What changed in this filing

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Item 2. Management’s Discussion and Analysis

2022-05-02 compared with 2021-10-25 · 164 added, 476 removed, 151 unchanged (81% of the section changed)

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

This MD&A is provided as a supplement to, and should be read in conjunction with, our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the Securities and Exchange Commission ( the “SEC”) on March 1, 2021 .

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

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

We are advancing a focused pipeline of innovative gene therapies, including product candidates for the treatment of hemophilia B, which effective May 6, 2021, we licensed to CSL Behring pursuant to the CSL Behring Agreement (as defined below), and Huntington’s disease.

We are advancing a pipeline of innovative gene therapies, including product candidates for the treatment of Huntington’s disease, hemophilia B, temporal lobe epilepsy and Fabry disease.

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

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Below is a summary of our recent significant business developments:

Acquisition of Corlieve Therapeutics

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

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

We are contractually required to acquire the remaining outstanding ordinary shares in February 2022 following the expiration of a minimum holding period (“Mandatorily redeemable shares”).

We recorded a liability related to these Mandatorily redeemable shares for an amount of EUR 0.7 million ($0.9 million) as of the Acquisition Date.

We financed the transaction from cash on hand.

Following its formation in November 2019, Corlieve obtained exclusive licenses to certain patents from two French research institutions that continue to collaborate with Corlieve and us.

At the same time, Corlieve also obtained an exclusive license from Regenxbio Inc.

for the use of AAV9 to deliver any sequence that affects the expression of the Glutamate inotropic receptor kainate type subunit 2 (“GRIK 2”) gene sequence in humans.

Corlieve and Regenxbio Inc.

simultaneously entered into a collaboration plan related to agreed joint preclinical research and development activities.

At the Acquisition Date, Corlieve and its Swiss subsidiary, Corlieve Therapeutics AG, employed seven employees.

Corlieve’s gene therapy program, AMT-260, employs miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy (“TLE”).

TLE affects approximately 1.3 million people in the U.S.

and Europe alone, of which approximately 0.8 million patients are unable to adequately control acute seizures with currently approved anti-epileptic therapies.

Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life.

In addition, Corlieve’s former and remaining shareholders are eligible to receive up to EUR 35.8 million ($42.5 million) upon the achievement of development milestones through Phase I/II and EUR 143.1 million ($169.9 million) upon the achievement of milestones associated with Phase III development and obtaining approval to commercialize AMT-260 in the United States of America and the European Union.

We may elect to pay up to 25% of such milestone payments through the issuance of our ordinary shares.

We recorded a EUR 20.2 million ($24.0 million) liability related to these contingent consideration payments.

We primarily allocated the total consideration of EUR 65.8 million ($78.1 million) consisting of the cash paid upon the Acquisition Date, the payment we owe in relation to the Mandatorily redeemable shares and the contingent consideration payments to the identifiable intangible assets related to the in-process research and development efforts of AMT-260 (“IPR&D intangible asset”).

The IPR&D intangible asset’s provisional fair value has been determined at EUR 53.3 million ($63.3 million) as of the Acquisition Date.

We recognized a EUR 13.3 million ($15.8 million) deferred tax liability in relation to this IPR&D intangible asset.

We allocated the excess of EUR 23.8 million ($28.2 million) of the total consideration over the net assets acquired to goodwill.

CSL Behring commercialization and license agreement

On June 24, 2020, (the “Signing Date”), uniQure biopharma B.V., a wholly-owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (as amended, the “CSL Behring Agreement”) with CSL Behring LLC (“CSL Behring”) pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, our investigational gene therapy for patients with hemophilia B (the “Product”).

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 on May 5, 2021.

CSL Behring is responsible for the development and commercialization of the Product.

We agreed to complete the validation of the current manufacturing process as well as to the development and validation of a next generation manufacturing process.

We will be entitled to receive a development milestone payment if we complete these activities in accordance with an agreed development plan and timeline.

CSL Behring will also be reimbursing us for our services to facilitate the completion of the HOPE-B clinical trial.

On the Signing Date, we and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we will supply the Product to CSL Behring at an agreed-upon price commensurate with the stand-alone selling price (“SSP”).

We will be responsible to supply the Product until such time that these capabilities may be transferred to CSL Behring or its designated contract manufacturing organization.

We recorded $462.4 million, including a $450.0 million upfront cash payment, following the closing of the CSL Behring Agreement as license revenue in the nine months ended September 30, 2021 (nil for three months ended September 30, 2021).

We are eligible to receive more than $0.3 billion in regulatory, development, and first commercial sale milestones, $1.3 billion in additional commercial milestones, and tiered double-digit royalties of up to a low-twenties percentage of net product sales arising from the collaboration.

Upon closing, we contractually owed to our licensors $16.7 million of the upfront payment received from CSL Behring.

The payments we received significantly increased our cash and cash equivalents position to $578.5 million as of September 30, 2021.

This is expected to fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules Growth Capital, Inc.

under our term loan facilities by 2023).

We expect that if we were to achieve the $0.3 billion regulatory, first commercial sales and development milestone payments, our runway would be further extended by 12 to 18 months.

Hemophilia B program – Etranacogene dezaparvovec (AMT-061)

Etranacogene dezaparvovec is our lead gene therapy candidate and includes an AAV serotype 5 (“AAV-5”) vector incorporating the functional human Factor IX (“FIX”) Padua variant.

We are currently conducting a pivotal study in patients with severe and moderately-severe hemophilia B.

In September 2021, we completed the last patient’s 78-week follow-up visit in the HOPE-B Phase III pivotal study, which enrolled a total of 54 patients.

On June 22, 2021, we presented new data from the HOPE-B study related to the 52-week period post-infusion.

Data showed that participants continued to demonstrate durable, sustained increases in FIX activity at 52-weeks post-infusion with a mean FIX activity of 41.5 percent of normal, as measured by a one-stage APTT-based clotting assay, compared to a mean FIX activity of 39.0 percent of normal at 26-weeks of follow-up.

During the 52-week period, a single dose of etranacogene dezaparvovec significantly reduced the annualized rate of bleeding requiring treatment by 80 percent from a prospectively collected 3.39 at baseline to 0.68 bleeding episodes per year (p-value <0.0001).

The annualized rate of spontaneous bleeding requiring treatment was also significantly reduced by 85 percent from a prospectively collected 1.16 at baseline to 0.18 bleeds per year during the 52-week period (p-value <0.0001).

Usage of FIX replacement therapy (IU/year and infusions/year) in all patients declined 96 percent during the 52-week period, with 52 of 54 patients (96 percent) successfully discontinuing their prophylactic FIX infusions.

As previously announced, of the two non-responders, one patient only received a partial dose (less than 10 percent of the dosage) due to an infusion reaction and a second patient had an unusually high pre-existing NAb titer of 3,212, which is expected in less than 1 percent of the general population.

All patients completed their 78-week follow-up visits by the end of September 2021, and we expect a Biologics License Application (“BLA”) to be submitted to the U.S.

Food and Drug Administration (“FDA”) and a marketing authorization application (“MAA”) to be submitted to EMA in the first half of 2022.

On December 21, 2020, our clinical trials of etranacogene dezaparvovec had been placed on clinical hold by the FDA following a preliminary diagnosis of hepatocellular carcinoma (“HCC”), a form of liver cancer, in one patient in the HOPE-B trial who was treated with etranacogene dezaparvovec.

