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) |
(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
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.
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Part I – FINANCIAL INFORMATION
Item 1.Financial Statements
uniQure N.V.
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 | $ | 778,706 | $ | 824,908 | ||
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS
Three months ended March 31, | ||||||
| 2026 | | 2025 | |||
(in thousands, except share and per share amounts) | ||||||
License revenues | $ | 3,562 | $ | 1,567 | ||
Total revenues | 3,562 | 1,567 | ||||
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 | ||||
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY
FOR THE THREE MONTHS ENDED MARCH 31, 2026 AND 2025
Accumulated | |||||||||||||||||
Additional | other | Total | |||||||||||||||
Ordinary shares | paid-in | comprehensive | Accumulated | shareholders’ | |||||||||||||
| No. of shares | | Amount | | capital | | loss | | deficit | | equity | ||||||
(in thousands, except share and per share amounts) | |||||||||||||||||
Balance at December 31, 2024 | 48,988,087 | $ | 2,945 | $ | 1,173,068 | $ | (52,800) | $ | (1,129,965) | $ | (6,752) | ||||||
Loss for the period | — | — | — | — | (43,637) | (43,637) | |||||||||||
Other comprehensive loss, net | — | — | — | (1,005) | — | (1,005) | |||||||||||
Follow-on public offering | 5,073,529 | 261 | 80,250 | — | — | 80,511 | |||||||||||
Exercises of share options | 15,928 | 1 | 158 | — | — | 159 | |||||||||||
Restricted and performance share units distributed during the period | 620,935 | 33 | (33) | — | — | — | |||||||||||
Share-based compensation expense | — | — | 4,410 | — | — | 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 | $ | 3,688 | $ | 1,582,371 | $ | (58,222) | $ | (1,328,936) | $ | 198,901 | ||||||
Loss for the period | — | — | — | — | (53,535) | (53,535) | |||||||||||
Other comprehensive loss, net | — | — | — | (1,663) | — | (1,663) | |||||||||||
Exercises of share options | 25,308 | 1 | 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 | 1 | 164 | — | — | 165 | |||||||||||
Share-based compensation expense | — | — | 5,090 | — | — | 5,090 | |||||||||||
Balance at March 31, 2026 | 63,033,249 | $ | 3,728 | $ | 1,587,971 | $ | (59,885) | $ | (1,382,471) | $ | 149,343 | ||||||
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS
Three months ended March 31, | ||||||
| 2026 | | 2025 | |||
(in thousands) | ||||||
Cash flows from operating activities | ||||||
Net loss | $ | (53,535) | $ | (43,637) | ||
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) | ||||
Share-based compensation expense | 5,090 | 4,410 | ||||
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) | ||||
Accounts payable | (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 | — | 80,511 | ||||
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans | 550 | 159 | ||||
Net cash generated from financing activities | 550 | 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 | ||
Cash and cash equivalents | $ | 139,994 | $ | 217,229 | ||
Restricted cash related to leasehold and other deposits | 1,542 | 1,429 | ||||
Total cash, cash equivalents and restricted cash | $ | 141,536 | $ | 218,658 | ||
Supplemental cash flow disclosures: | ||||||
Cash paid for interest | $ | (6,720) | $ | (6,491) | ||
The accompanying notes are an integral part of these unaudited consolidated financial statements.
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uniQure N.V.
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”).
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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.
3 | Current investment securities |
The following tables summarize the Company’s investments in government debt securities as of March 31, 2026 and December 31, 2025:
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.
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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. 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.
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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:
| a) | Contingent consideration |
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:
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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.
| b) | Liability related to pre-funded warrants |
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.
5 | Accrued expenses and other current liabilities |
Accrued expenses and other current liabilities include the following items:
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.
