Table of Contents
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, 2026
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 25a
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 Stock Market LLC (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 May 1, 2026, the registrant had 63,067,485 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
18
Item 3
Quantitative and Qualitative Disclosures About Market Risk
31
Item 4
Controls and Procedures
PART II – OTHER INFORMATION
Legal Proceedings
32
Item 1A
Risk Factors
Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities
33
Defaults Upon Senior Securities
Mine Safety Disclosures
Item 5
Other Information
Item 6
Exhibits
SPECIAL CAUTIONARY NOTICE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains “forward-looking statements” within the meaning of the 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 involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. These forward-looking statements include, without limitation, statements concerning: our financial position and performance, revenues, costs, expenses, liquidity, uses of cash and capital requirements; our need for additional financing or the time period for which our existing cash resources will be sufficient to meet our operating requirements; the success, progress, number, scope, cost, duration, timing or results of our research and development activities, preclinical and clinical trials, including the timing for initiation, completion or availability of results from any preclinical studies and clinical trials or for the submission, review or approval of any regulatory filing, including the timing of the Company’s Biologics License Application for AMT-130 to the United States Food and Drug Administration (the “FDA”) and Marketing Authorization Application for AMT-130 to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (“MHRA”); the anticipated or ultimate outcome, and timing, of interactions with regulatory agencies, including the FDA and MHRA; the timing of, and our ability to, obtain and maintain regulatory approvals for any of our product candidates; the potential benefits that may be derived from any of our product candidates; our strategies, prospects, plans, goals, expectations, forecasts or objectives; the success of our collaborations with third parties; our ability to identify and develop new product candidates and technologies; our intellectual property position; our commercialization, marketing and manufacturing capabilities and strategy, including plans for potential commercialization of AMT-130; our ability to achieve long-term growth; and developments and projections relating to our competitors in the industry.
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 (the “SEC”), including our most recent Annual Report on Form 10-K filed with the SEC on March 2, 2026 (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 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 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
UNAUDITED CONSOLIDATED BALANCE SHEETS
March 31,
December 31,
2026
2025
(in thousands, except share and per share amounts)
Current assets
Cash and cash equivalents
$
139,994
80,240
Current investment securities
446,556
542,301
Accounts receivable
3,562
5,863
Prepaid expenses
16,589
20,506
Other current assets and receivables
9,043
7,076
Total current assets
615,744
655,986
Non-current assets
Property, plant and equipment, net
12,014
13,800
Other investments
30,150
30,237
Operating lease right-of-use assets
12,261
12,525
Intangible assets, net
69,990
72,790
Goodwill
24,811
25,355
Deferred tax assets, net
8,194
8,654
Other non-current assets
5,542
5,561
Total non-current assets
162,962
168,922
Total assets
778,706
824,908
Current liabilities
Accounts payable
4,399
5,170
Accrued expenses and other current liabilities
43,201
41,292
Liability related to pre-funded warrants
8,605
12,595
Current portion of operating lease liabilities
2,992
3,862
Total current liabilities
59,197
62,919
Non-current liabilities
Long-term debt
49,942
49,699
Liability from royalty financing agreement
482,334
473,199
Operating lease liabilities, net of current portion
10,388
9,832
Contingent consideration
17,029
18,736
Deferred tax liability, net
7,796
7,967
Other non-current liabilities, net of current portion
2,677
3,655
Total non-current liabilities
570,166
563,088
Total liabilities
629,363
626,007
Commitments and contingencies
Shareholders' equity
Ordinary shares, €0.05 par value: 80,000,000 shares authorized at March 31, 2026, and December 31, 2025, and 63,033,249 and 62,336,717 shares issued and outstanding at March 31, 2026, and December 31, 2025, respectively.
3,728
3,688
Additional paid-in-capital
1,587,971
1,582,371
Accumulated other comprehensive loss
(59,885)
(58,222)
Accumulated deficit
(1,382,471)
(1,328,936)
Total shareholders' equity
149,343
198,901
Total liabilities and shareholders' equity
The accompanying notes are an integral part of these unaudited consolidated financial statements.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
Three months ended March 31,
License revenues
1,567
Total revenues
Operating expenses:
Cost of license revenues
(219)
(197)
Research and development expenses
(29,176)
(36,140)
Selling, general and administrative expenses
(20,068)
(10,908)
Total operating expenses
(49,463)
(47,245)
Other income
1,632
8,306
Other expense
(1,451)
(1,959)
Loss from operations
(45,720)
(39,331)
Interest income
5,229
4,127
Interest expense
(14,031)
(15,109)
Foreign currency (losses) / gains, net
(2,294)
7,172
Other non-operating gains, net
3,769
—
Loss before income tax expense
(53,047)
(43,141)
Income tax expense
(488)
(496)
Net loss
(53,535)
(43,637)
Other comprehensive loss:
Foreign currency translation losses, net
(1,663)
(1,005)
Total comprehensive loss
(55,198)
(44,642)
Loss per ordinary share - basic and diluted
Basic and diluted net loss per ordinary share
(0.85)
(0.82)
Weighted average shares - basic and diluted
62,742,847
53,110,580
3
UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITYFOR THE THREE MONTHS ENDED MARCH 31, 2026 AND 2025
Accumulated
Additional
other
Total
Ordinary shares
paid-in
comprehensive
shareholders’
No. of shares
Amount
capital
loss
deficit
equity
Balance at December 31, 2024
48,988,087
2,945
1,173,068
(52,800)
(1,129,965)
(6,752)
Loss for the period
Other comprehensive loss, net
Follow-on public offering
5,073,529
261
80,250
80,511
Exercises of share options
15,928
158
159
Restricted and performance share units distributed during the period
620,935
(33)
Share-based compensation expense
4,410
Balance at March 31, 2025
54,698,479
3,240
1,257,853
(53,805)
(1,173,602)
33,686
Balance at December 31, 2025
62,336,717
25,308
384
385
Restricted share units distributed during the period
657,285
38
(38)
Issuance of ordinary shares relating to employee stock purchase plan
13,939
164
165
5,090
Balance at March 31, 2026
63,033,249
4
UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
Cash flows from operating activities
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization
2,956
4,527
Amortization of discount on investment securities
(4,335)
(2,117)
Royalty financing agreement interest expense, net of interest paid
7,173
8,358
Deferred tax expense
458
496
Changes in fair value of contingent consideration
(1,338)
1,216
Changes in fair value of liability related to pre-funded warrants
(3,769)
Unrealized foreign exchange losses / (gains), net
2,178
(7,105)
Other items, net
(242)
(3,971)
Changes in operating assets and liabilities:
Accounts receivable, prepaid expenses, and other current assets and receivables
3,780
(2,979)
(557)
(2,093)
Accrued expenses, other liabilities, and operating leases
3,898
(1,205)
Net cash used in operating activities
(38,243)
(44,100)
Cash flows from investing activities
Proceeds on maturity of debt securities
98,700
85,000
Investment in debt securities
(64,775)
Purchases of property, plant, and equipment
(140)
(126)
Net cash generated from investing activities
98,560
20,099
Cash flows from financing activities
Proceeds from follow-on public offering of ordinary shares, net of issuance costs
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans
550
Net cash generated from financing activities
80,670
Currency effect on cash, cash equivalents and restricted cash
(1,132)
1,660
Net increase in cash, cash equivalents and restricted cash
59,735
58,329
Cash, cash equivalents and restricted cash at the beginning of period
81,801
160,329
Cash, cash equivalents and restricted cash at the end of period
141,536
218,658
217,229
Restricted cash related to leasehold and other deposits
1,542
1,429
Total cash, cash equivalents and restricted cash
Supplemental cash flow disclosures:
Cash paid for interest
(6,720)
(6,491)
5
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
1General business information
uniQure N.V. (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 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 Dutch Chamber of Commerce (Kamer van Koophandel) in Amsterdam, the Netherlands under number 54385229. The Company’s headquarters are in Amsterdam, the Netherlands, its registered office is located at Paasheuvelweg 25a, 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 (“U.S. GAAP”) and applicable rules and regulations of the United States Securities and Exchange Commission (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 Updates of the Financial Accounting Standards Board.
