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Form 10-Q uniQure N.V. For: Mar 31

May 2, 2022 7:32 AM EDT

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington D.C. 20549

FORM 10-Q

(Mark One)

QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended March 31, 2022

OR

TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from _______ to _______

Commission file number: 001-36294

uniQure N.V.

(Exact name of Registrant as specified in its charter)

The Netherlands

(State or other jurisdiction of incorporation or organization)

Not applicable

(I.R.S. Employer Identification No.)

Paasheuvelweg 25

1105 BP Amsterdam, The Netherlands

(Address of principal executive offices) (Zip Code)

+31-20-240-6000

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:

Title of each class:

Trading Symbol(s)

Name of each exchange on which registered

Ordinary Shares, par value €0.05

QURE

The Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.    Yes No .  

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).    Yes No .  

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer” “accelerated filer” and “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

Large accelerated filer  

Accelerated filer  

Non-accelerated filer

Smaller reporting company 

Emerging growth company

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13 (a) of the Exchange Act  

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.)    Yes No

As of April 27, 2022, the registrant had 46,647,620 ordinary shares, par value €0.05, outstanding.

TABLE OF CONTENTS

 

    

    

Page

PART I – FINANCIAL INFORMATION

Item 1

Financial Statements

2

Item 2

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

17

Item 3

Quantitative and Qualitative Disclosures About Market Risk

28

Item 4

Controls and Procedures

28

PART II – OTHER INFORMATION

Item 1

Legal Proceedings

29

Item 1A

Risk Factors

30

Item 2

Unregistered Sales of Equity Securities and Use of Proceeds

61

Item 3

Defaults Upon Senior Securities

61

Item 4

Mine Safety Disclosures

61

Item 5

Other Information

61

Item 6

Exhibits

61

SPECIAL CAUTIONARY NOTICE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains “forward-looking statements” as defined under federal securities laws. Forward-looking statements are based on our current expectations of future events and many of these statements can be identified using terminology such as “believes,” “expects,” “anticipates,” “plans,” “may,” “will,” “projects,” “continues,” “estimates,” “potential,” “opportunity” and similar expressions. These forward-looking statements, include, but are not limited to, statements related to the Covid-19 coronavirus pandemic, our collaboration and license agreements, our cash runway, the advancement of our clinical trials, and the impact of regulatory actions on our regulatory submission timelines.

Forward-looking statements are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied. The most significant factors known to us that could materially adversely affect our business, operations, industry, financial position or future financial performance include those discussed in Part II, Item 1A “Risk Factors,” as well as those discussed in Part I, Item 2 “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Quarterly Report on Form 10-Q, as well as other factors which may be identified from time to time in our other filings with the Securities and Exchange Commission (“SEC”), including our most recent Annual Report on Form 10-K filed with the SEC on February 25, 2022 (the “Annual Report”), or in the documents where such forward-looking statements appear. You should carefully consider that information before you make an investment decision.

You should not place undue reliance on these forward-looking statements, which speak only as of the date that they were made. Our actual results or experience could differ significantly from those anticipated in the forward-looking statements and from historical results, due to the risks and uncertainties described in this Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, and in our Annual Report, including in “Part I, Item 1A. Risk Factors,” as well as others that we may consider immaterial or do not anticipate at this time. These cautionary statements should be considered in connection with any written or oral forward-looking statements that we may make in the future or may file or furnish with the SEC. We do not undertake any obligation to release publicly any revisions to these forward-looking statements after completion of the filing of this Quarterly Report on Form 10-Q to reflect later events or circumstances or to reflect the occurrence of unanticipated events. All forward-looking statements attributable to us are expressly qualified in their entirety by these cautionary statements.

In addition, with respect to all of our forward-looking statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

1

Part I – FINANCIAL INFORMATION

Item 1.Financial Statements

uniQure N.V.

UNAUDITED CONSOLIDATED BALANCE SHEETS

March 31, 

December 31, 

    

2022

    

2021

(in thousands, except share and per share amounts)

Current assets

Cash and cash equivalents

$

524,886

$

556,256

Accounts receivable and contract asset

38,644

58,768

Prepaid expenses

12,422

10,540

Other current assets and receivables

2,260

2,675

Total current assets

578,212

628,239

Non-current assets

Property, plant and equipment, net of accumulated depreciation of $38.5 million as of March 31, 2022 and $36.9 million as of December 31, 2021, respectively.