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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:
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:
| Three months ended March 31, | |||||
| 2026 | | 2025 | |||
(in thousands) | ||||||
Research and development expenses | 1,977 | 2,463 | ||||
Selling, general and administrative expenses | 3,113 | 1,947 | ||||
Total | $ | 5,090 | $ | 4,410 | ||
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Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows:
Three months ended March 31, | ||||||
| 2026 | | 2025 | |||
(in thousands) | ||||||
Award type/ESPP | ||||||
Share options | $ | 1,807 | $ | 1,637 | ||
Restricted share units | 3,206 | 2,213 | ||||
Performance share units | 62 | 560 | ||||
ESPP | 15 | — | ||||
Total | $ | 5,090 | $ | 4,410 | ||
As of March 31, 2026, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans were:
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% 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:
Total weighted average grant date fair value of options issued during the period (in $ millions) | | | $ | 4.9 |
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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, | ||||
| 2026 | | 2025 | |
Assumptions | ||||
Expected volatility | 95.0% | 80.0% | ||
Expected terms | 10 years | 10 years | ||
Risk-free interest rate | 4.2% - 4.3% | 4.4% | ||
Expected dividend yield | 0.0% | 0.0% | ||
RSUs
The following table summarizes the RSU activity for the three months ended March 31, 2026:
RSUs | |||||
| | Weighted average | |||
Number of | grant-date fair | ||||
ordinary shares | value | ||||
Non-vested at December 31, 2025 | 2,395,420 | $ | 12.15 | ||
Granted | 997,100 | $ | 10.77 | ||
Vested | (657,285) | $ | 11.33 | ||
Forfeited | (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:
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ESPP
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.
9 | 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.
| (1) | Other segment items included in segment operating loss include costs related to intellectual property and insurance offset by income related to payments received from European authorities to subsidize the Company’s research and development in the Netherlands. |
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10 | 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:
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.
11 | 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.
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The potentially dilutive ordinary shares are summarized below:
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”.
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 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.
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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.
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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.
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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 statistically significant 75% slowing of disease progression as measured by composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) (p=0.003), which met the primary endpoint of the study. Treated patients had a mean change in cUHDRS from baseline of -0.38 compared to a change of -1.52 for patients in the propensity score-matched external control. |
| ● | A statistically significant 60% slowing of disease progression as measured by Total Function Capacity (“TFC”) (p=0.033), which met a key secondary endpoint of the study. Treated patients had a mean change in TFC from baseline of -0.36 compared to a change of -0.88 for patients in the propensity score-matched external control. |
| ● | Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test (“SDMT”), Stroop Word Reading Test (“SWRT”) and Total Motor Score (“TMS”). |
| o | An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of -0.44 compared to a change of -3.73 for patients in the propensity score-matched external control. |
| o | A 113% slowing of disease progression as measured by SWRT (nominal p=0.002), with a mean change in SWRT from baseline of 0.88 compared to a change of -6.98 for patients in the propensity score-matched external control. |
| o | A 59% slowing of disease progression as measured by TMS (nominal p=0.174), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control. |
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.
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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:
| ● | Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 0.34- to 82.2-fold above mean normal range (1.38-8.66 nmol; mean normal of 3.57 nmol) at the lowest dose, 1.6- to 312.52-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose. These increases were durable for the measured time period ranging from the longest follow-up period of more than a year in a treated patient (high-dose cohort) to the shortest follow-up period of four-months in a treated patient (mid-dose cohort). |
| ● | Six of 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”). |
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| ● | Plasma lyso-Gb3 levels were stable post-dose across all dose cohorts, regardless of ERT status through the cutoff date. |
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.
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Financial Overview
Key components of our results of operations include the following:
Three months ended March 31, | |||||||
| 2026 | | 2025 | | |||
(in thousands) | |||||||
Total revenues | $ | 3,562 | $ | 1,567 | |||
Cost of license revenues | (219) | (197) | |||||
Research and development expenses | (29,176) | (36,140) | |||||
Selling, general and administrative expenses | (20,068) | (10,908) | |||||
Net loss | (53,535) | (43,637) | |||||
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.