The unaudited consolidated financial statements are presented in United States (“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, 2026 are not necessarily indicative of the results to be expected for the full year ending December 31, 2026, 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 on Form 10-K for the year ended December 31, 2025 filed by the Company with the SEC on March 2, 2026 (the “Annual Report”).
6
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 liabilities at the date of the financial statements and reported amounts of revenues, expenses and tax related items during the reporting period. Actual results could differ from those estimates.
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, 2025, and the notes thereto, which are included in the Annual Report. There have been no material changes in the Company’s significant accounting policies during the three months ended March 31, 2026.
2.5Recent accounting pronouncements
There have been no new accounting pronouncements or changes to accounting pronouncements during the three months ended March 31, 2026, as compared to the recent accounting pronouncements described in Note 2.3.29 of the Annual Report, which could be expected to materially impact the Company’s unaudited consolidated financial statements.
The following tables summarize the Company’s investments in government debt securities as of March 31, 2026 and December 31, 2025:
At March 31, 2026
Amortized cost
Gross unrealized holding gains
Gross unrealized holding losses
Estimated fair value
Current investments securities:
Government debt securities (held-to-maturity)
295
446,851
At December 31, 2025
308
542,609
The Company invests in short-term U.S. and European government debt securities with high investment credit ratings. The U.S. and European government bonds are U.S. dollar and euro (€, or EUR) denominated, respectively. Investment securities with original maturities of 90 days or less when purchased are presented within cash and cash equivalents and measured at amortized cost. Inputs to the fair value of the investments are considered Level 2 inputs.
As of March 31, 2026 and December 31, 2025, no expected credit loss was identified with respect to the investment securities.
7
Fair value measurement
The Company measures certain financial assets and liabilities at fair value, either upon initial recognition or for subsequent accounting or reporting. ASC 820, Fair Value Measurement requires disclosure of the 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 from licensing and collaboration partners, other assets, accounts payable, accrued expenses and other current liabilities reflected in the Unaudited Consolidated Balance Sheets approximate their fair values due to their short-term maturities.
The Company’s material financial assets include cash and cash equivalents, restricted cash and investment securities. Cash and cash equivalents and restricted cash are measured at fair value using Level 1 inputs. Restricted cash is included in Other non-current assets within the unaudited Consolidated Balance Sheets. Investment securities are measured at amortized cost.
8
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, 2026 and December 31, 2025:
Quoted pricesin activemarkets(Level 1)
Significant otherobservableinputs(Level 2)
Significantunobservableinputs(Level 3)
Classification in Unaudited ConsolidatedBalance Sheets
Assets:
Restricted cash
1,561
Liabilities:
Consideration for post-acquisition services
638
Other non-current liabilities
19,374
31,969
580
17,609
26,214
The Company is required to pay up to EUR 143.1 million (or $164.5 million based on the foreign exchange rate on March 31, 2026) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of the remaining contractually defined milestones related to the development of AMT-260 in connection with the Company’s acquisition of uniQure France SAS.
The fair value of the contingent consideration as of March 31, 2026 was EUR 14.8 million ($17.0 million) (December 31, 2025: EUR 15.9 million ($18.7 million)) using discount rates of approximately 11.5% to 11.8% (December 31, 2025: 11.8%).
If, as of March 31, 2026, the Company had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 47.8 million ($55.0 million). If, as of March 31, 2026, the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.
The following table presents the changes in fair value of the contingent consideration (presented within non-current liabilities) between December 31, 2025 and March 31, 2026:
Amount of
contingent
consideration
Unrealized change in fair value (presented within Research and development expenses)
Currency translation effects
(369)
9
The Company classified the total contingent consideration liability as non-current as of March 31, 2026 and December 31, 2025. The classification of the contingent consideration within the Company’s Unaudited Consolidated Balance Sheets between current and non-current liabilities is based upon the Company’s best estimate of the timing of settlement of the remaining relevant milestones.
In September 2025, the Company completed a follow-on public offering which included the issuance of pre-funded warrants to purchase 526,316 of the Company’s ordinary shares at the public offering price of $47.50 per ordinary share, less a $0.0001 per ordinary share exercise price for each pre-funded warrant (“Pre-Funded Warrant(s)”). The obligation to issue ordinary shares upon the exercise of the Pre-Funded Warrants is classified as a liability related to pre-funded warrants.
The Pre-Funded Warrants are not exchange-quoted and are measured using a valuation technique whose significant inputs are observable; therefore the fair value measurement is classified within Level 2 of the fair value hierarchy.
Liability related
to pre-funded
warrants
Unrealized change in fair value (presented within Other non-operating gains, net)
(221)
Accrued expenses and other current liabilities include the following items:
Accruals for goods received from and services provided by vendors-not yet billed
23,493
20,490
Personnel-related accruals and liabilities
10,126
9,941
Current portion of firm purchase commitment liability (refer to Note 13, "Commitments and contingencies")
5,796
5,102
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 7, "Royalty Financing Agreement")
5,523
Other current liabilities
224
236
6Long-term debt
The total principal outstanding as of March 31, 2026 under the Company's amended venture debt loan facility with Hercules Capital, Inc. (the “2025 Amended Facility”) was $50.0 million. The amortized cost, including interest due presented as part of Accrued expenses and other current liabilities, was $50.3 million as of March 31, 2026, compared to $50.1 million as of December 31, 2025, and is recorded net of discount and debt issuance costs.