44,918

43,505

Operating lease right-of-use assets

24,941

25,573

Intangible assets, net, including an in-process research and development asset of $59.5 million

61,135

62,686

Goodwill

26,565

27,633

Deferred tax assets, net

15,442

15,647

Other non-current assets

5,937

5,897

Total non-current assets

178,938

180,941

Total assets

$

757,150

$

809,180

Current liabilities

Accounts payable

$

9,920

$

2,502

Accrued expenses and other current liabilities

19,945

28,487

Current portion of contingent consideration

9,081

Current portion of operating lease liabilities

5,754

5,774

Total current liabilities

44,700

36,763

Non-current liabilities

Long-term debt

101,421

100,963

Operating lease liabilities, net of current portion

28,232

28,987

Contingent consideration, net of current portion

22,059

29,542

Deferred tax liability, net

11,298

12,913

Other non-current liabilities

3,439

4,236

Total non-current liabilities

166,449

176,641

Total liabilities

211,149

213,404

Commitments and contingencies

Shareholders' equity

Ordinary shares, €0.05 par value: 80,000,000 shares authorized as of March 31, 2022 and December 31, 2021 and 46,641,448 and 46,298,635 ordinary shares issued and outstanding as of March 31, 2022 and December 31, 2021, respectively

2,821

2,802

Additional paid-in-capital

1,084,306

1,076,972

Accumulated other comprehensive loss

(39,306)

(28,856)

Accumulated deficit

(501,820)

(455,142)

Total shareholders' equity

546,001

595,776

Total liabilities and shareholders' equity

$

757,150

$

809,180

The accompanying notes are an integral part of these unaudited consolidated financial statements.

2

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND

COMPREHENSIVE LOSS

Three months ended March 31, 

    

2022

    

2021

(in thousands, except share and per share amounts)

Collaboration revenues

$

1,792

$

454

Total revenues

1,792

454

Operating expenses:

Research and development expenses

(45,003)

(32,656)

Selling, general and administrative expenses

(10,987)

(12,375)

Total operating expenses

(55,990)

(45,031)

Other income

311

352

Other expense

(193)

(233)

Loss from operations

(54,080)

(44,458)

Interest income

42

40

Interest expense

(2,515)

(1,551)

Foreign currency gains, net

8,567

4,626

Other non-operating gains, net

692

Loss before income tax benefit / (expense)

$

(47,294)

$

(41,343)

Income tax benefit / (expense)

616

(213)

Net loss

$

(46,678)

$

(41,556)

Other comprehensive loss:

Foreign currency translation adjustments

(10,450)

(7,560)

Total comprehensive loss

$

(57,128)

$

(49,116)

Basic and diluted net loss per ordinary share

$

(1.00)

$

(0.91)

Weighted average shares used in computing basic and diluted net loss per ordinary share

46,599,114

45,468,485

The accompanying notes are an integral part of these unaudited consolidated financial statements.

3

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY

FOR THE THREE-MONTH PERIOD ENDED MARCH 31

Accumulated

Additional

other

Total

Ordinary shares

paid-in

comprehensive

 Accumulated 

shareholders’

   No. of shares   

    

   Amount   

    

      capital      

    

income / (loss)

    

deficit

    

equity

(in thousands, except share and per share amounts)

Balance at December 31, 2020

44,777,799

$

2,711

$

1,016,018

$

9,907

$

(784,731)

$

243,905

Loss for the period

(41,556)

(41,556)

Other comprehensive loss

(7,560)

(7,560)

Issuance of ordinary shares

859,885

52

27,647

27,699

Exercise of share options

16,782

1

391

392

Restricted share units distributed during the period

269,089

16

(16)

Share-based compensation expense

5,761

5,761

Issuance of ordinary shares relating to employee stock purchase plan

1,174

49

49

Balance at March 31, 2021

45,924,729

$

2,780

$

1,049,850

$

2,347

$

(826,287)

$

228,690

Balance at December 31, 2021

46,298,635

$

2,802

$

1,076,972

$

(28,856)

$

(455,142)

$

595,776

Loss for the period

(46,678)

(46,678)

Other comprehensive loss

(10,450)

(10,450)

Exercise of share options

68,124

4

420

424

Restricted share units and performance share units distributed during the period

271,131

15

(15)

Share-based compensation expense

6,868

6,868

Issuance of ordinary shares relating to employee stock purchase plan

3,558

61

61

Balance at March 31, 2022

46,641,448

$

2,821

$

1,084,306

$

(39,306)

$

(501,820)

$

546,001

The accompanying notes are an integral part of these unaudited consolidated financial statements.

4

uniQure N.V.

UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS

Three months ended March 31, 

        

2022

        

2021

(in thousands)

Cash flows from operating activities

Net loss

$

(46,678)

$

(41,556)

Adjustments to reconcile net loss to net cash used in operating activities:

Depreciation and amortization expense

2,122

1,862

Share-based compensation expense

6,868

5,761

Deferred tax (income) / expense

(616)

213

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

1,512

-

Unrealized foreign exchange gains, net

(7,356)

(5,342)

Changes in operating assets and liabilities:

Accounts receivable and contract asset, prepaid expenses, and other current assets and receivables

17,209

(8,023)

Accounts payable

7,306

2,510

Accrued expenses, other liabilities, and operating leases

(6,078)

3,302

Net cash used in operating activities

(25,711)

(41,273)

Cash flows from investing activities

Purchases of property, plant, and equipment

(4,058)

(3,876)

Acquisition of remaining outstanding ordinary shares related to Corlieve acquisition

(822)

-

Net cash used in investing activities

(4,880)

(3,876)

Cash flows from financing activities

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

485

442

Proceeds from issuance of ordinary shares

-

28,734

Share issuance costs from issuance of ordinary shares

-

(1,161)

Proceeds from loan increment, net of debt issuance costs

-

34,603

Net cash generated from financing activities

485

62,618

Currency effect on cash, cash equivalents and restricted cash

(1,168)

(1,620)

Net (decrease) / increase in cash, cash equivalents and restricted cash

(31,274)

15,849

Cash, cash equivalents and restricted cash at beginning of period

559,353

247,680

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

$

528,079

$

263,529

Cash and cash equivalents

$

524,886

$

260,813

Restricted cash related to leasehold and other deposits

3,193

2,716

Total cash, cash equivalents and restricted cash

$

528,079

$

263,529

Supplemental cash flow disclosures:

Cash paid for interest

$

(1,906)

$

(1,410)

Non-cash (decrease) / increase in accounts payables and accrued expenses and other current liabilities related to purchases of property, plant, and equipment

$

(611)

$

-

The accompanying notes are an integral part of these unaudited consolidated financial statements.

5

1General business information

uniQure (the “Company”) was incorporated on January 9, 2012 as a private company with limited liability (besloten vennootschap met beperkte aansprakelijkheid) under the laws of the Netherlands. The Company is a leader in the field of gene therapy and seeks to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company’s business was founded in 1998 and was initially operated through its predecessor company, Amsterdam Molecular Therapeutics (AMT) Holding N.V (“AMT”). In 2012, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquired the entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT. Effective February 10, 2014, in connection with its initial public offering, the Company converted into a public company with limited liability (naamloze vennootschap) and changed its legal name from uniQure B.V. to uniQure N.V.

The Company is registered in the trade register of the Chamber of Commerce (Kamer van Koophandel) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, and its registered office is located at Paasheuvelweg 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.

The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.

2Summary of significant accounting policies

2.1Basis of preparation

The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the SEC regarding interim financial reporting. Any reference in these notes to applicable guidance is meant to refer to authoritative U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”).

The unaudited consolidated financial statements are presented in U.S. dollars, except where otherwise indicated. Transactions denominated in currencies other than U.S. dollars are presented in the transaction currency with the U.S. dollar amount included in parenthesis, converted at the foreign exchange rate as of the transaction date.

2.2Unaudited interim financial information

The interim financial statements and related disclosures are unaudited, have been prepared on the same basis as the annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for a fair statement of the financial position, results of operations and changes in financial position for the period presented.

Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been omitted. The results of operations for the three months ended March 31, 2022, are not necessarily indicative of the results to be expected for the full year ending December 31, 2022, or for any other future year or interim period. The accompanying financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report.

2.3Use of estimates

The preparation of the financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

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2.4Accounting policies

The principal accounting policies applied in the preparation of these unaudited consolidated financial statements are described in the Company’s audited financial statements as of and for the year ended December 31, 2021, and the notes thereto, which are included in the Company’s Annual Report. There have been no material changes in the Company’s significant accounting policies during the three months ended March 31, 2022.

2.5Recent accounting pronouncements

There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2022, as compared to the recent accounting pronouncements described in Note 2.3.23 of the Company’s Annual Report, which could be expected to materially impact the Company’s unaudited consolidated financial statements.

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Collaboration arrangements and concentration of credit risk

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 LLC, (“CSL Behring”), pursuant to which CSL Behring received exclusive global rights to etranacogene dezaparvovec, the Company’s 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 (the “HSR Act”) expired on May 5, 2021 (“Closing”).

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

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

The Company determined that the License Sale was completed on May 6, 2021, when it transferred the license and CSL Behring assumed full responsibility for the development and commercialization of the Product. At Closing, the Company evaluated the amounts of potential payments and the likelihood that the payments will be received. The Company utilized the most likely amount method to estimate the variable consideration to be included in the transaction price. Since the Company cannot control the achievement of regulatory and first commercial sales milestones, the Company concluded that the potential payments are constrained as of Closing. The Company determined that it 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. Similarly, the Company records expenses related to its existing license and other agreements as well as its financial advisor for a mid-single digit percentage of any such revenue recognized associated to meeting a milestone. The Company includes payments related to sales milestones in the transaction price when their achievement becomes probable, and it will include royalties on the sale of Product once these have been earned. During the three months ended March 31, 2022, the Company did not recognize any license revenue (nil for same period in 2021).