Research and development expenses
We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development. Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include:
| ● | employee-related expenses, including salaries, benefits, travel and share-based compensation expense; |
| ● | costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors; |
| ● | costs incurred to conduct consistency and comparability studies; |
| ● | costs incurred for the development and improvement of our manufacturing processes and methods; |
| ● | costs associated with research activities for enabling technology platforms; |
| ● | costs associated with the rendering of collaboration services; |
| ● | payments related to identifiable intangible assets without an alternative future use; |
| ● | payments to our licensors for milestones that have been achieved related to our product candidates; |
| ● | facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and, |
| ● | changes in the fair value of liabilities recorded in relation to the acquisition of uniQure France SAS. |
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Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including manufacturing campaigns, regulatory submissions, and enrollment of patients in clinical trials. The successful development of our product candidates is highly uncertain. Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgment 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; |
| ● | clinical trial protocols, speed of enrollment and resulting data; |
| ● | the effectiveness and safety of our product candidates; and, |
| ● | the timing of regulatory approvals. |
A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidates.
Selling, general and administrative expenses
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.
Other items, net
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.
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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:
Three months ended March 31, | |||||||||
| 2026 | | 2025 | | 2026 vs 2025 | ||||
(in thousands) | |||||||||
License revenues | $ | 3,562 | $ | 1,567 | $ | 1,995 | |||
Total revenues | 3,562 | 1,567 | 1,995 | ||||||
Operating expenses: | |||||||||
Cost of license revenues | (219) | (197) | (22) | ||||||
Research and development expenses | (29,176) | (36,140) | 6,964 | ||||||
Selling, general and administrative expenses | (20,068) | (10,908) | (9,160) | ||||||
Total operating expenses | (49,463) | (47,245) | (2,218) | ||||||
Other income | 1,632 | 8,306 | (6,674) | ||||||
Other expense | (1,451) | (1,959) | 508 | ||||||
Loss from operations | (45,720) | (39,331) | (6,389) | ||||||
Non-operating expense, net | (7,327) | (3,810) | (3,517) | ||||||
Net loss before income tax expense | $ | (53,047) | $ | (43,141) | $ | (9,906) | |||
Income tax expense | (488) | (496) | 8 | ||||||
Net loss | $ | (53,535) | $ | (43,637) | $ | (9,898) | |||
License revenues
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.
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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.
Temporal lobe epilepsy (AMT-260)
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.
Fabry disease (AMT-191)
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
| ● | We incurred $9.1 million in personnel and contractor-related expenses in the three months ended March 31, 2026, compared to $10.2 million for the same period in 2025. The decrease was due to a decrease in personnel-related expenses, as well as a decrease in severance costs and reorganization expenses related to the divestment of our commercial manufacturing activities in July 2024, incurred in the first quarter of 2025; |
| ● | We incurred $3.6 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended March 31, 2026 compared to $4.6 million in the same period in 2025. The decrease of $1.0 million was primarily driven by a revision to the estimated useful lives of certain assets, resulting in higher depreciation recognized during the three months ended March 31, 2025, compared with the current period; |
| ● | We recognized a $1.3 million gain in the three months ended March 31, 2026, related to a decrease in the fair value of contingent consideration associated with the acquisition of uniQure France SAS, compared to a $1.2 million loss for the same period in 2025. The decrease was primarily related to changes to the discount rate and expected timing of future achievement of the related remaining milestones; |
| ● | We incurred $2.0 million in share-based compensation expenses in the three months ended March 31, 2026, compared to $2.5 million for the same period in 2025; and, |
| ● | We incurred $1.5 million of other expenses for the three months ended March 31, 2026, compared to $2.1 million for the same period in 2025. |
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Selling, general and administrative expenses
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.
| ● | We incurred $9.9 million in personnel and contractor-related expenses in the three months ended March 31, 2026, compared to $5.5 million in the same period in 2025. The increase in 2026 was primarily the result of a higher number of employees recruited in the second half of 2025 in support of a potential commercial launch of AMT-130; |
| ● | We incurred $3.7 million in professional fees in the three months ended March 31, 2026 compared to $1.9 million in the same period in 2025. The increase was related to fees incurred in relation to legal and commercial services; |
| ● | We incurred $3.1 million in share-based compensation expenses in the three months ended March 31, 2026, compared to $1.9 million in the same period in 2025. The increase was primarily the result of a higher number of grants outstanding in the three months ended March 31, 2026, in comparison with the prior period; and, |
| ● | We incurred $1.7 million in other expenses in the three months ended March 31, 2026, compared to $0.6 million in the same period in 2025. |
Other income and expense
Three months ended March 31, | |||||||||
2026 | 2025 | 2026 vs 2025 | |||||||
(in thousands) | |||||||||
Research and development grants from Dutch authorities | $ | 958 | $ | 1,719 | $ | (761) | |||
Sublease income / (expense), net | 269 | 24 | 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 | 10 | ||||||
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:
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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.