On March 26, 2021, we submitted to the FDA the results of a comprehensive investigation that found it is highly unlikely the HCC was caused by etranacogene dezaparvovec.

On April 23, 2021, the FDA informed us that the clinical hold on our hemophilia B gene therapy program was removed after determining that we had satisfactorily addressed all issues identified.

Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the FDA and access to the current priority medicines (“PRIME”) initiative by the EMA.

Huntington’s disease program (AMT-130)

AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease.

AMT-130 utilizes our proprietary, gene-silencing miQURE platform and incorporates an AAV vector carrying a miRNA specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.

AMT-130 has received Orphan Drug and Fast Track designations from the FDA and Orphan Medicinal Product Designation from the EMA.

On April 5, 2021, we announced the completion of enrollment of all 10 patients in the first dose cohort of our Phase I/II clinical trial of AMT-130.

The study is a double-blinded, randomized, and controlled clinical trial being conducted in the United States in a total of 26 patients.

On May 27, 2021, we announced that the study’s independent Data Safety Monitoring Board (“DSMB”) recommended we continue patient dosing after reviewing safety data from all patients in the first dose cohort.

On each of June 16 and August 30, 2021, we completed two patient procedures in the second, higher-dose cohort, which will include a total of 16 patients.

In the study to date, a total of 14 blinded patient procedures have been completed across both dose cohorts, including eight patients treated with AMT-130 and six patients receiving imitation surgery.

Also on April 5, 2021, we announced plans for a Phase Ib/II clinical trial in Europe which, with enrollment beginning before the end of this year.

This open-label study will enroll 15 patients with early manifest Huntington’s disease across two dose cohorts.

Together with the U.S.

study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible.

European Society of Gene and Cell Therapy (“ESGCT”)

In October 2021, the Company presented preclinical data related to four programs at the ESGCT 28 th Annual Meeting:

● AMT-191, a gene-therapy candidate for the treatment of Fabry disease, confirming efficiency and cross correction in a Fabry mouse model, with increased gamma-linolenic acid in the liver, kidney, heart, and brain and normalized lysoglobotriaosylceramide3 levels in main target organs;

● AMT-230, an AAV gene therapy candidate for Parkinson’s Disease, reduces alpha synuclein in dopaminergic neurons and rescues motor phenotypes in a genetic model of Parkinson’s Disease;

● AMT-161, an AAV gene-therapy for the treatment of Amyotrophic lateral sclerosis, reduces the expression of C9ORF72 in human induced pluripotent stem cells derived neurons, reduces C9ORF72-containing ribonucleic acid (“RNA”) foci in a model of the disease and is distributed to relevant regions of the spinal cord and brain when expressed in non-human primates;

● AMT-260, a gene therapy candidate for temporal lobe epilepsy, reduces the expression of GluK2 in cortical neurons, reduces epileptiform activity and hyperlocomotion in a preclinical model of epilepsy and blocks epileptiform discharges in organotypic slices from patients with TLE.

AMT-130 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 Phase I/II clinical trial for AMT-130 in the U.S.

and a Phase Ib/II study in the EU.

Together, these studies are intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible.

AMT-130 has received Orphan Drug and Fast Track designations from the U.S.

Food and Drug Administration (“FDA”) and Orphan Medicinal Product Designation from the European Medicines Agency (“EMA”).

In February 2022, the first two patients were dosed in our European Phase Ib/II study of AMT-130.

The open-label study will enroll a total of 15 patients in two dose cohorts.

The first and second dose cohorts will include 6 patients and 9 patients, respectively.

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

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

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

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

The Phase I/II clinical 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 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.

CSL Behring collaboration

On June 24, 2020, (the “Signing Date”), uniQure biopharma B.V., a wholly owned subsidiary of uniQure N.V., entered into a commercialization and license agreement (the “CSL Behring Agreement”) with CSL Behring pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, the investigational gene therapy for patients with hemophilia B (the “Product”).

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 March 2022, CSL Behring submitted certain global regulatory submissions for etranacogene dezaparvovec.

On March 28, 2022, CSL Behring announced that the EMA accepted one of the global regulatory submissions - the Marketing Authorization Application (“MAA”) - under its accelerated assessment procedure.

As of March 31, 2022, we received $20.0 million of the $55.0 million owed to us by CSL Behring related to the global regulatory submissions.

The remaining $35.0 million was received in April 2022.

Preclinical programs

On May 2, 2022 we announced that we will be presenting certain preclinical findings on our gene therapy candidates for Huntington’s disease, 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 May 2022.

Covid pandemic

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

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

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.

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

Accordingly, we had mandated a work-from-home policy since March 2020 for all non-essential employees at our Amsterdam and Lexington facilities and had implemented additional protocols for our essential employees.

Effective May 29, 2021, Massachusetts lifted all industry restrictions, with the exceptions of remaining face-covering requirements for all public and private transportation systems and facilities housing vulnerable populations, and capacity has been increased to 100% for all industries.

Furthermore, the state of emergency was lifted on June 15, 2021.

Implementation of adequate cleaning and hygiene protocols are still encouraged.

We have implemented a mandatory Covid PCR testing protocol in our Lexington facility effective February 2021 that requires employees to have tested negative for Covid prior to entering the facility.

Effective April 2021, we implemented a policy whereby no employee or contractor may enter the Lexington facility unless they are either:

(i) fully vaccinated;

or (ii) have taken an approved test for Covid and obtained a negative result, and we expect that policy will remain in effect at least until December 31, 2021.

Also, in our Lexington facility, allowed occupancy has been increased to 100% in line with the permitted capacity guidelines and all employees are allowed to work at the Lexington facility subject to social distancing protocols, and a daily health screening and compliance with our Covid PCR testing policy for unvaccinated employees.

All employees that are not essential employees, however, remain able to work remotely until December 31, 2021, and we have been operating our Lexington facility significantly below 100% capacity.

On September 14, 2021, the Dutch government announced updated Covid-related measures effective as of September 25, 2021.

These updated measures require employees to work from home if possible and grant employers discretion with respect to the division of time spent between working from home or on site.

The mandatory rule to maintain 1.5 meters distance from others is also amended to a strong recommendation to maintain 1.5 meters distance from other people.

From November 1, 2021, employees are expected to be required at our Amsterdam office at least one day a week under our new remote working policy.

The broader implications of Covid, including the implications from the various variants, on our results of operations and overall financial performance remain uncertain.

Effective March 1, 2022, we have implemented our base Covid-19 Protocol within the Lexington facility which requires only unvaccinated employees to wear facemasks and apply social distancing at the office.

All non-essential Lexington employees are allowed to return to the office as space at the office allows and there is no longer a limitation on in-person meetings, although meetings organizers should still provide remote options for employees.

This protocol will remain in place until July 1, 2022, unless extended or otherwise amended.

Effective February 28, 2022, the Amsterdam location 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.

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

In February 2021, we commenced the expansion of our Amsterdam site to build additional laboratories to support the expansions of our research and development activities as well the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.

In May 2021, we entered into a sublease agreement to let an additional approximately 1,080 square meters of office space to accommodate the hiring of additional full-time employees.

The lease expires in October 2028 and includes an option to break the lease on October 31, 2023.

On October 21, 2021, we held an Extraordinary General Meeting of our shareholders and Ms.

Jacques was appointed to the Board of Directors (the “Board”).

Jacques will also serve as a member of the Audit Committee of the Board effective as of October 21, 2021.