The foreign currency loss on the loan facility in the three months ended March 31, 2026 was $1.1 million, compared to a foreign currency gain of $2.1 million during the same period in 2025. Interest expense during the three months ended March 31, 2026 was $1.4 million, compared to $1.8 million during the same period in 2025.
During the three months ended March 31, 2026, there were no material changes to the terms, available borrowing tranches, covenants, or collateral arrangements of the 2025 Amended Facility as described in Note 11, “Long-term debt” to the consolidated financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. As of March 31, 2026, the Company was in compliance with all applicable covenants under the facility.
10
7Royalty Financing Agreement
During the three months ended March 31, 2026, there were no material changes to the terms of the royalty purchase agreement entered into in May 2023 (the “Royalty Financing Agreement”) with HemB SPV, L.P. (the “Purchaser”), as described in Note 12, “Royalty Financing Agreement” within the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2025.
The following table presents the changes in the liability from royalty financing agreement for the period from December 31, 2025 to March 31, 2026:
Amount of liability
Balance as of December 31, 2025 (includes $5.5 million presented as "Accrued expenses and other current liabilities")
478,722
Royalty payments to the Purchaser
(5,523)
Interest expense for the period
12,621
Balance as of March 31, 2026 (includes $3.6 million presented as "Accrued expenses and other current liabilities")
485,820
The Company records the debt at amortized cost using the effective interest rate method based on projected cash flows. As of March 31, 2026, the effective interest rate is expected to be within a range of 10.5% to 12.0% per annum (March 31, 2025: 12.0% to 13.5%). Interest expense recorded in connection with the agreement was $12.6 million and $13.3 million for the three months ended March 31, 2026 and 2025, respectively.
8Share-based compensation
The Company’s share-based compensation plans include the amended and restated 2014 Share Incentive Plan (as amended, 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”). As of March 31, 2026, a total of 2,571,395 ordinary shares remain available for issuance under the 2014 Plan.
In June 2018, the Company’s shareholders adopted and approved the uniQure N.V. Employee Stock Purchase Plan (as amended, the “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, as amended. 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 the lower of: (i) 85% of the closing market price on the offering date or (ii) 85% of the closing market price on the purchase date.
2014 Plans and ESPP
Share-based compensation expense recognized by classification included in the Unaudited Consolidated Statements of Operations and Comprehensive Loss in relation to the 2014 Plans and the ESPP for the periods indicated below was as follows:
1,977
2,463
3,113
1,947
11
Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows:
Award type/ESPP
Share options
1,807
1,637
Restricted share units
3,206
2,213
Performance share units
62
560
ESPP
15
As of March 31, 2026, 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 years)
Award type
14,881
2.88
27,279
2.40
21
0.08
42,181
2.57
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 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% of each grant vests one year after 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.
The following table summarizes option activity under the 2014 Plans for the three months ended March 31, 2026:
Options
Number of ordinary shares
Weighted average exercise price
Outstanding at December 31, 2025
4,531,991
17.04
Granted
771,900
9.53
Forfeited
Expired
(11,466)
37.00
Exercised
(25,308)
15.24
Outstanding at March 31, 2026
5,267,117
15.90
Thereof, fully vested, and exercisable on March 31, 2026
2,825,796
20.06
Thereof, outstanding and expected to vest after March 31, 2026
2,441,321
11.09
Outstanding and expected to vest after December 31, 2025
1,999,315
11.81
Total weighted average grant date fair value of options issued during the period (in $ millions)
4.9
12
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:
Assumptions
Expected volatility
95.0%
80.0%
Expected terms
10 years
Risk-free interest rate
4.2% - 4.3%
4.4%
Expected dividend yield
0.0%
RSUs
The following table summarizes the RSU activity for the three months ended March 31, 2026:
Number of
grant-date fair
ordinary shares
value
Non-vested at December 31, 2025
2,395,420
12.15
997,100
10.77
Vested
(657,285)
11.33
(43,468)
15.72
Non-vested at March 31, 2026
2,691,767
11.78
Total weighted average grant date fair value of RSUs granted during the period (in $ millions)
10.7
RSUs are measured at grant date based on the market price of the Company’s ordinary shares and, except for certain grants made to non-executive directors, vest over a three-year period. RSUs granted to non-executive directors vest one year after the date of grant.
PSUs
The Company granted PSUs to certain employees in 2024 and 2026, that, with respect to the 2024 PSUs, will be earned upon the Board of Directors’ assessment of the level of achievement of agreed upon performance targets, and, with respect to the 2026 PSUs, will be earned upon management’s assessment of the level of achievement of agreed upon performance targets, subject, in each case, to the grantee’s continued employment. The fair value of PSUs granted in 2024 and 2026 was determined on the grant date by reference to the market price of the Company’s ordinary shares.
The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable. As of March 31, 2026, the PSUs granted in 2026 were assessed as not probable of achieving the requisite performance conditions in accordance with ASC 718, Compensation-Stock Compensation; therefore, no stock-based compensation expense has been recognized.
The following table summarizes the PSU activity for the three months ended March 31, 2026:
22,500
15.40
13
During the three months ended March 31, 2026, 13,939 ordinary shares were issued under the ESPP compared to nil ordinary shares issued during the same period in 2025. As of March 31, 2026, 72,773 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 ordinary shares as of March 31, 2025.
Segment reporting
The Company is advancing a pipeline of innovative gene therapies seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company manages the operations related to these research and development activities within one operating segment because they rely on a common set of infrastructure, resources and technology of the products and production processes, types of customers, distribution methods and regulatory environment.
The leadership team is identified as the Chief Operating Decision Maker (“CODM”).
The CODM allocates resources to research projects and clinical candidates based on scientific data as well as quantitative and qualitative expected risk-adjusted returns on investment. The CODM uses segment operating loss to monitor that cash operating losses remain within the approved budget.
The accounting policies of the operating segment are the same as those described in the summary of significant accounting policies.