The Company recognized $1.4 million of collaboration revenue in the three months ended March 31, 2022, compared to nil in the same period in 2021. The Company generates such collaboration revenue from services rendered in relation to completing the HOPE-B clinical trial on behalf of CSL Behring as well as additional development services that CSL Behring requested. These collaboration services are reimbursed at the pre-agreed full-time-employee rate (“FTE-rate”).

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As of December 31, 2021, the Company recorded accounts receivable of $2.9 million from CSL Behring related to clinical development services as well as a contract asset of $55.0 million associated with milestone payments due upon CSL Behring’s global regulatory submissions for etranacogene dezaparvovec, which were deemed to be probable. In March 2022, CSL Behring submitted the global regulatory submissions, and as of March 31, 2022, the Company collected $20.0 million of the total $55.0 million owed. As of March 31, 2022, the Company had accounts receivable of $38.3 million from CSL Behring, $35.0 million of which were related to the uncollected milestone payment associated with the global regulatory submissions. The remaining $35.0 million was received in April 2022.

Bristol-Myers Squibb collaboration

In May 2015, the Company and Bristol-Myers Squibb (“BMS”) entered into a collaboration and license agreement and various related agreements with BMS (“BMS CLA”). On December 1, 2020, the Company and BMS amended the BMS CLA (“amended BMS CLA”) to reduce the scope of the collaboration and license from potentially ten to then four collaboration targets (including a right to replace two of such targets by December 1, 2021). The Company is eligible to receive research, development, and regulatory milestone payments of up to $217.0 million for each of the four collaboration targets if defined milestones are achieved.

BMS may place purchase orders to provide limited services primarily related to analytical and development efforts in respect of the four collaboration targets. BMS may request such services for a period not to exceed the earlier of (i) the completion of all activities under a Research Plan and (ii) November 30, 2023. BMS reimburses the Company for these services.

Collaboration Revenue related to these contracted services is recognized when performance obligations are satisfied. The Company generated $0.4 million collaboration revenue for the three months ended March 31, 2022 ($0.5 million for the three months ended March 31, 2021).

 

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4

 Fair value measurement

The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. U.S. GAAP requires disclosure of methodologies used in determining the reported fair values and establishes a hierarchy of inputs used when available. The three levels of the fair value hierarchy are described below:

Level 1 – Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company can access at the measurement date.

Level 2 – Valuations based on quoted prices for similar assets or liabilities in markets that are not active or models for which the inputs are observable, either directly or indirectly.

Level 3 – Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and are unobservable.

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.

The carrying amount of cash and cash equivalents, accounts receivable, prepaid expenses, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Consolidated balance sheets approximate their fair values due to their short-term maturities.

The following table sets forth the Company’s assets and liabilities that are required to be measured at fair value on a recurring basis as of March 31, 2022, and December 31, 2021:

 

Quoted prices
in active
markets
(Level 1)

 

Significant
other
observable
inputs
(Level 2)

 

Significant
unobservable
inputs
(Level 3)

 

Total

 

Classification in Consolidated
balance sheets

(in thousands)

At December 31, 2021

Assets:

Cash and cash equivalents

$

556,256

$

$

$

556,256

Cash and cash equivalents

Restricted cash

3,097

3,097

Other non-current assets

Total assets

$

559,353

$

$

$

559,353

Liabilities:

Contingent consideration

29,542

29,542

Contingent consideration

Derivative financial instrument

2,805

2,805

Other non-current liabilities

Consideration for post-acquisition services

846

846

Other non-current liabilities

Total liabilities

$

$

$

33,193

$

33,193

At March 31, 2022

Assets:

Cash and cash equivalents

$

524,886

$

$

$

524,886

Cash and cash equivalents

Restricted cash

3,193

3,193

Other non-current assets

Total assets

$

528,079

$

$

$

528,079

Liabilities:

Contingent consideration

31,140

31,140

Current portion of contingent consideration; contingent consideration, net of current portion

Derivative financial instrument

2,116

2,116

Other non-current liabilities

Consideration for post-acquisition services

752

752

Other non-current liabilities

Total liabilities

$

$

$

34,008

$

34,008

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Derivative financial instrument

The Company issued a derivative financial instrument related to its collaboration with BMS.

On December 1, 2020, the Company and BMS agreed that upon the consummation of a change of control transaction of uniQure that occurs prior to December 1, 2026 or BMS’ delivery of a target cessation notice for all four collaboration targets, the Company (or its third party acquirer) shall pay to BMS a one-time, non-refundable, non-creditable cash payment of $70.0 million, provided that (x) if $70.0 million is greater than five percent (5.0%) of the net proceeds (as contractually defined) from such change of control transaction, the payment shall be an amount equal to five percent of such net proceeds, and (y) if $70.0 million is less than one percent of such net proceeds, the change of control payment shall be an amount equal to one percent of such net proceeds (“CoC-payment”). The Company has not consummated any change of control transaction as of March 31, 2022 that would obligate it to make a CoC-payment.

