same period in 2021.

Research and development expenses for the nine months ended September 30, 2022 were $139.3 million, compared to $101.2 million for the same period in 2021.

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,

Nine months ended September 30,

(in thousands)

1 unchanged sentence

Temporal lobe epilepsy (AMT-260)

Programs in preclinical development and platform related expenses

Fabry disease (AMT-190)

Etranacogene dezaparvovec (AMT-060/061)

Other programs in preclinical development and platform related expenses

Total direct research and development expenses

2 unchanged sentences

Share-based compensation expense

Other expenses

Fair value changes related to contingent consideration

Other expenses

Total other research and development expenses

2 unchanged sentences

Huntington disease (AMT-130)

In the six months ended June 30, 2022 and June 30, 2021, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States.

Expenses incurred for the six months ended June 30, 2022 also included costs related to our Phase Ib/II clinical trial in Europe .

In the nine months ended September 30, 2022, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States and in Europe.

In the nine months ended September 30, 2021, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States.

Temporal lobe epilepsy (AMT-260)

In the six months ended June 30, 2022 and June 30, 2021, we incurred $8.9 million and nil, respectively for the preclinical development of temporal lobe epilepsy, which we acquired from Corlieve on July 31, 2021.

Preclinical programs & platform development

In the six months ended June 30, 2022, we incurred $4.1 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of Fabry disease (AMT-191), as well as various other research programs and technology innovation projects.

In the six months ended June 30, 2021, we incurred $4.7 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of SCA3 (AMT-150) and Fabry disease (AMT-190), as well as various other research programs and technology innovation projects.

In the nine months ended September 30, 2022 and September 30, 2021, we incurred $11.4 million and $0.1 million, respectively, for the preclinical development of temporal lobe epilepsy, which we acquired from Corlieve on July 30, 2021.

Fabry disease (AMT-190)

In the nine months ended September 30, 2022 and September 30, 2021, we incurred $1.7 million and $0.3 million of costs, respectively, primarily related to our preclinical activities for the treatment of Fabry disease (AMT-190).

Hemophilia B (AMT-060/061)

In the six months ended June 30, 2022 and June 30, 2021, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial and Manufacturing Development.

In the nine months ended September 30, 2022 and September 30, 2021, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial and Manufacturing Development.

After the Closing, CSL Behring is responsible for the clinical and regulatory activities and commercialization of the Product, with the Company managing the existing trials on behalf of CSL Behring.

Direct research and development expenses related to clinical development incurred in the six months ended June 30, 2022 are presented net of reimbursements due from CSL Behring.

Direct research and development expenses related to clinical development incurred in the nine months ended September 30, 2022 and September 30, 2021 are presented net of reimbursements due from CSL Behring.

In the same periods in 2022 and 2021, we also incurred costs related to the long-term follow-up of patients in our Phase I/II clinical trial of AMT-060 and our Phase IIb clinical trial of etranacogene dezaparvovec.

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

Preclinical programs & platform development

In the nine months ended September 30, 2022, we incurred $3.9 million of costs primarily related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

In the nine months ended September 30, 2021, we incurred $6.2 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of SCA3 (AMT-150), as well as various other research programs and technology innovation projects.

Other research & development expenses

● We incurred $31.2 million in personnel and contractor related expenses in the six months ended June 30, 2022, compared to $24.7 million for the same period in 2021.

● We incurred $46.8 million in personnel and contractor related expenses in the nine months ended September 30, 2022, compared to $39.3 million for the same period in 2021.

The increase was primarily a result of the recruitment of personnel to support the development of our product candidates;

● We incurred $10.7 million in operating expenses and depreciation expenses related to our rented facilities in the six months ended June 30, 2022, compared to $9.0 million in the same period in 2021 as a result of investments we made to expand our facilities;

● We incurred $9.1 million in disposable costs compared to $6.9 million for the same period in 2021;

● We incurred $8.4 million in share-based compensation expenses in the six months ended June 30, 2022, compared to $6.0 million for the same period in 2021.