Income tax expense
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:
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
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.
Net cash generated from investing activities
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.
Net cash generated from financing activities
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:
| ● | Investments to develop AMT-130, AMT-191 and AMT-260 beyond completing the ongoing trials; |
| ● | activities to prepare for the potential commercialization of AMT-130 for Huntington’s disease, if we can align on the requirements for marketing authorization with the respective regulatory authorities; |
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| ● | earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones; |
| ● | contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement; |
| ● | the scope, timing, results, and costs of our current and planned clinical trials; |
| ● | the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements; |
| ● | the extent to which we acquire or in-license other businesses, products, product candidates or technologies; |
| ● | the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates; |
| ● | the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates; |
| ● | the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies; |
| ● | the cost, timing and outcome of regulatory reviews associated with our product candidates; |
| ● | our ability to enter into collaboration arrangements in the future; and |
| ● | the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims. |
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.
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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.
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Item 2.Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities
None.
Item 3.Defaults Upon Senior Securities
None.
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:
(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.
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EXHIBIT INDEX
* 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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Exhibit 10.1
Execution version
[***] CERTAIN INFORMATION HAS BEEN EXCLUDED PURSUANT TO REGULATION S-K, ITEM 601(B)(10)(IV) FROM THIS DOCUMENT BECAUSE IT IS BOTH NOT MATERIAL AND IS THE TYPE THAT THE REGISTRANT TREATS AS PRIVATE OR CONFIDENTIAL
Termination and amendment Agreement
This Termination and Amendment Agreement (this “Termination Agreement”) is by and between uniQure biopharma B.V., a Netherlands private company (“uniQure Bio”) and CSL Behring LLC, a limited liability company organized and existing under the laws of Delaware (“CSL”) and entered into as of April 15, 2026 (the “Termination Execution Date”). Each of uniQure Bio and CSL may be referred to in this Termination Agreement individually as a “Party” and collectively as the “Parties”.
WHEREAS, CSL and uniQure Bio are parties to that certain Commercialization and License Agreement executed as of June 24, 2020 (the “CLA”) relating to the commercialization of uniQure Bio’s proprietary gene therapy product known as etranacogene dezaparvovec, consisting of an AAV5 viral vector carrying a gene cassette with the LP-1 promoter and the Padua variant of Factor IX (FIX-Padua) (the “Product”);
WHEREAS, uniQure Bio and CSL are parties to that certain Development and Commercial Supply Agreement executed as of June 24, 2020 (as amended to date, “CSA”) relating to the supply of Product (and other Licensed Products) by uniQure Bio to CSL;
WHEREAS, currently Genezen MA, Inc. (“Genezen”) manufactures and supplies Product to uniQure Bio’s affiliate, uniQure, Inc. (“uniQure US”) under a supply agreement (the “Genezen CSA”) for further supply of such Product by uniQure Bio to CSL under the CSA;
WHEREAS, uniQure Bio and CSL have agreed that (a) CSL will assume responsibility, and uniQure Bio and its Affiliates will have no further responsibility, for manufacturing the Product (and other Licensed Products) for use by and on behalf of CSL pursuant to the terms and conditions of the CLA as amended hereby and (b) such manufacturing will be performed through Genezen as CSL’s approved contract manufacturer under a direct agreement between Genezen and CSL (collectively (a) and (b), the “Manufacturing Responsibility Transfer”);
WHEREAS, in connection with the Manufacturing Responsibility Transfer, CSL and uniQure Bio agree that the CSA expires on the Termination Effective Date (as defined herein) and is deemed terminated on the terms and conditions set forth herein;
WHEREAS, in connection with the Manufacturing Responsibility Transfer, Genezen will transfer certain reagents being used or held for use under the Genezen CSA to CSL; and
WHEREAS, in connection with the Manufacturing Responsibility Transfer, CSL and uniQure Bio have agreed to make certain amendments and modifications to the CLA, and in
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accordance with Section 16.8 (Entire Agreement; Amendments) of the CLA, CSL and uniQure Bio desire to memorialize such amendments and modifications in this Termination Agreement.