The Company determined that the CoC-payment should be recorded as a derivative financial liability as of December 1, 2020 and that subsequent changes in the fair market value of this derivative financial liability should be recorded in profit and loss. The fair market value of the derivative financial liability is materially impacted by probability that market participants assign to the likelihood of the occurrence of a change of control transaction that would give rise to a CoC-payment. This probability represents an unobservable input. The Company determines the fair market value of the derivative financial liability by using a present value model based on expected cash flow. The expected cash flows are materially impacted by the probability that market participants assign to the likelihood of the occurrence of a change of control transaction within the biotechnology industry. The Company estimated this unobservable input using the best information available as of March 31, 2022 and December 31, 2021. The Company obtained reasonably available market information that it believed market participants would use in determining the likelihood of the occurrence of a change-of control transaction within the biotechnology industry. Selecting and evaluating market information involves considerable judgement and uncertainty. Based on all such information and its judgment, the Company estimated that the fair market value of the derivative financial liability (presented within “Other non-current liabilities”) as of March 31, 2022 was $2.1 million and as of December 31, 2021 was $2.8 million. The decrease of the fair value market value of the derivative financial liability of $0.7 million for the three months ended March 31, 2022 was recorded as a gain within Other non-operating (losses) / gains (nil in same period 2021).

Contingent consideration

The Company is required to pay up to EUR 178.8 million ($198.8 million at March 31, 2022) to the former shareholders of Corlieve upon the achievement of contractually defined milestones in connection with the Company’s acquisition of Corlieve. The fair market value of the contingent consideration was determined using unobservable initial inputs with respect to (i) the probability of achieving the relevant milestones, or POS, (ii) the estimated timing of achieving such milestones, and (iii) the interest rate used to discount the payments. The Company determined the fair market value of the contingent consideration by calculating the probability-adjusted payments based on each milestone’s probability of achievement. The probability-adjusted payments were then discounted to present value using a discount rate representing the Company’s credit risk. The discount rate was determined using the effective interest rate of the Company’s existing debt facility adjusted for difference in maturity dates based on market data on effective yields for US bonds with a CCC credit rating.

The fair value of the contingent consideration as of March 31, 2022 was $31.1 million using discount rates of approximately 10.3% as well as a 55.0% likelihood of AMT-260 advancing into clinical development by no later than early 2024. If as of March 31, 2022 the Company had assumed a 100% likelihood of AMT-260 advancing into clinical development, then the fair value of the contingent consideration would have increased to $51.7 million. If as of March 31, 2022 the Company assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would be released to income. Changes in fair value of the contingent consideration are recognized within research and development expenses in the consolidated statements of operations.

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The following table presents the changes in fair value of contingent consideration between December 31, 2021 and March 31, 2022:

Amount of

contingent

consideration

2022

(in thousands)

Balance at December 31, 2021

$

29,542

Change in fair value (presented within research and development expenses)

2,204

Currency translation effects

(606)

Balance at March 31, 2022

$

31,140

As of March 31, 2022, the Company classified $9.1 million of the total contingent consideration of $31.1 million as current liabilities. The balance sheet classification between current and non-current liabilities is based upon the Company’s best estimate of the timing of settlement of the remaining relevant milestones.  

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Accrued expenses and other current liabilities

Accrued expenses and other current liabilities include the following items:

March 31, 

December 31, 

    

2022

    

2021

(in thousands)

Accruals for goods received from and services provided by vendors-not yet billed

$

10,341

$

13,012

Personnel related accruals and liabilities

9,604

12,603

Accrued contract fulfillment costs and costs to obtain a contract

2,872

Total

$

19,945

$

28,487

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6 Long-term debt

On June 14, 2013, the Company entered into a venture debt loan facility with Hercules Capital, Inc. (formerly known as Hercules Technology Growth Capital, Inc.) (“Hercules”). The facility was amended and restated in 2014, 2016, 2018 (“2018 Amended Facility”), January 2021 (“2021 Amended Facility”) and in December 2021 (“2021 Restated Facility”). On January 29, 2021, the Company drew down $35.0 million as part of the amendment of the facility at that time.