Russian-Ukrainian war

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

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

Intellectual Property

On May 11, 2021, Pfizer, Inc.

filed three petitions in the United States Patent & Trademark Office (“USPTO”) seeking Inter Partes Review of U.S.

filed three petitions at the USPTO seeking Inter Partes Review of U.S.

9,982,248 (the “‘248 Patent”) and 10,465,180 (the “‘180 Patent” and together with the ‘248 Patent, the “Patents”).

The petitions collectively seek to invalidate all claims of the Patents, which relate to the expression of factor IX protein using a modified “Padua” polypeptide having a leucine in position 338.

The petitions collectively seek to invalidate all claims of the Patents.

In August 2021, we filed our responses asking the USPTO to deny institution of the IPR proceedings.

We are awaiting a decision by the USPTO on whether to institute these proceedings.

On November 17, 2021, the PTAB issued decisions granting institution on all three IPR proceedings.

Our response to the petition was filed on March 3, 2022.

Pfizer’s reply to our response is due not later than June 17, 2022.

Financial Overview

Key components of our results of operations include the following:

Three months ended September 30,

Nine months ended September 30,

(in thousands)

Three months ended March 31,

(in thousands)

Total revenues

Cost of contract revenue

Research and development expenses

Selling, general and administrative expenses

Net (loss) / income

As of September 30, 2021 and December 31, 2020, we had cash and cash equivalents of $578.5 million and $244.9 million, respectively.

We had a net loss of $36.5 million and net income of $321.4 million in the three and nine months ended September 30, 2021, respectively, compared to a net loss of $53.8 million and $124.3 million for the same periods in 2020.

As of September 30, 2021 and December 31, 2020, we had accumulated deficits of $463.4 million and $784.7 million, respectively.

We recorded a net loss in the three months ended September 30, 2021, and we recorded net income in the nine months ended September 30, 2021, as a result of closing the CSL Behring transaction on May 6, 2021.

As of March 31, 2022 and December 31, 2021, we had cash and cash equivalents of $524.9 million and $556.3 million, respectively.

We had a net loss of $46.7 million in the three months ended March 31, 2022 compared to a net loss of $41.6 million for the same period in 2021.

As of March 31, 2022 and December 31, 2021, we had accumulated deficits of $501.8 million and $455.1 million, respectively.

We anticipate that our expenses will increase substantially as we:

● advance the clinical development of AMT-130 for our Huntington’s disease gene therapy program;

● advance multiple research programs related to gene therapy candidates targeting liver-directed and central nervous system (“CNS”) diseases;

● advance multiple research programs related to gene therapy candidates targeting liver-directed and CNS diseases;

● continue to expand our employee base to support research and development, as well as general and administrative functions;

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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 income/loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures.

On an ongoing basis, we evaluate our assumptions, estimates and judgments, including those related to what we believe to be our critical accounting policies:

● the treatment of the CSL Behring Agreement,

● our arrangements with Bristol-Myers Squibb (“BMS”), including the amended collaboration and license agreement that we entered into with BMS in December 2020 (the “amended BMS CLA”);

● the treatment of the Corlieve Transaction;

● share-based payments;

● corporate income taxes related to valuation allowance;

● accounting for operating leases under ASC 842.

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.

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In making estimates and judgments, management employs critical accounting policies.

During the nine months ended September 30, 2021, there were no material changes to our critical accounting policies as reported in our Annual Report on Form 10-K for the year ended December 31, 2020, which was filed with the SEC on March 1, 2021 , with the exceptions of determining the accounting treatment in accordance with ASC 805 related to the Corlieve Transaction and determining the accounting treatment in accordance with ASC 606 related to the CSL Behring Agreement.

CSL Behring Agreement

The preparation of our consolidated financial statements for the three- and nine- month period ended September 30, 2021, required us to analyze the accounting treatment of the CSL Behring Agreement.

The effectiveness of the transaction had been contingent on completion of review under antitrust laws in the United States, Australia, and the United Kingdom.

The transaction had previously been cleared by the Australian and United Kingdom antitrust authorities and became fully effective on May 6, 2021 (“Closing”), after the waiting period under the HSR Act expired on May 5, 2021.

We identified two material performance obligations related to the CSL Behring Agreement:

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

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

We continued to develop the Product between the Signing Date and Closing and performed certain reimbursable activities to fulfill the transfer of the global rights (“Additional Covenants” and together with the License the “License Sale”).

We determined that the fixed upfront payment of $450.0 million and the $12.4 million that we received in relation to the Additional Covenants should be allocated to the License Sale.

In addition, we concluded that variable milestone payments, sales milestone payments and royalties should be allocated to the License Sale performance obligation as well.

We determined that the sale of the License was completed on May 6, 2021, when we transferred the License and CSL Behring assumed full responsibility for the development and commercialization of the Product.

At Closing we evaluated the amounts of potential payments and the likelihood that the payments will be received.

We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.

Since we cannot control the achievement of regulatory and first commercial sales milestones, we concluded that the potential payments are constrained as of Closing.

We determined that we would recognize revenue related to these payments, only to the extent that it becomes probable that no significant reversal of recognized cumulative revenue will occur thereafter.

We will include payments related to sales milestones in the transaction price when their achievement becomes probable, and we will include royalties on the sale of Product once these have been earned.

We recognized nil and $462.4 million of revenues related to the License Sale in the three and nine months ended September 30, 2021, respectively.

We determined that the variable milestone payment related to Manufacturing Development Services should be allocated to the Manufacturing Development performance obligation.

We concluded that this milestone payment represents the SSP of the services based on the estimated cost of providing the services including a reasonable margin.

The services related to Manufacturing Development will be provided between Closing and the completion of an agreed manufacturing development plan.

The variable consideration will be reduced if we do not complete the development by pre-agreed dates.

We utilized the most likely amount method to estimate the variable consideration to be included in the transaction price.

Completion of Manufacturing Development is partially dependent on the timing of regulatory submissions by CSL Behring as well as regulatory approvals of the developed manufacturing processes.

Since we cannot control the timing or outcome of any regulatory decisions, we concluded that we would recognize revenue related to this payment when it becomes probable that the milestone has been achieved.

We did not recognize any revenue related to Manufacturing Development during the three and nine months ended September 30, 2021.

We recognized $0.5 million and $0.9 million of collaboration revenue in the three and nine months ended September 30, 2021, respectively, compared to nil in the same periods in the prior year.

We generate such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring.

CSL Behring may request additional development services or to request that we support the transfer of manufacturing to a party designated by CSL Behring.

These collaboration services will be reimbursed at the pre-agreed full-time-employee rate (“FTE-rate”).

We concluded that these rights at Closing do not represent material rights.

We also analyzed the impact of Closing on our net deferred tax asset in the Netherlands.

Prior to Closing we had determined that the potential Closing would represent insufficient positive evidence to overcome the significant negative evidence of having been loss-making during the past three years.

Accordingly, we had therefore recorded a full valuation allowance related to our net deferred tax assets.

We determined that in accordance with Dutch tax law the upfront payment as well as payments related to the Additional Covenants, to the extend such activities were conducted in the 12 months ended December 31, 2020, can more likely than not be included in the Dutch tax return for the 12-month period ended December 31, 2020.

Including this income in our 2020 tax position will consume substantially all of our Dutch net operating losses for the years 2011 to 2018.