Revenue
Less:
Employee related expenses
(16,786)
(14,216)
Laboratory and development expenses
(14,761)
(15,967)
Professional fees
(4,507)
(2,057)
Information technology system costs
(1,659)
(1,001)
Facility expenses
(1,547)
(1,667)
Other segment items(1)
(3,513)
(969)
Segment operating loss
(39,211)
(34,310)
Reconciliation
Depreciation and amortization expense
(2,956)
(4,527)
(5,090)
(4,410)
Fair value gain / (loss) - contingent consideration
1,338
(1,216)
Fair value gain - liability related to pre-funded warrants
Interest expense - Royalty Financing Agreement
(12,621)
(13,309)
Interest expense - Hercules loan facility
(1,410)
(1,800)
Other reconciling items
199
5,132
Consolidated loss before income tax expense
14
Other investments and concentration of credit risk
The following table summarizes the Company’s other investments as of March 31, 2026 and December 31, 2025:
Carrying amount of investments valued under measurement alternative
15,280
15,615
Convertible note receivable
14,870
14,622
The Company did not recognize any upward adjustments, downward adjustments, or impairments relating to investments carried under the measurement alternative during the three months ended March 31, 2026, and 2025.
Genezen
In 2024, as part of the Company’s divestment of its commercial manufacturing operations to Genezen Holdings Inc. and its subsidiary Genezen MA, Inc. (together “Genezen”), the Company entered into a commercial supply agreement (the “Genezen CSA”), as well as a development and other manufacturing agreement, with Genezen. The Company recorded a balance of prepaid expenses of $12.3 million and $13.9 million as of March 31, 2026 and December 31, 2025, respectively, for services that the Company expects to receive from Genezen. In addition, as of March 31, 2026, the Company holds a convertible note receivable of $14.9 million which is due in September 2029 and for which Genezen is the counterparty (December 31, 2025: $14.6 million). These balances represent a concentration of credit risk, and the Company’s maximum exposure to loss is the carrying value of these assets. No expected credit losses were recognized during the three months ended March 31, 2026 or 2025.
CSL Behring
As of March 31, 2026 and December 31, 2025, accounts receivable from CSL Behring LLC (“CSL Behring”) totaled $3.6 million and $5.9 million, respectively, in each case related to royalty revenue.
Income taxes
The Company recorded a deferred tax expense of $0.5 million related to its U.S. operations during the three months ended March 31, 2026 ($0.5 million for the three months ended March 31, 2025).
The effective income tax rate of 0.9% during the three months ended March 31, 2026 (three months ended March 31, 2025: 1.1%) is substantially lower than the enacted rate of 25.8% in the Netherlands as the Company recorded a valuation allowance against its net deferred tax assets in the Netherlands and a partial valuation allowance against its net deferred tax assets in France.
12Basic and diluted loss per share
Diluted loss per ordinary share is calculated by adjusting the weighted average number of ordinary shares outstanding, assuming conversion of all potentially dilutive ordinary shares. As the Company incurred a loss in the three months ended March 31, 2026 and March 31, 2025, all potentially dilutive ordinary shares would have an antidilutive effect, if converted, and thus have been excluded from the computation of diluted loss per share for the three months ended March 31, 2026 and March 31, 2025. The ordinary 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, 2026 and March 31, 2025, respectively.
The potentially dilutive ordinary shares are summarized below:
Anti-dilutive ordinary share equivalents
Stock options under 2014 Plans
5,283,396
Non-vested RSUs and PSUs
2,714,267
2,242,926
Pre-Funded Warrants
526,316
13,936
Total anti-dilutive ordinary share equivalents
8,521,636
7,526,322
13Commitments and contingencies
In the course of its business, the Company enters as a licensee into contracts with other parties regarding the development and marketing of its pipeline products. Among other payment obligations, the Company is obligated to pay royalties to the licensors based on future sales levels and milestone payments whenever specified development, regulatory and commercial milestones are met. As both future sales levels and the timing and achievement of milestones are uncertain, the financial effect of these agreements cannot be reliably estimated. The Company also has obligations to make future payments that become due and payable upon the collection of milestone payments from CSL Behring. The achievement and timing of these milestones is not fixed or determinable.
As of March 31, 2026, the remaining minimum purchase commitments in connection with the Genezen CSA amount to $16.2 million, with $13.3 million to be paid within the next 12 months. As of March 31, 2026, CSL Behring’s minimum purchase commitments to the Company over the next 12 months, in connection with the development and commercial supply agreement between the Company and CSL Behring (the “CSL Behring CSA”), amount to $16.4 million, of which $16.2 million relates to such minimum purchase commitments to Genezen. In April 2026, the Company entered into agreements with Genezen and CSL Behring to terminate the Genezen CSA and the CSL Behring CSA. Refer to Note 14, “Subsequent events”.
On February 10, 2026, a class action complaint captioned Christopher Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124, was filed against the Company, certain of its executive officers and another party in the United States District Court for the Southern District of New York. The complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended, and Rule 10b-5 promulgated thereunder, on behalf of a putative class of investors who purchased or otherwise acquired the Company’s ordinary shares between September 24, 2025 and October 31, 2025. Plaintiff seeks to recover damages allegedly caused by purported false and misleading statements and omissions with respect to the Company’s Phase I/II study of AMT-130 and the timing of the potential BLA filing for AMT-130. The Company intends to vigorously defend against the claims in this action.
At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. At this time, the Company is unable to predict the outcome of the class action litigation or reasonably estimate a range of possible losses.
16
14Subsequent events
Termination agreements with CSL Behring and Genezen
Since the Company’s July 2024 divestiture of its commercial manufacturing activities to Genezen, the Company had been sourcing etranacogene dezaparvovec-drlb (“HEMGENIX®”) under the Genezen CSA. The Company entered into the Genezen CSA to enable it to meet its obligations to supply HEMGENIX® in accordance with the June 2020 commercialization and license agreement with CSL Behring (the “CSL Behring Agreement”) (refer to Note 10, “Other Investments and concentration of credit risk,” and Note 13, “Commitments and contingencies”) until such time that the HEMGENIX® manufacturing technology had been transferred to CSL Behring or a contract manufacturing organization designated by CSL Behring. The Company, since July 2024, had served as an agent in the sale of HEMGENIX® to CSL Behring as title to HEMGENIX® drug product supply had been directly passing from Genezen to CSL Behring. In April 2026, the Company entered into agreements with CSL Behring and Genezen that provide for i) the termination of its obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA, once the contractually specified batches have been supplied to CSL Behring, which the Company expects to occur in mid-2026, and ii) the designation of Genezen as CSL Behring’s contract manufacturing organization. In addition, the CSL Behring Agreement was amended to terminate certain manufacturing-related terms associated with both the CSL Behring CSA and the Genezen CSA, as well as the Company’s development support that CSL Behring could request from time to time with respect to HEMGENIX®. All other terms of the CSL Behring Agreement remain in full force and effect.