NOW, THEREFORE, in consideration of the foregoing premises, the mutual promises and covenants of the Parties contained herein, and other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, the Parties, intending to be legally bound, do hereby agree as follows:
1. | Definitions. Capitalized terms used but not otherwise defined herein shall have the meaning ascribed to them in the CLA or CSA, as applicable. |
2. | Manufacturing Transfer. As used herein, the “Termination Effective Date” shall mean the first date on which both (a) the batches designated as the “Final CSA Batches” in Appendix A have been Manufactured under the CSA and (b) those Final CSA Batches that uniQure Bio determines are suitable for delivery to CSL (the “Final Conforming CSA Batches”) are made available for delivery to CSL. Notwithstanding anything to the contrary in the CLA or CSA (including Section 5.3.3 (Manufacturing Responsibility Transfer Plan) of the CLA), CSL and uniQure Bio agree that the “Manufacturing Responsibility Cutover Date” under the CLA and CSA shall be deemed to occur on the Termination Effective Date. For the avoidance of doubt, the Parties expressly agree that as of and following the Termination Execution Date, (x) uniQure Bio shall have no obligation (i) to Manufacture or accept an order for the Manufacture of any Licensed Products other than the Final CSA Batches, or (ii) to perform or accept an order for any Development or Manufacturing-related services (including, but not limited to, stability testing services, but not including activities reasonably requested to be performed under Article 3 of the CLA), in each case under any agreement, including the CLA and CSA, and (y) CSL shall not request or place any purchase order for Licensed Product or other services under the CSA. Furthermore, the Parties expressly agree that as of the Termination Effective Date, uniQure Bio shall have no obligation to, and CSL assumes responsibility for, Manufacture of the Licensed Products for use by and on behalf of CSL in the Field in the Territory under and pursuant to the CLA. If CSL desires to engage a CMO (other than Genezen) to perform such Manufacture, uniQure Bio shall have the right to approve the use of such CMO if required by, and in accordance with, Section 2.2.2 (Right to Subcontract) of the CLA. |
3. | Termination of CSA. |
(a) | Termination. CSL and uniQure Bio acknowledge that the Term of the CSA expires pursuant to Section 14.1.2 (Term) of the CSA, and the CSA is deemed terminated as of, the Termination Effective Date. CSL and uniQure Bio agree that Section 14.7.1 (Effect of Termination) and Section 14.7.2 (Effect of Termination) of the CSA shall not apply with respect to such termination. |
(b) | Final Supply. The Final Conforming CSA Batches will be delivered to CSL and paid for by CSL in accordance with the Commercial Supply Terms upon confirmation such Final Conforming CSA Batches meet the terms of the CSA. The Parties acknowledge that any batches of Licensed Product subsequent to the Final CSA Batches ([***]) (“Follow-On Product”) will be deemed as fully Manufactured under the supply arrangement between |
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Genezen (or one of its affiliates) and CSL (or one of its Affiliates) entered into in connection with the Manufacturing Responsibility Transfer (the “CSL-Genezen Agreement”) even if Licensed Product or vials of Licensed Product for such subsequent batches are a work-in-process or were Manufactured as of the Termination Effective Date. The Parties further acknowledge that, as between Genezen and uniQure Bio, Genezen is responsible for Manufacturing Licensed Products subsequent to the Final CSA Batches ([***]) and as of the Termination Execution Date, uniQure Bio is not the exclusive manufacturer of Licensed Product for CSL. Neither uniQure Bio nor any of its Affiliates shall have any obligations, responsibilities or liabilities under the CSA or the CLA (including pursuant to Section 11.2 (By uniQure) thereof) with respect to the Manufacture of any Follow-On Product or other Licensed Product or products under the CSL-Genezen Agreement, including, but not limited to, product liability claims.