We treated the adjustment to our 2020 filing position resulting in the release of the valuation allowance related to our Dutch net operating losses for the years 2011 to 2018 as a discrete event in the three months ended June 30, 2021.

We allocated part of the release to the tax benefit of share issuance cost incurred in 2014, 2015, 2017 and 2018.

This resulted in an increase of additional paid- in capital as well as the recognition of deferred tax expenses of $3.0 million during the nine months ended September 30, 2021 (nil for three months ended September 30, 2021).

Based on changes to Dutch tax law enacted in June 2021 our remaining net operating tax loss related to 2019 can be carried forward indefinitely.

We continue to expect to incur tax losses for the foreseeable future, including 2021.

As such, we retain our full valuation allowance related to the remaining net deferred asset as of September 30, 2021.

Corlieve Transaction

The preparation of our consolidated financial statements for the three- and nine- month period ended September 30, 2021, required us to analyze the accounting treatment for the Corlieve Transaction.

We evaluated whether or not the Corlieve Transaction should be accounted for as a business combination or asset acquisition by first applying a screen test to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets.

Based on the provisional fair values of the gross assets acquired, we determined the screen test was not met.

We further analyzed whether or not the acquired inputs and processes that have the ability to create outputs would meet the definition of a business.

Significant judgment is required in the application of the screen test to determine whether an acquisition is a business combination or an acquisition of assets.

We performed the screen test using provisional fair values.

The finalization of the valuation might result in a different outcome of the screen test.

In this case, we would be required to account for the transaction as an asset acquisition.

In accordance with ASC 805, we in this case, among others, would not record any goodwill and expense all identifiable intangible assets without alternative future use.

Accounting for business combinations requires management to make significant estimates and assumptions as of the Acquisition Date which are inherently uncertain.

We identified various licenses that combined with the results of the research and development activities conducted in relation to AMT-260 since formation of Corlieve in 2019 constitute an IPR&D intangible asset.

The IPR&D intangible asset was measured at its fair value as of the Acquisition Date.

We calculated the fair value of the IPR&D intangible asset using a present value model based on expected cash flows.

Estimating the amounts and timing of cash flows required to complete the development of AMT-260 as well as net sales, cost of goods sold, and sales and marketing costs involved considerable judgment and uncertainty.

The expected cash flows are materially impacted by the probability of successfully completing the various stages of development (i.e., dosing of first patient in clinical trial, advancing into late-stage clinical development and obtaining approval to commercialize a product candidate) as well as the weighted average cost of capital of 10.4% used to discount the expected cash flows.

Based on all of these assumptions, we estimated the fair value of the IPR&D intangible asset at EUR 53.3 million ($63.3 million) as of the Acquisition Date.

Unanticipated events and circumstances may occur that could affect either the accuracy or validity of such assumptions, estimates or actual results.

The IPR&D intangible asset is accounted for as indefinite-lived intangible asset and is measured at the fair value of the incomplete research project acquired as of the Acquisition Date.

The IPR&D intangible asset is considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts and is not amortized.

If and when development is complete, which generally occurs when regulatory approval to market a product is obtained, the associated assets would be deemed finite-lived and would then be amortized based on an estimate of its remaining useful life.

In case of abandonment, the IPR&D intangible asset would be written-off.

In accordance with ASC 350, Intangibles – Goodwill and Other, we test indefinite-lived intangible assets for impairment on an annual basis and between annual tests if we become aware of any events occurring or changes in circumstances that would indicate the fair value of the asset is below its carrying amount.

As of September 30, 2021, no impairment trigger of indefinite-lived intangible assets was identified.

Goodwill represents the excess of the consideration transferred over the fair value of assets acquired and liabilities assumed in a business combination.

Goodwill is not amortized but is evaluated for impairment on an annual basis and between annual tests if we become aware of any events occurring or changes in circumstances that would more likely than not reduce the fair value of the reporting unit below its carrying amount.

As of September 30, 2021, no impairment trigger of goodwill was identified.

We recorded contingent consideration related to amounts potentially payable to Corlieve’s former shareholders.

The amounts payable in accordance with the SPA are contingent upon realization of certain milestones associated with the TLE research program.

Contingent consideration is measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.

Changes in contingent consideration can result from changes in the assumed achievement and timing of estimated milestones, the estimated timing in which milestones are achieved and the discount rate used to estimate the fair value of the liability.

In the absence of new information, changes in fair value reflect the passage of time towards achievement of the milestones.

We recognize revenue when the customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services.

To determine revenue recognition for arrangements that we determine are within the scope of ASC 606, we perform the following five steps:

(i) identify the contract with the customer;

(ii) identify the performance obligations in the contract;

(iii) determine the transaction price;

(iv) allocate the transaction price to the performance obligations in the contract;

and (v) recognize revenue when (or as) we satisfy a performance obligation.

We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer.

At contract inception, once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations and assess whether each promised good or service is distinct.

We then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.

We estimate the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract.

The consideration may include fixed consideration or variable consideration.

At inception of each arrangement that includes variable consideration, we evaluate the amount of potential payments and the likelihood that the payments will be received.

We utilize either the most likely amount method or expected amount method to estimate the amount expected to be received based on which method best predicts the amount expected to be received.

The amount of variable consideration included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur in a future period.

We allocate the transaction price based on the estimated SSP of each of the performance obligations.

Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts that we expect to receive for the satisfaction of each performance obligation.

We recognize revenue when or as control of the performance obligation transfers to the customer.

For performance obligations which consist of licenses and other promises, we utilize judgement to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time.

Following the Closing of the CSL Behring agreement, we recognize license revenue related to the License Sale of the global rights to the Product.

We determined that our performance obligation related to the License Sale was satisfied on Closing and recognized $462.4 million in license revenue related to a $450.0 million upfront payment and $12.4 million of payments in relation to Additional Covenants.

We will recognize additional license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of Product have been earned.

We recognize collaboration revenues associated with Manufacturing Development.

We will recognize collaboration revenue related to a contractual development milestone when it becomes probable the milestone will be achieved.

We recognize collaboration revenues associated with Development Services that will be reimbursed by CSL Behring relating to clinical development activities.

These services are provided by our employees.

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

In May 2015, we and Bristol-Myers Squibb (“BMS”) entered into a collaboration and license agreement and various related agreements with BMS (“BMS CLA”).

We have recognized license revenues associated with the amortization of the non-refundable upfront payment and target designation fees we received from BMS in accordance with the BMS CLA.

We evaluated our outstanding performance obligation following the amendment of the BMS CLA on December 1, 2020 (“amended BMS CLA”) and determined that our remaining performance obligation related to license revenues is immaterial.

We updated our measure of progress accordingly and amortized the remaining balance of unrecognized revenue as of December 1, 2020.

In accordance with the amended BMS CLA, we continue to be eligible to receive research, development, and regulatory milestone payments as well as sales milestone payments and royalties for each of the four active Collaboration Targets if defined milestones are achieved in relation to the license to our technology and know-how.

We will recognize revenue from these payments when earned or as sales occur.

We recognize collaboration revenues associated with Collaboration Target-specific pre-clinical analytical development and process development activities that are reimbursable by BMS under the BMS CLA and the amended BMS CLA as well as other related agreements.

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

Cost of Contract Revenues

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

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

There were no material changes to our critical accounting policies during the three months ended March 31, 2022 or reasonably possible changes of our critical accounting estimates as of March 31, 2022 that could have had a material impact on our results of operations for the three-month period ended March 31, 2022.