● We incurred $17.2 million in operating expenses and depreciation expenses related to our rented facilities in the nine months ended September 30, 2022, compared to $13.8 million in the same period in 2021 as a result of investments we made to expand our facilities;

● We incurred $15.1 million in disposable costs compared to $11.9 million for the same period in 2021 as we continue to expand our activities to support the development of our product candidates;

● We incurred $12.1 million in share-based compensation expenses in the nine months ended September 30, 2022, compared to $9.2 million for the same period in 2021.

The increase was primarily a result of an increase in awards granted, including those to newly recruited personnel;

● We incurred $4.7 million of other expenses for the six months ended June 30, 2022, compared to $3.0 million for the same period in 2021;

● We incurred $2.0 million of expenses in the six months ended June 30, 2022 related to an increase in the fair value of contingent consideration associated with the acquisition of Corlieve Therapeutics SAS (“Corlieve”), compared to nil for the same period in 2021.

● We incurred $7.5 million of expenses in the nine months ended September 30, 2022 related to an increase in the fair value of contingent consideration associated with the acquisition of Corlieve Therapeutics SAS (“Corlieve”), compared to $0.4 million for the same period in 2021;

● We incurred $6.9 million of other expenses for the nine months ended September 30, 2022, compared to $4.5 million for the same period in 2021.

Selling, general and administrative expenses

Selling, general and administrative expenses for the six months ended June 30, 2022 were $23.5 million, compared to $30.3 million for the same period in 2021.

● We incurred $9.5 million in personnel and contractor related expenses in the six months ended June 30, 2022, compared to $8.1 million in the same period in 2021.

The increase in the six months ended June 30, 2022, relates to the recruitment of personnel;

● We incurred $6.2 million in share-based compensation expenses in the six months ended June 30, 2022, compared to $6.8 million in the same period in 2021;

● We incurred $2.7 million in professional fees in the six months ended June 30, 2022, compared to $5.0 million in the same period in 2021.

Selling, general and administrative expenses for the nine months ended September 30, 2022 were $36.8 million, compared to $42.3 million for the same period in 2021.

● We incurred $14.8 million in personnel and contractor related expenses in the nine months ended September 30, 2022, compared to $11.8 million in the same period in 2021.

The increase in the nine months ended September 30, 2022, relates to the recruitment of personnel;

● We incurred $9.9 million in share-based compensation expenses in the nine months ended September 30, 2022, compared to $9.5 million in the same period in 2021;

● We incurred $4.3 million in professional fees in the nine months ended September 30, 2022, compared to $6.5 million in the same period in 2021.

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

The decrease from the prior period is primarily related to a decrease in professional fees related to the licensing transaction with CSL Behring;

● We incurred nil financial advisory fees in relation to our licensing transaction with CSL Behring in the six months ended June 30, 2022, compared to $4.5 million in the same period in 2021.

● We incurred nil financial advisory fees in relation to our licensing transaction with CSL Behring in the nine months ended September 30, 2022, compared to $4.5 million in the same period in 2021.

Other items, net

In the six months ended June 30, 2022, we recognized $0.3 million in other income in relation to the equity stake received in VectorY B.V.

In the nine months ended September 30, 2022, we recognized $0.3 million in other income in relation to the equity stake received in VectorY B.V.

in conjunction with a settlement agreement entered into in April 2021 compared to $3.0 million such income for the same period in 2021.

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

CARES Act in the six months ended June 30, 2022, compared to $1.3 million in other income for the same period in 2021.

We recognized $2.7 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, 2022, compared to $3.0 million for the same period in 2021.

CARES Act in the nine months ended September 30, 2022, compared to $2.6 million in other income for the same period in 2021.

We recognized $3.9 million in other income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the nine months ended September 30, 2022, compared to $3.0 million for the same period in 2021.

Other income also includes income from subleasing of a portion of our Amsterdam facility.

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Other non-operating items, net

Our other non-operating items, net, for the six months ended June 30, 2022 and 2021 were as follows:

Six months ended June 30,

Our other non-operating items, net, for the nine months ended September 30, 2022 and 2021 were as follows:

Nine months ended September 30,

(in thousands)

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

We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $28.0 million during the six months ended June 30, 2022, compared to a net gain of $11.2 million during the same period in 2021.

We recognize fair value changes related to the derivative financial liability related to a contingent payment due to BMS upon a change of control as described elsewhere in this Quarterly Report on Form 10-Q.