(c) | Non-Conformance. The Parties acknowledge that if any of the Final CSA Batches do not conform to any of the requirements set forth in Section 8.1 of the CSA, uniQure Bio shall not be required to re-Manufacture any such batches and Genezen shall be responsible for such re-Manufacture pursuant to the CSL-Genezen Agreement. uniQure Bio shall also not be required to re-Manufacture any [***], other than [***], that is Manufactured into [***] prior to the Termination Effective Date, and that do not conform to any of the requirements set forth in Section 8.1 of the CSA; provided however, that Genezen shall re-Manufacture any such batches pursuant to the CSL-Genezen Agreement with no further payment owed by CSL. |
(d) | Survival. Subject to the terms of this Termination Agreement, following the CSA’s termination, Section 14.8 (Survival of Provisions) of the CSA, and the other provisions of the CSA referred to therein, shall survive and continue to apply in accordance with their terms. The Parties also acknowledge and agree that, for any Licensed Product made under the CSA, ownership of any Patent Rights and Know-How associated therewith are governed exclusively by the terms and conditions of the CLA. |
(e) | [***] Product. Any batches of Licensed Product that [***] CSL designated [***] of the Manufacturing Process (as defined in the CSA) for up to [***] under the Manufacturing Process as approved by Regulatory Authorities ([***]) (such period, the “[***]”) are referred to herein as “[***]”. Neither uniQure Bio nor any of its Affiliates shall have any obligation, responsibility or liability (including pursuant to Article 11 (Indemnity and Insurance) of the CSA) for (i) any [***], (ii) any drug product Manufactured [***] or (iii) any batches of Product [***] of the Manufacturing Process ((i), (ii) and (iii), “[***]”), and any obligations, responsibilities or liabilities with respect to [***] shall be allocated between CSL and Genezen pursuant to the CSL-Genezen Agreement. CSL will indemnify, defend and hold harmless the uniQure Indemnitees from and against all Losses incurred in connection with any Third Party Claim to the extent arising or relating to the Exploitation of any [***], including for clarity any product liability claims arising from [***]. The indemnification procedure set forth in Section 11.3 (Indemnification Procedure) shall apply to the indemnity under the preceding sentence as though that indemnity was given under Section 11.1 (By Partner) of the CSA. Except as expressly set forth herein, this Termination Agreement shall have no effect on [***]. |
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(f) | Stability Testing Services. In addition to the Manufacturing and supply of Licensed Products under the CSA, the Parties acknowledge that at CSL’s request, uniQure Bio, itself or through its subcontractor Genezen MA, performed certain stability testing services in relation to units of drug substance and drug product of the Licensed Product pursuant to purchase orders issued by CSL to uniQure Bio. These services shall cease as of the Termination Execution Date and CSL shall pay to uniQure Bio [***] for stability testing services performed in [***] and [***] for stability testing services performed in. uniQure shall provide CSL with an invoice on the Termination Execution Date for such amounts and CSL shall pay such amounts within [***] of receipt of invoice. |
4. | Amendments and Modifications to CLA. |
(a) | Amendments to Article 1 (Definitions). Section 1.108 (“Manufacturing Responsibility Cutover Date”) is hereby amended in full to read as follows: |
“1.108 ‘Manufacturing Responsibility Cutover Date’ means the date of Partner assumption of Manufacturing responsibility, which shall be the “Termination Effective Date” as defined in the Termination and Amendment Agreement between the Parties executed as of April 15, 2026, as may be amended from time to time in accordance with its terms.”