Research and development expenses

7 unchanged sentences

● costs associated with the rendering of collaboration services as well as the continued development of Product between the Signing Date and Closing;

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

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

8 unchanged sentences

● the timing of regulatory approvals;

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

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

A change in the outcome of any of these variables with respect to our product candidates that we may develop, including as a result of the Covid pandemic, 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 incur expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.

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

Our general and administrative expenses consist principally of employee, office, consulting, legal and other

professional and administrative expenses.

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

Our selling costs in the three months ended March 31, 2021 included employee expenses, as well as professional fees related to the preparation of a commercial launch of etranacogene dezaparvovec.

Other items, net

Our other income primarily consists of payments received to subsidize our research and development efforts, income from the subleasing of our Amsterdam facility, 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”).

Our other income primarily consists of income from the subleasing of our Amsterdam facility.

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

Results of Operations

Comparison of the three months ended September 30, 2021 and 2020

The following table presents a comparison of our results of operations for the three months ended September 30, 2021 and 2020.

Three months ended September 30,

Comparison of the three months ended March 31, 2022 and 2021

The following table presents a comparison of our results of operations for the three months ended March 31, 2022 and 2021.

Three months ended March 31,

(in thousands)

7 unchanged sentences

Other non-operating items, net

Net loss before income tax expense

Income tax expense

Our revenue for the three months ended September 30, 2021 and 2020 was as follows:

Three months ended September 30,

Net loss before income tax income / (expense)

Income tax income / (expense)

Our collaboration revenue for the three months ended March 31, 2022 and 2021 was as follows:

Three months ended March 31,

(in thousands)

License revenue

Collaboration revenue

Collaboration revenue CSL Behring

Collaboration revenue BMS

Total revenues

The CSL Behring transaction closed on May 6, 2021.

We did not recognize any license revenue related to the CSL Behring Agreement for the three months ended September 30, 2021.

Additionally, we did not recognize any license revenue related to the CSL Behring Agreement in the three months ended September 30, 2020.

We recognized $1.8 million of license revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the three months ended September 30, 2020.

We did not recognize any license revenue under the amended BMS CLA during the three months ended September 30, 2021.

We recognized $2.0 million of collaboration revenue in the three months ended September 30, 2021, compared to $0.0 million for the same period in 2020.

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

Our collaboration revenue increased further as a result of increased services provided to BMS following our December 2020 amendment of the BMS CLA.

Research and development expenses

Research and development expenses for the three months ended September 30, 2021 were $36.4 million, compared to $36.3 million for the same period in 2020.

Research and development expenses for the three months ended March 31, 2022 were $45.0 million, compared to $32.7 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 September 30,

Three months ended March 31,

(in thousands)

Etranacogene dezaparvovec (AMT-060/061)

Huntington's disease (AMT-130)

Programs in preclinical development and platform related expenses

Etranacogene dezaparvovec (AMT-060/061)

Total direct research and development expenses

7 unchanged sentences

Direct research and development expenses

Etranacogene dezaparvovec (AMT-060/061)

In the three months ended September 30, 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial.

We dosed a total of 54 patients between January 2019 and March 2020.

In the same period, 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.

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

We also incurred costs for manufacturing development.

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

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

Huntington disease (AMT-130)

In the three months ended September 30, 2021, and September 30, 2020, our external costs for the development of AMT-130 were primarily related to the execution of our Phase I/II clinical trial in the United States.

In addition, our external costs in the three months ended September 30, 2021 included expenses related to the preparation of a second Phase I/II clinical trial that will be conducted in Europe.

In the three months ended March 31, 2022, and March 31, 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.

The costs incurred for the three months ended March 31, 2022 also included costs related to our Phase I/IIb clinical trial in Europe .

Preclinical programs & platform development

In the three months ended September 30, 2021, we incurred $2.5 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of SCA3 (AMT-150), Fabry disease (AMT-191) and temporal lobe epilepsy (AMT-260), as well as various other research programs and technology innovation projects, compared to costs of $1.9 million in the same period in 2020.

In the three months ended March 31, 2022, we incurred $4.6 million of costs primarily related to our preclinical activities associated with product candidates for the treatment of Fabry disease (AMT-191) and temporal lobe epilepsy (AMT-260), as well as various other research programs and technology innovation projects.

In the three months ended March 31, 2021, we incurred $2.3 million of costs primarily related to our preclinical activities primarily 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.

Hemophilia B (AMT-060/061)

In the three months ended March 31, 2022 and March 31, 2021, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial.

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 for commercialization of the Product.

We also incurred costs for Manufacturing Development.

After the Closing, CSL Behring is responsible for the clinical and regulatory development 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 March 31, 2022 are presented net of reimbursements due from CSL Behring.

I n the same period in 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.

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

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

Other research & development expenses

● We incurred $14.6 million in personnel and contractor related expenses in the three months ended September 30, 2021, compared to $11.8 million for the same period in 2020.

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

● We incurred $4.8 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended September 30, 2021, compared to $4.4 million in the same period in 2020;

● We incurred $3.3 million in share-based compensation expenses in the three months ended September 30, 2021, compared to $3.9 million for the same period in 2020.

The decrease was primarily a result of compensation expense recorded during the three months ended September 30, 2020 related to the termination of one of our executives;

● We incurred $1.3 million of other expenses for the three months ended September 30, 2021, compared to $3.7 million for the same period in 2020.

The decrease was primarily due to $3.0 million of expense recorded for the three months ended September 30, 2020 related to license payments that have no alternative future use;

● We incurred $0.4 million of expenses in the three months ended September 30, 2021 related to an increase in the fair value of contingent consideration associated with the Corlieve Transaction, compared to nil for the same period in 2020.

● We incurred $16.2 million in personnel and contractor related expenses in the three months ended March 31, 2022, compared to $11.6 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 $5.3 million in operating expenses and depreciation expenses related to our rented facilities in the three months ended March 31, 2022, compared to $4.6 million in the same period in 2021;

● We incurred $4.5 million in disposable costs compared to $3.3 million for the same period in 2021;

● We incurred $4.1 million in share-based compensation expenses in the three months ended March 31, 2022, compared to $2.7 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 $2.6 million of other expenses for the three months ended March 31, 2022, compared to $2.5 million for the same period in 2021;

● We incurred $2.2 million of expenses in the three months ended March 31, 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.

Selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended September 30, 2021 were $12.0 million, compared to $10.8 million for the same period in 2020.

● We incurred $3.8 million in personnel and contractor related expenses in the three months ended September 30, 2021, compared to $3.6 million in the same period in 2020;

● We incurred $2.7 million in share-based compensation expenses in the three months ended September 30, 2021, compared to $2.5 million in the same period in 2020;

● We incurred $1.5 million in professional fees in the three months ended September 30, 2021, compared to $2.0 million in the same period in 2020.

Selling, general and administrative expenses for the three months ended March 31, 2022 were $11.0 million, compared to $12.4 million for the same period in 2021.

● We incurred $4.4 million in personnel and contractor related expenses in the three months ended March 31, 2022, compared to $3.9 million in the same period in 2021.

The increase in the three months ended March 31, 2022, relates to the recruitment of personnel;

● We incurred $2.8 million in share-based compensation expenses in the three months ended March 31, 2022, compared to $3.1 million in the same period in 2021;

● We incurred $1.1 million in professional fees in the three months ended March 31, 2022, compared to $2.9 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.