Pursuant to the 2021 Restated Facility, Tranche A and Tranche B of the 2021 Amended Facility  with a total outstanding balance of $70.0 million were consolidated into one tranche with a total commitment of $100.0 million. The Company drew down an additional $30.0 million at the time of the December 2021 amendment, resulting in total principal outstanding as of March 31, 2022 of $100.0 million. The 2021 Restated Facility extended the loan’s maturity date from June 1, 2023 until December 1, 2025. The interest-only period was extended from January 1, 2023 to December 1, 2024, or December 1, 2025 if, prior to June 30, 2024, either (a) the Biologics License Application (“BLA”) for AMT-061 is approved by the U.S. Food and Drug Administration (“FDA”) or (b) AMT-130 is advanced into a pivotal trial. The interest rate is adjustable and is the greater of (i) 7.95% and (ii) 7.95% plus the prime rate less 3.25% per annum. Under the 2021 Restated Facility, the Company owes a back-end fee of 4.85% of the outstanding debt. The Company is required to repay the facility in equal monthly installments of principal and interest between the end of the interest-only period and the maturity date. The Company continues to owe a $2.5 million back-end fee related to the 2021 Amended Facility which is due on June 1, 2023.

The amortized cost (including interest due presented as part of accrued expenses and other current liabilities) of the 2021 Restated Facility was $102.1 million as of March 31, 2022, compared to $101.6 million as of December 31, 2021, and is recorded net of discount and debt issuance costs. The foreign currency loss on the facility in the three months ended March 31, 2022, was $2.1 million compared to a foreign currency loss of $3.2 million during the same period in 2021 for the 2018 Amended Facility and 2021 Amended Facility.

Interest expense associated with the 2021 Restated Facility during the three months ended March 31, 2022, was $2.4 million compared to $1.5 million during the same period in 2021 for the 2018 Amended Facility and 2021 Amended Facility.

As a covenant in the 2021 Restated Facility the Company has periodic reporting requirements and is required to keep a minimum cash balance deposited in bank accounts in the United States equivalent to the lesser of (i) 65% of the outstanding balance of principal due or (ii) 100% of worldwide cash and cash equivalents. This restriction on cash and cash equivalents only relates to the location of the cash and cash equivalents, and such cash and cash equivalents can be used at the discretion of the Company. The Company, beginning on April 1, 2023, is also required to keep a minimum of unrestricted cash of at least 50% of the loan amount outstanding. 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 minimum cash covenant will be lowered to at least 30% of the loan amount outstanding and its effectiveness will be deferred to April 1, 2024. In combination with other covenants, the 2021 Restated Facility restricts the Company’s ability to, among other things, incur future indebtedness and obtain additional debt financing, to make investments in securities or in other companies, to transfer assets, to perform certain corporate changes, to make loans to employees, officers, and directors, and to make dividend payments and other distributions to its shareholders. The Company secured the facilities by directly or indirectly pledging its total assets of $757.2 million, with the exception of $101.4 million of cash and cash equivalents and other current assets held by uniQure N.V.

The 2021 Restated Facility contains provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Hercules to declare all principal, interest and other amounts owed by the Company immediately due and payable. As of March 31, 2022, the Company was in material compliance with all covenants and provisions.

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7Share-based compensation

The Company’s share-based compensation plans include the 2014 Amended and Restated Share Option Plan (the “2014 Plan”) and inducement grants under Rule 5653(c)(4) of the Nasdaq Global Select Market with terms similar to the 2014 Plan (together the “2014 Plans”). At the annual general meeting of shareholders in June 2021, the Company’s shareholders approved an increase in the number of ordinary shares authorized for issuance under the 2014 Plan from 8,601,471 to 12,601,471.

a)2014 Plans

Share-based compensation expense recognized by classification included in the Consolidated statements of operations and comprehensive loss in relation to the 2014 Plans for the periods indicated below was as follows:

    

Three months ended March 31, 

2022

2021

(in thousands)

Research and development

$

4,050

$

2,674

Selling, general and administrative

2,813

3,080

Total

$

6,863

$

5,754

Share-based compensation expense recognized by award type was as follows:

Three months ended March 31, 

2022

2021

(in thousands)

Award type

Share options

$

3,255

$

2,840

Restricted share units

3,270

2,560

Performance share units

338

354

Total

$

6,863

$

5,754

As of March 31, 2022, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:

    

Unrecognized

  

Weighted average

    

share-based

    

remaining

compensation

period for

expense

     recognition     

(in thousands)

(in years)

Award type

Share options

$

34,041

2.94

Restricted share units

35,252

2.40

Performance share units

444

0.46

Total

$

69,737

2.65

The Company satisfies the exercise of share options and vesting of Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) through newly issued ordinary shares.

Share options

Share options are priced on the date of grant and, except for certain grants made to non-executive directors, vest over a period of four years. The first 25% vests after one year from the initial grant date and the remainder vests in equal quarterly installments over years two, three and four. Certain grants to non-executive directors vest in full after one year. Any options that vest must be exercised by the tenth anniversary of the initial grant date.