(b) | Partner Manufacturing Technology. CSL will promptly notify uniQure of any modifications to the Manufacturing Process that are notified to a Regulatory Authority. |
(c) | Modifications to Article 4 (Development Program). Notwithstanding anything to the contrary in the CLA, including in Section 4.1.2 (Development Diligence) (first sentence only), Section 4.1.3 (uniQure Additional Development Activities) and Section 4.4 (uniQure Development Plans and Budgets) of the CLA, CSL and uniQure Bio agree that as of the Manufacturing Responsibility Cutover Date, CSL may no longer propose additional Development activities to be performed by uniQure Bio. |
(d) | Amendments to Article 5 (Manufacturing). |
i. | The following sentence is hereby added to become the final sentence of Section 5.2 (Manufacturing Development Plan) of the CLA: |
“As of the Manufacturing Responsibility Cutover Date, (i) the Manufacturing Development Plan will be deemed terminated regardless of the status of [***] and (ii) uniQure shall have no further obligations (x) to allocate capacity for, or perform, activities under the Manufacturing Development Plan, including under this Section 5.2 (Manufacturing Development Plan) and Section 5.3.1 (uniQure Responsibility) or (y) with respect to any costs or expenses associated with the performance of any activities that were set forth in the Manufacturing Development Plan as of the Manufacturing Responsibility Cutover Date.”
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ii.The following sentence is hereby added to become the final sentence of Section 5.3.1 (uniQure Responsibility) of the CLA:
“For clarity, from and after the Manufacturing Responsibility Cutover Date, uniQure shall have no obligations to supply to Partner or its Affiliates the Licensed Products for Development or Commercialization in accordance with this Agreement or to perform any activities that were allocated to uniQure under the Manufacturing Development Plan.”
iii.Section 5.3.3 (Manufacturing Responsibility Transfer Plan) of the CLA is hereby amended as follows (deleted language in strikethrough text):
“5.3.3 Manufacturing Responsibility Transfer Plan. Following the Manufacturing Responsibility Transfer Notice Date, upon Partner’s request, the Parties will collaborate reasonably with each other through the JSC to prepare, as soon as reasonably practicable, a written plan for the transfer of uniQure Manufacturing Know-How to Partner or its designated CMO that is not already Manufacturing the Licensed Product, in accordance with the timeline to be set forth therein (the date set forth in such plan for completion of such transfer and assumption of Manufacturing responsibility, the “Manufacturing Responsibility Cutover Date”), which plan the JSC may agree to update in accordance with Article 7 (Governance) from time to time (as updated from time to time, the “Manufacturing Responsibility Transfer Plan”).”
5. | Manufacturing Technology Transfer Plan. Notwithstanding Section 5.3.2 (Transfer of Manufacturing Responsibility), Section 5.3.3 (Manufacturing Responsibility Transfer Plan) or Section 5.4 (Transfer of Manufacturing Know-How) of the CLA, CSL and uniQure Bio agree as follows: |
i. | On September 6, 2022, CSL informed uniQure Bio by delivery of notice under Section 5.3.2 (Transfer of Manufacturing Responsibility) of the CLA, that CSL elected to Manufacture its requirements for Licensed Products through the Specified CMO [***] and the Parties developed a Manufacturing Responsibility Transfer Plan titled [***] for the transfer of uniQure Manufacturing Know-How (the “Manufacturing Transfer Plan”) to [***] in accordance with Section 5.4 (Transfer of Manufacturing Know-How) of the CLA. |
ii. | In connection with the Manufacturing Responsibility Transfer and CSL’s engagement of Genezen as its CMO for Manufacture of the Licensed Products, the Parties hereby terminate the Manufacturing Transfer Plan as of the Termination Execution Date, and any and all activities and obligations allocated to uniQure Bio in the Manufacturing Transfer Plan or in the CLA or CSA with respect to any transfer to [***] are considered completed. The Parties agree that neither Party shall owe any amounts to the other Party with respect to the Manufacturing Transfer Plan or the activities associated with the Manufacturing Transfer Plan. |
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iii. | The Parties expressly agree that uniQure Bio will not be required to conduct any further transfer or related activities in accordance with the terms of Section 5.4 (Transfer of Manufacturing Technology) of the CLA or otherwise to facilitate CSL’s, [***] or any other Third Party’s assumption of Manufacturing responsibility for the Licensed Products in accordance with the CLA. Notwithstanding the foregoing, uniQure Bio (or its applicable Affiliate) will permit Genezen, under a separate written agreement, to continue to retain possession of certain necessary uniQure Manufacturing Know-How in Genezen’s possession as of the Termination Effective Date solely as necessary to Manufacture the Product for CSL under the CSL-Genezen Agreement during the Term of the CLA. |
6. | Amendments to Article 7 (Governance). |
(a) | The following sentence is hereby added to become the final sentence of Section 7.6 (Project Management) of the CLA: |
“Upon the Manufacturing Responsibility Cutover Date, (a) the Project Team carrying out the Manufacturing Development Plan will be considered dissolved by the Parties and (b) the Project Team and Project Managers will no longer have any obligation to consult with respect to Manufacturing Development Plan activities.”