Other items, net

We recognized $1.3 million in other income of employee retention credit under the U.S.

CARES Act in the three months ended September 30, 2021, compared to no such income for the same period in 2020.

We recognized $0.0 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, 2021, compared to $0.5 million for the same period in 2020.

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

Other non-operating items, net

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

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

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

We issued warrants to BMS in 2015.

We recognized changes in the fair value of these warrants within other non-operating income / (expense).

Following the termination of the BMS warrants on December 1, 2020, we no longer recognize changes in the fair value of these warrants within other non-operating (expense) / income.

As of the same date, we recognized a 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 .

Our other non-operating items, net, for the three months ended September 30, 2021 and 2020 were as follows:

Three months ended September 30,

Our other non-operating items, net, for the three months ended March 31, 2022 and 2021 were as follows:

Three months ended March 31,

(in thousands)

1 unchanged sentence

Interest expense - Hercules long-term debt

Foreign currency gains / (losses), net

Foreign currency gains, net

Other non-operating gains

Total other non-operating income / (loss), net

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 $10.4 million during the three months ended September 30, 2021, compared to a net loss of $8.7 million during the same period in 2020.

In the three months ended September 30, 2021, we recognized income of nil within other non-operating gains as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $0.3 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.

Income tax expense

We recognized $0.1 million of deferred tax expense in the three months ended September 30, 2021, and nil in the same period ended 2020.

Comparison of the nine months ended September 30, 2021 and 2020

The following table presents a comparison of the nine months ended September 30, 2021 and 2020.

Nine months ended September 30,

(in thousands)

Total revenues

Operating expenses:

Cost of contract revenues

Research and development expenses

Selling, general and administrative expenses

Total operating expenses

Other expense

Income / (loss) from operations

Non-operating items, net

Income / (loss) before income tax expense

Income tax expense

Net income / (loss)

Our revenue for the nine months ended September 30, 2021 and 2020 was as follows:

Nine months ended September 30,

(in thousands)

License revenue

Collaboration revenue

Total revenues

We recognized $462.4 million of license revenue related to the License Sale on Closing of the CSL Behring Agreement in May 2021.

We did not recognize any such license revenue related to the CSL Behring Agreement in the nine months ended September 30, 2020.

We recognized $3.4 million of license revenue related to upfront payments and target designation fees received from BMS in 2015 under the BMS CLA for the nine months ended September 30, 2020.

We did not recognize any license revenue under the amended BMS CLA during the nine months ended September 30, 2021.

We recognized $3.9 million of collaboration revenue in the nine months ended September 30, 2021, compared to $0.1 million for the same period in 2020.

The increase in collaboration revenue primarily relates to the increased activities under the amended BMS CLA and the CSL Behring Agreement.

Cost of contract revenues

We recognized $23.2 million in cost of contract revenues for the nine months ended September 30, 2021, compared to nil during the same period in 2020.

We recorded $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.

Research and development

Research and development expenses for the nine months ended September 30, 2021 were $101.2 million, compared to $90.7 million for the same period in 2020.

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.

Nine months ended September 30,

(in thousands)

Etranacogene dezaparvovec (AMT-060/061)

Huntington's disease (AMT-130)

Programs in preclinical development and platform related expenses

Total direct research and development expenses

Employee and contractor-related expenses

Facility expenses

Share-based compensation expense

Other expenses

Fair value changes related to contingent consideration

Total other research and development expenses

Total research and development expenses

Direct research and development expenses

Etranacogene dezaparvovec (AMT-060/061)

In the nine months ended September 30, 2021 and 2020, the external costs for our hemophilia B program were primarily related to the execution of our Phase III clinical trial.

We dosed a total of 54 patients between January 2019 and March 2020.

We also incur 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.

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

We also incurred costs for manufacturing development.

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

Direct research and development expenses related to clinical development incurred between Closing and September 30, 2021, are presented net of reimbursements due from CSL Behring.

Huntington disease (AMT-130)

In the nine months ended September 30, 2021 and September 30, 2020, our external costs for the development of Huntington’s disease were primarily related to the execution of our Phase I/II clinical trial in the United States.

In addition, our external costs in the nine months ended September 30, 2021 included expenses related to the preparation of a second Phase I/II clinical trial that will be conducted in Europe.

Preclinical programs & platform development

In the nine months ended September 30, 2021, we incurred $6.5 million of costs primarily related to our preclinical activities primarily associated with product candidates for the treatment of SCA3 (AMT-150), Fabry disease (AMT-191) and temporal lobe epilepsy (AMT-260), as well as various other research programs and technology innovation projects, compared to costs of $4.7 million in the same period in 2020.

These expenses exclude costs related to our product candidate for Hemophilia A (AMT-180), which was deprioritized in June 2020.

Other research & development expenses

● We incurred $39.3 million in personnel and contractor related expenses in the nine months ended September 30, 2021, compared to $31.1 million for the same period in 2020.

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

● We incurred $13.8 million in operating expenses and depreciation expenses related to our rented facilities in the nine months ended September 30, 2021, compared to $12.6 million in the same period in 2020.

The increase is related to the expansion of our organization;

● We incurred $9.2 million in share-based compensation expenses in the nine months ended September 30, 2021, compared to $9.2 million for the same period in 2020;

● We incurred $4.5 million of other expenses for the nine months ended September 30, 2021, compared to $4.8 million for the same period in 2020.

The decrease was primarily due to $3.0 million of expense recorded in the nine months ended September 30, 2020 related to license payments that have no alternative future, offset by an increase in various other expenses as a result of expanding our organization;

● We incurred $0.4 million of expenses in the nine months ended September 30, 2021 related to an increase in the fair value of contingent consideration associated with the Corlieve Transaction, compared to nil for the same period in 2020.

Selling, general and administrative expenses

Selling, general and administrative expenses for the nine months ended September 30, 2021 were $42.3 million, compared to $31.4 million for the same period in 2020.

● We incurred $11.8 million in personnel and contractor related expenses in the nine months ended September 30, 2021, compared to $9.7 million in the same period in 2020.

The increase was primarily related to the recruitment of personnel;

● We incurred $9.5 million in share-based compensation expenses in the nine months ended September 30, 2021, compared to $7.2 million in the same period in 2020.

The increase was primarily related to the increase in awards granted, including those to newly recruited personnel;

● We incurred $6.5 million in professional fees in the nine months ended September 30, 2021, compared to $6.2 million in the same period in 2020.

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

● We incurred $4.5 million in financial advisory fees in relation to our licensing transaction with CSL Behring in the nine months ended September 30, 2021, compared to nil in the same period in 2020.

Other items, net

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

in conjunction with the Settlement Agreement in the nine months ended September 30, 2021, compared to no such income for the same period in 2020.

We recognized $2.6 million in other income of employee retention credit under the U.S.

CARES Act in the nine months ended September 30, 2021, compared to no such income for the same period in 2020.

We recognized $3.0 million in income from payments received from European authorities to subsidize our research and development efforts in the Netherlands in the nine months ended September 30, 2021, compared $1.0 million for the same period in 2020.

Other non-operating items, net

Total other non-operating income, net

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

1 unchanged sentence

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

We issued warrants to BMS in 2015.

We recognized changes in the fair value of these warrants within other non-operating income / (expense).

Following the termination of the BMS warrants on December 1, 2020, we no longer recognize changes in the fair value of these warrants within other non-operating (expense) / income.