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The following tables summarize option activity under the Company’s 2014 Plans for the three months ended March 31, 2022:

Options

Number of

Weighted average

    

ordinary shares

    

exercise price

Outstanding at December 31, 2021

3,308,325

$

31.02

Granted

1,011,876

$

16.04

Forfeited

(70,868)

$

36.21

Expired

(55,511)

$

37.86

Exercised

(64,124)

$

6.36

Outstanding at March 31, 2022

4,129,698

$

27.55

Thereof, fully vested and exercisable on March 31, 2022

1,925,690

$

26.44

Thereof, outstanding and expected to vest after March 31, 2022

2,204,008

$

28.53

Total weighted average grant date fair value of options issued during the period (in $ millions)

$

9.2

Proceeds from option sales during the period (in $ millions)

$

0.4

The fair value of each option issued is estimated at the respective grant date using the Hull & White option pricing model with the following weighted-average assumptions:

Three months ended March 31, 

Assumptions

    

2022

    

2021

Expected volatility

70%

75%

Expected terms

10 years

10 years

Risk free interest rate

2.12%

1.21 - 1.71%

Expected dividend yield

0%

0%

Restricted share units (“RSUs”)

The following table summarizes the RSUs activity for the three months ended March 31, 2022:

RSU

    

    

Weighted average

Number of

grant-date fair

ordinary shares

value

Non-vested at December 31, 2021

710,617

$

38.89

Granted

1,273,763

$

16.04

Vested

(195,261)

$

39.90

Forfeited

(45,050)

$

27.93

Non-vested at March 31, 2022

1,744,069

$

22.37

Total weighted average grant date fair value of RSUs granted during the period (in $ millions)

$

20.4

RSUs vest over one to three years. RSUs granted to non-executive directors vest one year from the date of grant.

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Performance share units (“PSUs”)

The following table summarizes the PSUs activity for the three months ended March 31, 2022:

PSU

    

    

Weighted average

Number of

grant-date fair

ordinary shares

value

Non-vested at December 31, 2021

632,930

$

33.54

Granted

8,100

$

16.04

Vested

(77,330)

$

57.56

Forfeited

(19,800)

$

29.35

Non-vested at March 31, 2022

543,900

$

30.01

PSUs granted in 2019 vested on the third anniversary of the grant, subject to the grantee’s continued employment.

The Company granted shares to certain employees in September and December 2021 that will be earned upon achievement of defined milestones. Earned shares will vest upon the later of a minimum service period of one year or three years, or the achievement of defined milestones, subject to the grantee’s continued employment. In addition, portions of the December 2021 shares granted to executives and other members of senior management are subject to achieving a minimum total shareholder return relative to the Nasdaq biotechnology index. The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable.

b)Employee share purchase plan (“ESPP”)

In June 2018, the Company’s shareholders adopted and approved an ESPP allowing the Company to issue up to 150,000 ordinary shares. The ESPP is intended to qualify under Section 423 of the Internal Revenue Code of 1986. Under the ESPP, employees are eligible to purchase ordinary shares through payroll deductions, subject to any plan limitations. The purchase price of the ordinary shares on each purchase date is equal to 85% of the lower of the closing market price on the offering date and the closing market price on the purchase date of each three-month offering period. During the three months ended March 31, 2022, 3,558 ordinary shares were issued under the ESPP compared to 1,174 during the same period in 2021. As of March 31, 2022, a total of 123,744 ordinary shares remain available for issuance under the ESPP plan compared to a total of 130,852 as of March 31, 2021.

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Income taxes

The Company recorded $0.6 million deferred tax benefit in relation to its operations in the United States, France and Switzerland during the three-month period ended March 31, 2022. The Company recorded $0.2 million net deferred tax expense in the prior year in the United States as of March 31, 2021.

The effective income tax rate of (1.3)% during the three months ended March 31, 2022 is substantially lower than the enacted rated of 25.8% in the Netherlands as the Company recorded a valuation allowance against its net deferred tax assets in the Netherlands. The effective income tax rate during the three months ended March 31, 2021 was 0.5% as the Company had recorded a valuation allowance against all its net deferred tax assets in the Netherlands.

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9Basic and diluted earnings per share

Diluted earnings per share are calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. As the Company has incurred a loss, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of loss per share. The shares are presented without giving effect to the application of the treasury method or exercise prices that would be above the share price as of March 31, 2022 and March 31, 2021, respectively.

The potentially dilutive ordinary shares are summarized below:

March 31, 

    

2022

    

2021

(ordinary shares)

Stock options under 2014 Plans and previous plan

4,139,698

3,345,646

Non-vested RSUs and PSUs

2,287,969

796,982

Employee share purchase plan

745

729

Total potential dilutive ordinary shares

6,428,412

4,143,357

10Subsequent events

None.