We recognized a net foreign currency gain, related to our borrowings from Hercules and our cash and cash equivalents as well as loans between entities within the uniQure group, of $42.3 million during the nine months ended September 30, 2022, compared to a net gain of $21.6 million during the same period in 2021.

Income tax benefit / (expense)

We recognized $0.9 million of deferred tax benefit in the six months ended June 30, 2022, and $3.5 million of deferred tax expense for the same period in 2021.

We recognized $1.3 million of deferred tax benefit in the nine months ended September 30, 2022, and $3.6 million of deferred tax expense for the same period in 2021.

Financial Position, Liquidity and Capital Resources

As of June 30, 2022, we had cash, cash equivalents and restricted cash of $503.7 million.

As of September 30, 2022, we had cash, cash equivalents and restricted cash of $443.4 million.

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

We believe that our cash and cash equivalents will fund our operations into the first half of 2025 assuming the achievement of $175.0 million of first commercial sales milestones under the CSL Behring Agreement.

We believe that our cash and cash equivalents will fund our operations into the first half of 2025 assuming the achievement of $100.0 million of first commercial sale milestone in the United States and $75.0 million of first commercial sale milestone in any of the five contractually defined European countries as of or prior to July 1, 2023 under the CSL Behring Agreement.

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

As of June 30, 2022, we had an outstanding loan amount owed to Hercules Capital, Inc.

As of September 30, 2022, we had an outstanding loan amount owed to Hercules Capital, Inc.

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

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

As of June 30, 2022, we had fixed lease payment obligations of $60.3 million, with $7.0 million payable within 12 months.

As of September 30, 2022, we had fixed lease payment obligations of $58.6 million, with $8.0 million payable within 12 months.

Commitments related to Corlieve acquisition (nominal amounts)

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The commitments include payments related to post-acquisition services that we agreed to as part of the Stock Purchase Agreement between the Company and Corlieve (“SPA”).

As of June 30, 2022, our commitment amounts include up to $43.1 million in potential milestone payments through Phase I/II development and $167.2 million in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the United States and European Union.

As of September 30, 2022, our commitment amounts include up to $40.4 million in potential milestone payments through Phase I/II development and $156.8 million in potential milestone payments associated with Phase III development and the approvals of AMT-260 in the United States 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, 2022, of $1.05/€1.00.

As of June 30, 2022, we expect these obligations will become payable between 2023 and 2031.

These payments are owed in Euro and have been translated at the foreign exchange rate as of September 30, 2022, of $0.98/€1.00.

As of September 30, 2022, we expect these obligations will become payable between 2023 and 2031.

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

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

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

Six months ended June 30,

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

Nine months ended September 30,

(in thousands)

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We continue to incur losses in the current period.

We recorded a net loss of $39.1 million and $85.7 million in the three and six months ended June 30, 2022, compared to a net income of $399.5 million and $357.9 million during the same periods in 2021.

As of June 30, 2022, we had an accumulated deficit of $540.9 million.

We recorded a net loss of $47.9 million and $133.6 million in the three and nine months ended September 30, 2022, compared to a net loss of $36.5 million and net income of $321.4 million during the same periods in 2021.

As of September 30, 2022, we had an accumulated deficit of $588.7 million.

Sources of liquidity

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Net Cash used in operating activities

Net cash used in operating activities was $41.4 million for the six months ended June 30, 2022 and consisted of net loss of $85.7 million adjusted for non-cash items, including depreciation and amortization expense of $4.1 million, share-based compensation expense of $14.7 million, changes in the fair value of contingent consideration and the derivative financial liability of $1.4 million, unrealized foreign exchange gains of $24.5 million and a change in deferred taxes of $0.9 million.

Net cash used in operating activities was $90.4 million for the nine months ended September 30, 2022 and consisted of net loss of $133.6 million adjusted for non-cash items, including depreciation and amortization expense of $6.2 million, share-based compensation expense of $22.3 million, changes in the fair value of contingent consideration and the derivative financial liability of $6.9 million, unrealized foreign exchange gains of $36.4 million and a change in deferred taxes of $1.3 million.

Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $46.1 million.