As of the same date, we recognized a derivative financial liability related to a contingent payment due to BMS upon the consummation of a CoC-payment as described elsewhere in this Quarterly Report on Form 10-Q .

Nine months ended September 30,

(in thousands)

Interest income

Interest expense

Foreign currency gains / (losses), net

Other non-operating gains, net

Total non-operating income / (loss), net

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 $21.6 million during the nine months ended September 30, 2021, compared to a net loss of $7.7 million during the same period in 2020.

In the nine months ended September 30, 2021, we recognized income of nil within other non-operating items as there was no change in the fair value of the BMS derivative financial liability for the CoC-payment, compared to income of $2.5 million during the same period in 2020 related to changes in the fair value of the BMS warrants from the BMS CLA.

Income tax expense

We recognized $3.6 million of deferred tax expense for the nine months ended September 30, 2021, and nil in the same period ended 2020.

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 $8.6 million during the three months ended March 31, 2022, compared to a net gain of $4.6 million during the same period in 2021.

Income tax benefit / (expense)

We recognized $0.6 million of deferred tax benefit in the three months ended March 31, 2022, and $0.2 million of deferred tax expense for the same period in 2021.

Financial Position, Liquidity and Capital Resources

As of September 30, 2021, we had cash, cash equivalents and restricted cash of $581.3 million which include payments received from CSL Behring following the Closing.

This is expected to fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facility by 2023).

We expect that if we were to achieve the $0.3 billion in regulatory, first commercial sales, and development milestone payments, our runway would be further extended by 12 to 18 months.

Nine months ended September 30,

As of March 31, 2022, we had cash, cash equivalents and restricted cash of $528.1 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.

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

As of March 31, 2022, we had an outstanding loan amount owed to Hercules Capital, Inc.

(“Hercules”) for an aggregate principal amount of $100.0 million, with $8.3 million payable within 12 months.

Future interest payments and financing fees associated with the loan total $34.5 million, with $8.3 million payable within 12 months.

We are contractually required to repay the $100.0 million in equal installments between December 2024 and December 2025 or in full in December 2025 if, prior to June 30, 2024, either (a) the Biologics License Application (“BLA”) for AMT-061 is approved by the FDA or (b) AMT-130 is advanced into a pivotal trial.

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

As of March 31, 2022, we had fixed lease payment obligations of $63.3 million, with $7.0 million payable within 12 months.

The fixed lease payment obligations include payments owed under signed lease arrangements that are expected to commence in 2022.

Commitments related to Corlieve acquisition (nominal amounts)

In relation to the Corlieve acquisition, 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 Stock Purchase Agreement between the Company and Corlieve (“SPA”).

As of March 31, 2022, our commitment amount is $223.7 million.

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 with the exception of a payment we made in February 2022 to acquire the remaining outstanding shares as well as certain payments for post-acquisition services in 2022.

These payments are owed in Euro and have been translated at the foreign exchange rate as of March 31, 2022, of $1.11/€1.00.

As of March 31, 2022, we expect these obligations will become payable between 2022 and 2031.

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 License Sale to CSL Behring.

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

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

Three months ended March 31,

(in thousands)

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

Net cash generated from / (used in) operating activities

Net cash used in operating activities

Net cash used in investing activities

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

As a result of receiving the upfront payment upon Closing of the CSL Behring Agreement, we generated $335.0 million cash flows from operations during the nine months ended September 30, 2021.

We recorded net loss of $36.5 million in the three months ended September 30, 2021 and net income of $321.4 during the nine months ended September 30, 2021, compared to a net loss of $53.8 million and $124.3 million during the same periods in 2020.

As of September 30, 2021, we had an accumulated deficit of $463.4 million.

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 $46.7 million in the three months ended March 31, 2022, compared to a net loss of $41.6 million during the same periods in 2021.

As of March 31, 2022, we had an accumulated deficit of $501.8 million.

Sources of liquidity

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

In addition, we could potentially receive up to $0.3 billion in regulatory, first commercial sales and development milestone payments from CSL Behring.

On March 1, 2021, we entered into a Sales Agreement with SVB Leerink with respect to an ATM offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.

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

On March 1, 2021, we entered into a Sales Agreement with SVB Leerink LLC (“SVB Leerink”) with respect to an at the market (“ATM”) offering program, under which we may, from time to time in our sole discretion, offer and sell through SVB Leerink, acting as agent, our ordinary shares, up to an aggregate offering price of $200.0 million.

We will pay SVB Leerink a commission equal to 3% of the gross proceeds of the sales price of all ordinary shares sold through it as a sales agent under the Sales Agreement.

In the nine months ended September 30, 2021, we received net proceeds of $29.6 million from the issuance of 921,730 million ordinary shares that took place during March and April of this year.

On December 6, 2018, we signed the 2018 Amended Facility with Hercules that both refinanced our then-existing $20.0 million credit facility and provided us with an additional unconditional commitment of $15.0 million as well as a conditional commitment of $15 million that expired on June 30, 2020.

At signing, we drew down an additional $15.0 million, for a total outstanding amount of $35.0 million.

The 2018 Amended Facility extended the loan’s maturity date until June 1, 2023.

The interest-only period was initially extended from November 2018 to January 1, 2021.

The interest-only period was further extended to January 1, 2022 as a result of raising more than $90.0 million in equity financing in September 2019.

The interest-only period was again further extended to June 1, 2023 as a result of the 2021 Amended Facility.

The variable interest rate of the 2018 Amended Facility is equal to the greater of (i) 8.85% or (ii) 8.85% plus the prime rate less 5.50%.

Under the 2018 Amended Facility, we paid a facility fee equal to 0.50% at signing of the $35.0 million loan outstanding and will owe a back-end fee of 4.95% of the outstanding debt.

In addition, in May 2020, we paid a back-end fee of $1.0 million in relation to the 2016 Amended Facility.

On January 29, 2021, we and Hercules entered into the 2021 Amended Facility.

Pursuant to the 2021 Amended Facility, Hercules agreed to an additional Facility of $100.0 million (“Tranche B”), increasing the aggregate principal amount of the term loan facilities from $35.0 million to up to $135.0 million.

On January 29, 2021, we drew down $35.0 million of the Tranche B.

We may draw down the remaining $65.0 million under the Tranche B in a series of one or more advances of not less than $20.0 million each until December 15, 2021.

Advances under the Tranche B bear interest at a rate equal to the greater of (i) 8.25% or (ii) 8.25% plus the prime rate, less 3.25% per annum.

The principal balance and all accrued but unpaid interest on advances under the Tranche B is due on June 1, 2023, which date may be extended by us by up to two twelve-month periods.

Advances under the Tranche B may now be prepaid without charge as six-months have passed from the Closing Date.

As of September 30, 2021, $70.0 million was outstanding under the 2018 Amended Facility and the 2021 Amended Facility (December 31, 2020:

$35.0 million under the 2018 Amended Facility).

We are subject to the same covenants under our 2018 Amended Facility and 2021 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 2018 Amended Facility and 2021 Amended Facility may limit our ability to obtain debt financing.

In the year ended December 30, 2021, we received net proceeds of $29.6 million from the issuance of 921,730 ordinary shares that took place during March and April of last year.

On January 29, 2021, we drew down $35 million under our 2021 amendment (“2021 Amended Facility”) with Hercules.

We drew down a further $30 million under our December 2021 amendment (“2021 Restated Facility”) with Hercules in December 2021.