The Company expects that by the time the contractually specified batches have been supplied to CSL Behring, it will i) have fully amortized the carrying amount of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA of $7.3 million as of March 31, 2026, and ii) have fully released the firm purchase commitment liability with a March 31, 2026 carrying amount of $5.8 million.
17
Item 2.Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion of our results of operations and financial condition should be read in conjunction with our unaudited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Quarterly Report on Form 10-Q, including the disclosures under Part II, Item 1A “Risk Factors,” and our audited financial information and the notes thereto included in our Annual Report on Form 10-K (the “Annual Report”). Our unaudited consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the United States (“U.S. GAAP”) and unless otherwise indicated are presented in United States (“U.S.”) dollars.
Overview
We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, refractory mesial temporal lobe epilepsy (“MTLE”), and Fabry disease.
Recent Product Candidate Developments
Huntington’s disease program (AMT-130)
AMT-130 is our novel gene therapy candidate for the treatment of Huntington’s disease, which utilizes our proprietary, gene-silencing miQURE® platform and incorporates a miRNA, specifically designed to silence the huntingtin gene and the potentially highly toxic exon 1 protein fragment.
We are currently conducting Phase I/II clinical trials of AMT-130 in the U.S. and Europe. We completed the enrollment of all 26 patients in the first two cohorts of our U.S. study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023. In 2025, we completed enrollment of all 12 patients in the third cohort, and we treated six patients with the high-dose of AMT-130 in a fourth cohort to evaluate the safety and efficacy of AMT-130 in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.
Data from Phase I/II Clinical Studies
In September 2025, we announced positive topline data from the three-year analysis of the ongoing Phase I/II studies of AMT-130 for the treatment of Huntington’s disease. We analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 36 months of follow-up and were evaluated at that time point. Outcomes for each dose group were compared to a propensity score-matched external control drawn from the Enroll-HD natural history data set (n=940 for high-dose; n=626 for low-dose).
Topline 36-month efficacy results for patients receiving high-dose AMT-130 were as follows (data cutoff as of June 30, 2025):
A mean reduction from baseline in cerebrospinal neurofilament light protein (“CSF NfL”) of -8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies. CSF NfL is a well-characterized, supportive biomarker of neurodegeneration. Elevation in CSF NfL has been shown to be strongly associated with greater clinical severity of Huntington’s disease.
We believe that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the high-dose group, compared to the variable trends observed in the low-dose group, reflect a dose-dependent response to AMT-130.
Various other supportive analyses of the results from the AMT-130 high-dose treatment group, including those using a propensity score-weighted external control and comparisons to the TRACK-HD and PREDICT-HD datasets, were consistent with the primary analysis.
AMT-130 was generally well-tolerated in the Phase I/II studies, with a manageable safety profile at both doses. The most common adverse events in the treatment groups were related to the administration procedure.
19
Regulatory Update
From November 2024 through April 2025, we held three Type B meetings with the U.S. Food and Drug Administration (“FDA”). As part of these interactions, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis of a Biologics License Application (“BLA”) submission under the FDA’s accelerated approval pathway. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and reductions in CSF NfL may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.
In October 2025, we met with the FDA at a pre-BLA meeting to discuss the application for AMT-130. In December 2025, we announced that in the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.
In January 2026, we met with the FDA at a Type A meeting to discuss AMT-130. In March 2026, following receipt of the final meeting minutes from the Type A meeting, we announced that the FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.
We intend to continue engaging with the FDA and have scheduled a Type B meeting with the agency in the second quarter of 2026.
In April 2026, we announced that, following feedback from a pre-submission meeting with the United Kingdom’s (“UK”) Medicines and Healthcare products Regulatory Agency (“MHRA”) regarding AMT-130, we plan to submit a UK Marketing Authorization Application for AMT-130 in the third quarter of 2026.
Temporal lobe epilepsy program (AMT-260)
We are conducting a Phase I/IIa clinical trial, GenTLE, of AMT-260 for the treatment of MTLE in the U.S. GenTLE consists of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and initial efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept data.
In September 2025, we completed enrollment of the first three patients in the first cohort administering AMT-260 to patients with lesions in the non-dominant hemisphere of the brain. Following a review by the independent data monitoring committee (“IDMC”), we expanded the first cohort into MTLE in the dominant hemisphere and initiated a second cohort. We completed enrollment of six patients into the first cohort in 2025. We also initiated enrollment of a second cohort in 2025, which is expected to include an additional six patients.
Fabry disease program (AMT-191)
We are conducting a Phase I/II clinical trial of AMT-191 for the treatment of Fabry disease. The multicenter, open-label clinical trial consists of three dose-ranging cohorts of three or more patients each to assess safety, tolerability, and efficacy of AMT-191 in patients with Fabry disease.
In February 2026, we announced updated preliminary data from the Phase I/II study of AMT-191 for Fabry disease. Updated preliminary safety and exploratory efficacy data included the following, with a data cutoff date as of January 8, 2026:
20
As of February 18, 2026, all 11 dosed patients were withdrawn from ERT.
AMT-191 continued to show a manageable safety profile. No SAEs related to AMT-191 were observed at the 4x1013 gc/kg and 2x1013 gc/kg doses. No additional SAEs were observed at the 6x1013 gc/kg dose beyond the five previously reported in September 2025 in two patients. Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity.
Amyotrophic Lateral Sclerosis (AMT-162)
EPISOD1 is a Phase I/II multi-center, open-label trial of AMT-162 for the treatment of amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”) in the U.S. In September 2025, we voluntarily paused enrollment in EPISOD1 upon the recommendation of the IDMC following a review of available preliminary data related to the safety and efficacy of AMT-162 in the context of a dose limiting toxicity, which resulted in a SAE determined to be related to AMT-162, that was observed in one patient in the second cohort. Following review of the preliminary efficacy and safety data generated from EPISOD1, we have decided to discontinue development of AMT-162. We will continue to collect safety data from the five patients dosed in EPISOD1, consistent with applicable safety and regulatory requirements.
CSL Behring Collaboration
In June 2020, we entered into a commercialization and license agreement with CSL Behring LLC (the “CSL Behring Agreement”) pursuant to which CSL Behring LLC (“CSL Behring”) received exclusive global rights to HEMGENIX®.