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Item 2.Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (“MD&A”) is intended to help the reader understand our results of operations and financial condition. This MD&A is provided as a supplement to, and should be read in conjunction with, our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report. Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S. GAAP”) and unless otherwise indicated are presented in U.S. dollars.

Overview

We are a leader in the field of gene therapy and seek to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a pipeline of innovative gene therapies, including 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. We produce our Adeno-associated virus (“AAV”) -based gene therapies in our own facilities with a proprietary, commercial-scale, current good manufacturing practices (“cGMP”)-compliant, manufacturing process. We believe our Lexington, Massachusetts-based facility is one of the world’s most versatile gene therapy manufacturing facilities.

Business Developments

Below is a summary of our recent significant business developments:

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 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. 

17

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 variants of the Sars-CoV 2 virus causing Covid have been identified.

a)Workplace and employees

Throughout the pandemic, we have been implementing measures to address the impact of Covid on our business. We have implemented a series of protocols governing the operations of our Lexington facility to comply with the requirements of the various orders and guidance from the Commonwealth of Massachusetts and other related orders, guidance, laws, and regulations. We continue to monitor local government rules and recommendations and office protocols will be aligned with these rules and recommendations.

Effective 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. Currently, these have not materially impacted our development timelines and we continue to adapt to the current environment to minimize the effect to our business. However, we may experience more pronounced disruptions in our operations in the future.

Russian-Ukrainian war

Our business is not directly impacted by the war as we do not operate in either Russia or the Ukraine. However, the war might potentially amplify the disruptive impact of the Covid pandemic.

18

Intellectual Property

On May 11, 2021, Pfizer, Inc. filed three petitions at the USPTO seeking Inter Partes Review of U.S. Patent Nos. 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. In August 2021, we filed our responses asking the USPTO to deny institution of the IPR 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 March 31, 

    

2022

    

2021

(in thousands)

Total revenues

$

1,792

$

454

Research and development expenses

(45,003)

(32,656)

Selling, general and administrative expenses

(10,987)

(12,376)

Net loss

(46,678)

(41,556)

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 CNS diseases;
continue to expand our employee base to support research and development, as well as general and administrative functions;
acquire or in-license rights to new therapeutic targets or product candidates;
continue to expand, enhance and optimize our technology platform, including our manufacturing capabilities, next-generation viral vectors and promoters, and other enabling technologies;
maintain, expand, and protect our intellectual property portfolio, including in-licensing additional intellectual property rights from third parties; and
make potential future milestone payments related to the acquisition of Corlieve, if any.

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

19

Critical Accounting Policies and Estimates

In preparing our consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the SEC we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. A summary of our critical accounting policies as well as a discussion of our critical accounting estimates are presented in our Annual Report. There were no material changes to our critical accounting policies during the 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

We expense research and development (“R&D”) expenses as incurred. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:

employee-related expenses, including salaries, benefits, travel and share-based compensation expense;
costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors;
costs incurred to conduct characterization, consistency and comparability studies;
costs incurred for the development and improvement of our manufacturing processes and methods;
costs associated with research activities for enabling technology platforms, such as next-generation vectors, promoters and re-administration of gene therapies;
costs associated with the rendering of collaboration services as well as the continued development of Product between the Signing Date and Closing;
facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and
changes in the fair value of liabilities recorded in relation to our acquisition of Corlieve.

Our research and development expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:

the scope, rate of progress and expense of our research and development activities;
our ability to successfully manufacture and scale-up production;
clinical trial protocols, speed of enrollment and resulting data;
the effectiveness and safety of our product candidates;
the timing of regulatory approvals; and
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.

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Selling, general and administrative expenses

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

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

    

2022

    

2021

    

2022 vs 2021

(in thousands)

Total revenues

$

1,792

$

454

$

1,338

Operating expenses:

Research and development expenses

(45,003)

(32,656)

(12,347)

Selling, general and administrative expenses

(10,987)

(12,375)

1,388

Total operating expenses

(55,990)

(45,031)

(10,959)

Other income

311

352

(41)

Other expense

(193)

(233)

40

Loss from operations

(54,080)

(44,458)

(9,622)

Other non-operating items, net

6,786

3,115

3,671

Net loss before income tax income / (expense)

$

(47,294)

$

(41,343)

$

(5,951)

Income tax income / (expense)

616

(213)

829

Net loss

$

(46,678)

$

(41,556)

$

(5,122)

Revenue

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

Three months ended March 31, 

    

2022

    

2021

    

2022 vs 2021

(in thousands)

Collaboration revenue CSL Behring

$

1,436

$

$

1,436

Collaboration revenue BMS

356

454

(98)

Total revenues

$

1,792

$

454

$

1,338

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Research and development expenses

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.