We are subject to certain covenants under our 2021 Restated Facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.

In addition, our pledge of assets as collateral to secure our obligations under the 2021 Restated Facility may limit our ability to obtain debt financing.

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

To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all.

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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.

Net Cash generated from / used in operating activities

Net cash generated from operating activities was $335.0 million for the nine months ended September 30, 2021 and consisted of 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 of $23.2 million, a change in deferred taxes of $3.6 million and other non-cash items, net, of $2.9 million.

Net Cash used in operating activities

Net cash used in operating activities was $25.7 million for the three months ended March 31, 2022 and consisted of net loss of $46.7 million adjusted for non-cash items, including depreciation and amortization expense of $2.1 million, share-based compensation expense of $6.9 million, changes in the fair value of contingent consideration and the derivative financial liability of $1.5 million, unrealized foreign exchange gains of $7.4 million and a change in deferred taxes of $0.6 million.

Net cash generated from operating activities also included favorable changes in operating assets and liabilities of $18.4 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 an increase in 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 amount 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 operating activities was $97.7 million for the nine months ended September 30, 2020 and consisted of a net loss of $124.3 million adjusted for non-cash items, including depreciation and amortization expense of $8.4 million, share-based compensation expense of $16.5 million, fair value gains on derivative financial instruments of $2.5 million, unrealized foreign exchange losses of $7.8 million, and a decrease in other non-cash items, of $2.1 million.

There was a net decrease in accounts receivable and contract asset, prepaid expenses, and other current assets and receivables of $17.2 million, primarily related to the collection of $20.0 million of the contract asset related to CSL milestones of $55.0 million in March 2022.

These changes also relate to a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $1.2 million.

Net cash used in operating activities was $41.3 million for the three months ended March 31, 2021 and consisted of a net loss of $41.6 million adjusted for non-cash items, including depreciation and amortization expense of $1.9 million, share-based compensation expense of $5.8 million, unrealized foreign exchange gains of $5.3 million and deferred tax expense of $0.2 million.

Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $2.2 million.

These changes primarily related to a net increase in accounts receivable and accrued income, prepaid expenses, and other current assets of $1.2 million and a net decrease in accounts payable, accrued expenses and other liabilities of $0.1 million.

These changes primarily related to a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $8.0 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 $5.8 million primarily related to an increase in contract liability for CSL expenses to be reimbursed and an increase in accruals for services provided by vendors not yet billed offset by the pay-out of employee bonuses.

Net cash used in investing activities

In the nine months ended September 30, 2021, we used $63.8 million in our investing activities compared to $6.8 million for the same period in 2020.

Nine months ended September 30,

In the three months ended March 31, 2022, we used $4.9 million in our investing activities compared to $3.9 million for the same period in 2021.

Three months ended March 31,

(in thousands)

Acquisition of Corlieve, net of cash acquired

Build out of Amsterdam site

Build out of Lexington site

Acquisition of licenses, patents, and other rights

Acquisition of remaining outstanding ordinary shares related to Corlieve acquisition

Total investments

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.

The build out of the Amsterdam site consumed $9.9 million cash during the nine months ended September 30, 2021, compared to $3.4 million for the same period in 2020.

The increase is a result of the construction of additional laboratories to support the expansions of our research and development activities as well as the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale.

The build out of the Amsterdam site consumed $3.7 million cash during the three months ended March 31, 2022, compared to $2.4 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.

Net cash generated from financing activities

In the nine months ended September 30, 2021, we generated $65.0 million in our financing activities compared to $3.7 million for the same period in 2020.

Nine months ended September 30,

In the three months ended March 31, 2022, we generated $0.5 million in our financing activities compared to $62.6 million for the same period in 2021.

Three months ended March 31,

(in thousands)

Cash flows from financing activities

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

Proceeds from loan increment, net of debt issuance costs

Proceeds from issuance of ordinary shares, net of issuance costs

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

Repayment of debt assumed through Corlieve Transaction

Net cash generated from financing activities

In January 2021, we received $34.6 million net proceeds from the Hercules 2021 Amended Facility.

We received net proceeds of $29.6 million associated with our ATM offering in March and April 2021.

Upon the acquisition of Corlieve, Corlieve held a loan with an outstanding amount equal to EUR 1.0 million ($1.2 million).

During the nine months ended September 30, 2021, the loan was repaid in its entirety.

During the three months ended March 31, 2022, we received $0.5 million from the exercise of options to purchase ordinary shares in relation to our share incentive plans compared to $0.4 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).

We received net proceeds of $27.6 million associated with our ATM offering in March 2021 (nil for same period in 2022).

Funding requirements

We believe our cash and cash equivalents as of September 30, 2021 will fund our operations into the first half of 2024 (assuming a full repayment of funds borrowed from Hercules under our term loan facilities by 2023).

We expect that if we were to achieve the $0.3 billion in regulatory, first commercial sales and development milestone payments, our runway would further be extended by 12 to 18 months.

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

● achieving the milestones and royalties as defined within the CSL Behring Agreement;

● achieving the first commercial sales milestones and royalties as defined within the CSL Behring Agreement.

Achieving the first commercial sales milestones amongst others are subject to CSL Behring obtaining required regulatory approvals, including MAA approval from the EMA, BLA approval from the FDA, and approval of a diagnostic test for neutralizing antibodies from the FDA;

● the payment of milestone payments that we might owe to former shareholders of Corlieve;

● the repayments of the principal amount of our venture debt loan with Hercules, which following the December 15, 2021 amendment will be due in equal installments between December 2024 and December 2025 or in full in December 2025 if, prior to June 30, 2024, either (a) the BLA for AMT-061 is approved by the FDA or (b) AMT-130 is advanced into a pivotal trial;

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

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

● the repayments of the principal and other fees associated with our venture debt loan with Hercules, which following the January 29, 2021 amendment will be due in June 2023;

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

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

● the costs associated with process validation and inspection readiness of etranacogene dezaparvovec.

Contractual obligations and commitments

The table below sets forth our contractual obligations and commercial commitments as of September 30, 2021, that are expected to have an impact on liquidity and cash flows in future periods.

(in thousands)

Debt obligations (including $14.0 million interest payments)

Operating lease obligations

Commitments related to Corlieve acquisition (nominal amounts)

In relation to the Corlieve Transaction, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractual milestones.

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

The timing of achieving these milestones and consequently the timing of payments is generally uncertain with the exception of a payment we owe upon acquiring the remaining outstanding shares as well as certain payments for post-acquisition services in 2022.

These payments are owed in Euro and have been translated at the foreign exchange rate as of September 30, 2021, of $1.16/€1.00.

As of September 30, 2021, we expect these obligations will become payable between 2022 and 2031.

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

We also 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).

We have not included these commitments on our balance sheet or in the table above because the achievement and timing of these milestones is not fixed and determinable.

We also have obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring.

We have not included these commitments on our balance sheet or in the table above because the achievement and timing of these milestones is not fixed and determinable.

We enter into contracts in the normal course of business with clinical research organizations (“CROs”) for preclinical research studies and clinical trials, research supplies and other services and products for operating purposes.

These contracts generally provide for termination on notice, and therefore are cancelable contracts and not included in the table of contractual obligations and commitments.

Off-Balance Sheet Arrangements

As of September 30, 2021, we did not have any off-balance sheet arrangements as defined in Item 303(a)(4) of Regulation S-K.

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