We and CSL Behring also entered into a development and commercial supply agreement, pursuant to which, among other things, we agreed to supply the product to CSL Behring until such time that these capabilities are transferred to CSL Behring or its designated contract manufacturing organization (the “CSL Behring CSA”). CSL Behring in September 2022 informed us about their intent to transfer contract manufacturing to a designated third party. In July 2024 as part of our divestment of our commercial manufacturing operations to Genezen Holdings Inc. and its subsidiary Genezen MA, Inc. (together “Genezen”), we entered into a commercial supply agreement to transfer the manufacturing and supply activities to Genezen such that Genezen manufactures and supplies CSL Behring’s commercial demand for HEMGENIX® on our behalf (the “Genezen CSA”).
In April 2026, we entered into agreements with CSL Behring and Genezen that provide for (i) the termination of our obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA, once the contractually specified batches have been supplied to CSL Behring, which we expect to occur in mid-2026, and (ii) the designation of Genezen as CSL Behring’s contract manufacturing organization. In addition, the CSL Behring Agreement was amended to terminate certain manufacturing-related terms associated with both the CSL Behring CSA and the Genezen CSA, as well as our development support that CSL Behring could request from time to time with respect to HEMGENIX®. All other terms of the CSL Behring Agreement remain in full force and effect.
Financial Overview
Key components of our results of operations include the following:
As of March 31, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $586.6 million and $622.5 million, respectively. We had a net loss of $53.5 million in the three months ended March 31, 2026, compared to a net loss of $43.6 million for the same period in 2025. As of March 31, 2026 and December 31, 2025, we had accumulated deficits of $1,382.5 million and $1,328.9 million, respectively. See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.
Critical Accounting Policies and Estimates
In preparing our unaudited consolidated financial statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the “SEC”) we make assumptions, judgments and estimates that can have a significant impact on our net loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. We base our assumptions, judgments and estimates on historical experience and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not clear from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies. A summary of our critical accounting policies, as well as a discussion of our critical accounting estimates, are presented in our Annual Report. There were no material changes to our critical accounting policies during the three months ended March 31, 2026.
We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:
22
Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgment due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of:
A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidates.
Our selling, 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 other income generally consists of payments received to subsidize our research and development efforts and income from the subleasing of our Amsterdam facility and our Lexington, MA research and development facility.
23
Results of Operations
Comparison of the three months ended March 31, 2026 and 2025
The following table presents a comparison of our results of operations for the three months ended March 31, 2026 and 2025:
2026 vs 2025
1,995
(22)
6,964
(9,160)
(2,218)
(6,674)
508
(6,389)
Non-operating expense, net
(7,327)
(3,810)
(3,517)
Net loss before income tax expense
(9,906)
(9,898)
We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended March 31, 2026 and 2025, we recognized $3.6 million and $1.6 million of license revenues, respectively.
R&D expense
R&D expenses for the three months ended March 31, 2026 were $29.2 million, compared to $36.1 million for the same period in 2025. Other research and development expenses are separately classified in the table below. These other expenses are not allocated to specific projects, as they are deployed across multiple projects under development.
Huntington's disease (AMT-130)
7,980
8,230
(250)
Temporal lobe epilepsy (AMT-260)
2,827
2,075
752
Fabry disease (AMT-191)
1,539
2,187
(648)
Amyotrophic lateral sclerosis (AMT-162)
894
1,831
(937)
Programs in preclinical development and platform related expenses
1,134
1,280
(146)
Total direct research and development expenses
14,374
15,603
(1,229)
Employee and contractor-related expenses
9,060
10,159
(1,099)
3,619
4,550
(931)
(486)
Fair value changes related to contingent consideration
(2,554)
Other expenses
1,484
2,149
(665)
Total other research and development expenses
14,802
20,537
(5,735)
Total research and development expenses
29,176
36,140
(6,964)
24
Direct research and development expenses
Huntington’s disease (AMT-130)
In the three months ended March 31, 2026 and 2025, we incurred costs of $8.0 million and $8.2 million respectively, primarily related to clinical trials and Chemistry, Manufacturing, and Controls (“CMC”) development. We incurred costs of $4.5 million and $3.0 million related to clinical trials and CMC development, respectively, compared to costs of $5.7 million and $2.0 million in the comparative period.
In the three months ended March 31, 2026 and March 31, 2025 we incurred costs of $2.8 million and $2.1 million respectively, for the development of AMT-260. We incurred costs of $2.6 million and $0.2 million related to clinical trials and CMC development, respectively, compared to costs of $1.6 million and $0.4 million in the comparative period.
In the three months ended March 31, 2026 and March 31, 2025, we incurred costs of $1.5 million and $2.2 million, respectively, related to our development of AMT-191, primarily in relation to our Phae I/II trial.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the three months ended March 31, 2026 and March 31, 2025, we incurred $0.9 million and $1.8 million of expenses, respectively, primarily in relation to our Phase I/II clinical trial.
Preclinical programs & platform development
In the three months ended March 31, 2026 and March 31, 2025, we incurred $1.1 million and $1.3 million of costs, respectively, related to activities associated with product candidates for various other research programs and technology innovation projects.
Other research & development expenses
25
Selling, general and administrative expenses for the three months ended March 31, 2026 were $20.1 million, compared to $10.9 million for the same period in 2025.
9,855
5,485
4,370
1,903
1,752
1,166
Intellectual property fees
872
283
589
Depreciation and facility costs
337
380
(43)
Information technology costs
520
281
239
1,716
629
1,087
Total selling, general and administrative expenses
20,068
10,908
9,160
Other income and expense
Research and development grants from Dutch authorities
958
1,719
(761)
Sublease income / (expense), net
269
245
Sale of critical reagents to Genezen
6,000
(6,000)
Supply of HEMGENIX® to CSL Behring
(1,185)
(1,525)
340
Other income, net
139
129
Total other items, net
181
6,347
(6,166)
Other non-operating items, net
Our other non-operating items, net, for the three months ended March 31, 2026 and March 31, 2025 were as follows:
1,102
688
Interest expense - Hercules debt facility
390
(9,466)
Total non-operating items, net
26
We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $5.2 million in interest income in the three months ended March 31, 2026, compared to $4.1 million in the same period in the prior year. Our interest income increased by $1.1 million, primarily due to increases in amounts of our investment securities during the three months ended March 31, 2026, compared to the same period in the prior year.
In May 2023, uniQure biopharma B.V. entered into an agreement (the “Royalty Financing Agreement”) with HemB SPV, L.P. to sell certain current and future royalties due to uniQure biopharma B.V. from CSL Behring from the net sales of HEMGENIX® pursuant to the CSL Behring Agreement. We recognized non-cash interest expenses related to the Royalty Financing Agreement of $12.6 million and $13.3 million in the three months ended March 31, 2026 and 2025, respectively.