There was a net decrease in accounts receivable and contract asset, prepaid expenses, and other current assets and receivables of $42.0 million, primarily related to the collection of $55.0 million of the contract asset related to CSL milestones of $55.0 million in March 2022 and April 2022.

There was an increase in inventory balances of $2.9 million related to the production of etranacogene dezaparvovec under the CSL Behring Agreement.

There was an increase in inventories of $4.1 million related to the production of etranacogene dezaparvovec under the CSL Behring Agreement.

These changes also relate to a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $8.2 million, primarily related to an increase in accounts payable.

Net cash generated from operating activities was $375.2 million for the six months ended June 30, 2021 and consisted of a net income of $357.9 million adjusted for non-cash items, including depreciation and amortization expense of $3.7 million, share-based compensation expense of $12.8 million, unrealized foreign exchange gains, net of $12.3 million, deferred tax expense of $3.5 million and other non-cash items of $2.9 million.

Net cash used in operating activities also included favorable changes in operating assets and liabilities of $12.6 million.

These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $3.4 million primarily related to the increase in a contract asset and receivable recorded for CSL expenses to be reimbursed as well as an increase in various prepayments for the year and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $15.9 million primarily related to an increase in amounts to be paid to licensors upon the CSL Behring Closing.

Net cash generated from operating activities was $335.0 million for the nine months ended September 30, 2021 and consisted of a net income of $321.4 million adjusted for non-cash items, including depreciation and amortization expense of $5.4 million, share-based compensation expense of $18.7 million, a change in fair value of contingent consideration of $0.4 million, unrealized foreign exchange gains, net of $23.2 million, deferred tax expense of $3.6 million and other non-cash items of $2.9 million.

Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $11.6 million.

These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.0 million primarily related to various prepayments and a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $13.6 million primarily related to an increase in amounts to be paid to licensors upon the CSL Behring Closing and an increase in various other accruals for services provided by vendors and personnel accruals.

Net income primarily consisted of $462.4 million license revenue recognized on Closing.

Net cash used in investing activities

In the six months ended June 30, 2022, we used $9.5 million in our investing activities compared to $6.2 million for the same period in 2021.

Six months ended June 30,

In the nine months ended September 30, 2022, we used $14.5 million in our investing activities compared to $63.8 million for the same period in 2021.

Nine months ended September 30,

(in thousands)

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Build out of Lexington site

Acquisition of remaining outstanding ordinary shares related to Corlieve acquisition

Acquisition of Corlieve, net of cash acquired

Total investments

The build out of the Amsterdam site and Lexington site consumed $6.4 million and $2.2 million cash respectively during the six months ended June 30, 2022, compared to $4.5 million and $1.7 million for the same period in 2021.

The increase is a result of additional investments to support the expansions of our research and development activities.

The build out of the Amsterdam site and Lexington site consumed $8.3 million and $4.4 million cash respectively during the nine months ended September 30, 2022, compared to $9.9 million and $4.0 million for the same period in 2021.

We paid EUR 42.1 million ($49.9 million), net of EUR 2.8 million ($3.4 million) of cash acquired, during the nine months ended September 30, 2021 to acquire 97.7% of the outstanding shares of Corlieve on July 30, 2021.

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

Net cash generated from financing activities

In the six months ended June 30, 2022, we generated $0.5 million in our financing activities compared to $65.1 million for the same period in 2021.

Six months ended June 30,

In the nine months ended September 30, 2022, we generated $0.8 million in our financing activities compared to $65.0 million for the same period in 2021.

Nine months ended September 30,

(in thousands)

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Proceeds from issuance of ordinary shares, net of issuance costs

Repayment of debt assumed through Corlieve Transaction

Net cash generated from financing activities

During the six months ended June 30, 2022, we received $0.5 million from the exercise of options to purchase ordinary shares in relation to our share incentive plans compared to $1.0 million for the same period in 2021.

During the nine months ended September 30, 2022, we received $0.8 million from the exercise of options to purchase ordinary shares in relation to our share incentive plans compared to $2.0 million for the same period in 2021.

In January 2021, we received $34.6 million net proceeds from the Hercules 2021 Amended Facility (nil for same period in 2022).

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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