We recognized interest expense of $1.4 million and $1.8 million in the three months ended March 31, 2026, and March 31, 2025, respectively, related to the Hercules loan facility. Our interest expense decreased by $0.4 million, primarily due to improved terms following the September 2025 amendment as well as a decrease in market interest rates.
We conduct transactions and hold monetary assets and liabilities denominated in foreign currencies, principally the euro and the U.S. dollar. Monetary balances are remeasured at period-end exchange rates, and resulting foreign currency transaction gains and losses are recorded in the Unaudited Consolidated Statements of Operations and Comprehensive Loss as incurred.
We recognized a net foreign currency loss, related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group, of $2.3 million during the three months ended March 31, 2026, compared to a net gain of $7.2 million during the same period in 2025.
We issued pre-funded warrants as part of our September 2025 follow-on public offering. We recognize changes related to a liability recorded in relation to this issuance at fair value. We recognized a $3.8 million gain in the three months ended March 31, 2026 related to a decrease in the fair value of the liability related to pre-funded warrants, compared to nil for the same period in 2025.
We recognized $0.5 million of deferred tax expense in the three months ended March 31, 2026, and $0.5 million of deferred tax expense for the same period in 2025.
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Financial Position, Liquidity and Capital Resources
As of March 31, 2026, we had cash and cash equivalents, restricted cash and investment securities of $588.1 million. We believe our cash and cash equivalents, restricted cash, and investment securities will be sufficient to fund our projected operating expenses into the second half of 2029. Such operating expenses include expenses to fund the ongoing clinical trials of AMT-130, AMT-191 and AMT-260, as well as the potential investment of a material portion of the proceeds from our September 2025 public follow-on offering primarily into advancing certain of our clinical candidates into late state development. The amount and timing of our actual expenditures may vary significantly depending on when we commence, as well as the design of, any Phase III trials for AMT-130, AMT-191, and AMT-260 that we may conduct. We have based our estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, and as such, we may require additional funding.
If the BLA for AMT-130 is approved prior to June 15, 2027, provided that confirmatory trials to the extent and in the manner required to support full approval (if applicable) remain ongoing or are being planned, we could draw down up to $100.0 million under our senior secured term loan facility with Hercules. Additional funding could also include a combination of public equity offerings, collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible. If adequate funds are not available to us on acceptable terms when we need them, we may be unable to pursue further development of our clinical product candidates.
Our current material cash requirements include the following contractual and other obligations:
Debt
As of March 31, 2026, we had an outstanding loan amount owed to Hercules for an aggregate principal amount of $50.0 million. The loan has an interest-only period until October 1, 2028 and we are contractually required to repay the $50.0 million in equal installments between October 1, 2028 and October 1, 2030. The interest-only period will be extended to October 1, 2029 if the BLA for AMT-130 is approved prior to June 15, 2027. The loan will be interest-only until October 1, 2030 if certain commercial milestones are met prior to December 31, 2028. Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $20.7 million, with $5.4 million payable within the next 12 months.
Leases
We have entered into lease arrangements for facilities, including corporate, laboratories and office space. As of March 31, 2026, we had fixed lease payment obligations of $20.9 million, with $4.6 million payable within the next 12 months. Following the closing of the Lexington Transaction, we assigned our lease for our prior manufacturing facility in Lexington, MA to Genezen. As of March 31, 2026, we remain obligated under a guarantee of lease payments of $15.2 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.
Commitments related to uniQure France SAS acquisition (nominal amounts)
In connection with our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractually defined milestones. The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. As of March 31, 2026, our remaining commitment amounts include EUR 160.0 million ($184.0 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and European Union. The timing of achieving these milestones and consequently the timing of payments, as well as whether the milestones will be achieved at all, is generally uncertain. These payments are owed in euro and have been translated at the foreign exchange rate as of March 31, 2026 of $1.15/€1.00. As of March 31, 2026, we expect these obligations will become payable between 2030 and 2034. If and when due, up to 25% of the milestone payments can be settled with our ordinary shares.
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Commitments related to licensors and financial advisors
We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our sale of the exclusive global rights of HEMGENIX® to CSL Behring. We also owe payments to a financial advisor related to certain payments we will collect under the CSL Behring Agreement.
The table below summarizes our consolidated cash flow data for the three months ended March 31, 2026 and 2025:
Cash, cash equivalents and restricted cash at the beginning of the period
Foreign exchange impact
We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Therapeutics Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon closing of the CSL Behring Agreement. We continued to incur losses in the current period. We recorded a net loss of $53.5 million in the three months ended March 31, 2026, compared to a net loss of $43.6 million during the same period in 2025. As of March 31, 2026, we had an accumulated deficit of $1,382.5 million.
Sources of liquidity
From our first institutional venture capital financing in 2006 through to the current period, we have funded our operations primarily through private and public placements of equity securities, debt securities, pre-funded warrants, and payments from our collaboration partners, as well as $370.1 million through the sale of a portion of royalties due from our collaboration partner CSL Behring in 2023. Between July 2021 and July 2023, we collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL Behring and other milestones collected from CSL Behring, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Agreement) on net sales of HEMGENIX®.
We are subject to certain covenants under the senior secured term loan facility with Hercules 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 senior secured term loan facility with Hercules may limit our ability to obtain debt financing.
To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all. If financing is not available when needed, including through debt or equity financings, or is available only on unfavorable terms, we may be unable to meet our cash needs. If we raise additional funds through collaborations, strategic alliances or marketing, distribution, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows.
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Net cash used in operating activities was $38.2 million for the three months ended March 31, 2026 and consisted of a net loss of $53.5 million, adjusted for non-cash items including depreciation and amortization expense of $3.0 million, amortization of the discount on investment securities of $4.3 million, share-based compensation expense of $5.1 million, $7.2 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $0.5 million, changes in the fair value of contingent consideration of $1.3 million, changes in the fair value of the liability related to pre-funded warrants of $3.8 million and unrealized foreign exchange losses of $2.2 million. Net cash used in operating activities also included favorable changes in operating assets and liabilities of $7.1 million. There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $3.8 million. There was a net increase in accounts payable, accrued expenses, other liabilities, income taxes payable and operating leases of $3.3 million.
Net cash used in operating activities was $44.1 million for the three months ended March 31, 2025 and consisted of net loss of $43.6 million adjusted for non-cash items, including depreciation and amortization expense of $4.5 million, amortization of the discount on investment securities of $2.1 million, share-based compensation expense of $4.4 million, $8.4 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $0.5 million, changes in the fair value of contingent consideration of $1.2 million, and unrealized foreign exchange gains of $7.1 million. Net cash used in operating activities also included unfavorable changes in operating assets and liabilities of $6.3 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $3.0 million. There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $3.3 million.
In the three months ended March 31, 2026, we generated $98.6 million from our investing activities compared to $20.1 million generated during the same period in 2025.
Proceeds from maturity of debt securities
Capital expenditures
In the three months ended March 31, 2026, net cash generated from financing activities was $0.6 million, compared to net cash generated from financing activities of $80.7 million in the same period in 2025.
Funding requirements
Our future capital requirements will depend on many factors, including but not limited to:
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Item 3.Quantitative and Qualitative Disclosures about Market Risk
We are exposed to a variety of financial risks in the normal course of our business, including market risk (including currency, price, and interest rate risk), credit risk and liquidity risk. Our overall risk management program focuses on the preservation of capital and the unpredictability of financial markets and has sought to minimize potential adverse effects on our financial performance and position.
Our market risks and exposures to such market risks during the three months ended March 31, 2026, have not materially changed from our market risks and our exposure to market risk discussed in Part II, Item 7A of our Annual Report.
Item 4.Controls and Procedures
Evaluation of Disclosure Controls and Procedures
Our management, with the participation of our chief executive officer (“CEO”) and chief financial officer (“CFO”), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”) as of March 31, 2026. Based on such evaluation, our CEO and CFO concluded that as of March 31, 2026, our disclosure controls and procedures were effective to ensure that information required to be disclosed by it in reports the Company files or submits under the Exchange Act is recorded, processed, summarized and reported within the time periods specified in the rules and forms of the SEC, and that such material information is accumulated and communicated to the Company’s management, including its Principal Executive Officer and Principal Financial Officer, to allow timely decisions regarding required disclosure. Because of the inherent limitations in all control systems, any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and management necessarily is required to apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Furthermore, the Company’s controls and procedures can be circumvented by the individual acts of some persons, by collusion of two or more people or by management override of such control, and misstatements due to error or fraud may occur and not be detected on a timely basis.
Changes in Internal Control over Financial Reporting
During the period covered by this Quarterly Report on Form 10-Q, we implemented a new enterprise resource planning (“ERP”) system in January 2026.
In connection with the ERP implementation, we adapted our internal control activities, including those related to information produced by the entity, user access and segregation of duties, roles and permissions, and approval workflows. The implementation also impacted processes supporting the maintenance of financial records, transaction processing, and financial reporting.
Except for the ERP system implementation described above, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
Part II – OTHER INFORMATION
Item 1.Legal Proceedings
On February 10, 2026, a class action complaint captioned Christopher Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124, was filed against us, certain of our executive officers and another party in the United States District Court for the Southern District of New York. The complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended, and Rule 10b-5 promulgated thereunder, on behalf of a putative class of investors who purchased or otherwise acquired our ordinary shares between September 24, 2025 and October 31, 2025. Plaintiff seeks to recover damages allegedly caused by purported false and misleading statements and omissions with respect to our Phase I/II study of AMT-130 and the timing of the potential BLA filing for AMT-130. We intend to vigorously defend against the claims in this action.
At each reporting date, we evaluate whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. At this time, we are unable to predict the outcome of the class action litigation or reasonably estimate a range of possible losses.
Item 1A.Risk Factors
For a discussion of our risks, please see “Item 1A. Risk Factors” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025. There have been no material changes to the risk factors disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025.
Item 2.Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities
None.
Item 3.Defaults Upon Senior Securities
Item 4.Mine Safety Disclosures
Not applicable.
Item 5.Other Information
During the three months ended March 31, 2026, the following adopted a “Rule 10b5-1 trading arrangement” (as defined in Item 408(a) of Regulation S-K) that is intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act and our policies on insider trading:
Name & Title
Date Adopted(1)
Aggregate Number of Ordinary Shares to be Purchased or Sold Pursuant to Trading Arrangement
Expiration Date(2)
Madhavan Balachandran, Non-executive Director
March 10, 2026
21,685
December 31, 2026
(1) Date of adoption of this Rule 10b5-1 trading arrangement is in accordance with applicable SEC rules and regulations. The first trade pursuant to this Rule 10b5-1 trading arrangement will be, in accordance with applicable SEC rules and regulations, on a date after the date of adoption of the Rule 10b5-1 trading arrangement, to the extent triggered under its terms.
(2) The Rule 10b5-1 trading arrangement is scheduled to expire on the date listed in the table, subject to earlier termination upon the sale of all shares subject to the Rule 10b5-1 trading arrangement, or as otherwise provided in the Rule 10b5-1 trading arrangement.
Other than those disclosed above, none of our directors or officers adopted, modified, or terminated a “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement,” in each case as defined in Item 408 of Regulation S-K, during the three months ended March 31, 2026.
Item 6.Exhibits
See the Exhibit Index immediately preceding the signature page to this Quarterly Report on Form 10-Q for a list of exhibits filed or furnished with this report, which Exhibit Index is incorporated herein by reference.
EXHIBIT INDEX
10.1*+
Termination and Amendment Agreement, dated April 15, 2026, by and between uniQure biopharma B.V. and CSL Behring LLC.
31.1*
Certification of Chief Executive Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the Securities and Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
31.2*
Certification of Chief Financial Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the Securities and Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.
32.1±
Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.
101*
The following financial information from our Quarterly Report on Form 10-Q for the period ended March 31, 2026, filed with the Securities and Exchange Commission on May 5, 2026, is formatted in Inline Extensible Business Reporting Language (“iXBRL”): (i) Consolidated Balance Sheets; (ii) Consolidated Statements of Operations and Comprehensive Loss; (iii) Consolidated Statements of Shareholders’ Equity; (iv) Consolidated Statements of Cash Flows; and (v) Notes to Consolidated Financial Statements (tagged as blocks of text).
104*
The cover page from our Quarterly Report on Form 10-Q for the period ended March 31, 2026, filed with the Securities and Exchange Commission on May 5, 2026, is formatted in Inline Extensible Business Reporting Language (“iXBRL”).
* Filed herewith.
± Furnished herewith.
+Portions of this exhibit have been omitted pursuant to Item 601(b)(10)(iv) of Regulation S-K.
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
UNIQURE N.V.
By: /s/ Matthew Kapusta
Matthew Kapusta
Chief Executive Officer
(Principal Executive Officer)
By: /s/ Christian Klemt
Christian Klemt
Chief Financial Officer
(Principal Financial and Accounting Officer)
Date: May 5, 2026
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