(b)
i. | A new Section 7.8 is added to the CLA, reading as follows: |
“7.8 Dissolution of JSC. On and from the Manufacturing Responsibility Cutover Date, the JSC will be dissolved, and notwithstanding the remainder of this Article 7 (Governance), no further meetings of the JSC will be required to take place, and CSL will have final decision-making authority over any matters that would have been decided by the JSC relating to the Exploitation of the Licensed Products in the Field and will not be required to consult with uniQure on any such matters, except for matters referred to in clauses (ii), (iii), and (iv) of Section 7.5.2(a) (No Change; Status Quo).”
ii. | CSL shall continue to report any safety matters with respect to the Licensed Product as required by the Safety Data Exchange Agreement in place between CSL and uniQure Bio. |
iii. | In connection with the foregoing new Section 7.8, CSL shall provide any information that it was required under the CLA to provide to the JSC directly to uniQure; provided that any information that was to be provided under the CLA by CSL in advance of, or at, a JSC meeting shall be provided by CSL to uniQure [***]; provided further that, notwithstanding any different timing required under this Agreement prior to the Manufacturing Responsibility Cutover Date, CSL shall provide any information relating to the compliance of Sublicensees or Subcontractors required under Section 2.2.5 to uniQure [***]. |
7. | Manufacturing Release. |
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(a) | Release. |
i. | Unless and until uniQure does not satisfy its obligation under Sections 2 and 4 of the Payment Agreement referred to in Section 10 of this Agreement to pay the Termination Amount, CSL, on behalf of itself and its Affiliates and its and their respective legal predecessors, successors, and assigns, and each of their respective current and former officers, directors, shareholders, members, employees, agents, attorneys, and representatives, in their capacities as such (collectively, “Releasors”) hereby releases, waives, and forever discharges uniQure Bio and its Affiliates and its and their respective legal predecessors, successors, and permitted assigns, and each of their respective current and former officers, directors, shareholders, members, employees, agents, attorneys, and representatives, in their capacities as such (collectively, “Releasees”) from any and all past, present, and future claims, demands, obligations, liabilities, and causes of action of any nature whatsoever, legal or equitable, whether known or unknown, foreseen or unforeseen, that any of the Releasors has or shall or may have under federal, state, or local constitutions, laws, ordinances, regulations, orders, or common law, but excluding those claims, demands, obligations, liabilities, or causes of action identified in clause (a)(ii) of this Section 7 of this Termination Agreement, relating to or arising out of, under or in connection with the Manufacturing or supply of (a) the batches of Licensed Products designated as the “Selected Batches” in Appendix B, (b) any Final CSA Batches where the Licensed Product has been [***] of the Manufacturing Process, or (c) any Final Conforming CSA Batch that has a [***] (such batches, the “Selected Batches,” and such claim, demands, obligations, liabilities, and causes of action, “Released Claims”). For clarity, Released Claims include claims relating to or arising out of, under or in connection with any out-of-specification results observed for any Selected Batches after initial inspection under the CSA. |
ii. | The Released Claims do not include, and nothing in this Termination Agreement or otherwise limits the liability, if any, of uniQure Bio or any other Releasee for, claims, demands, obligations, liabilities, or causes of action with respect to any portion of the Selected Batches that [***] prior to the Termination Execution Date. CSL represents, warrants and covenants that (a) it will not [***] and (b) that CSL shall only [***] as set forth in this Section 7(a)(ii). In the event that CSL wishes to use [***] for non-commercial research purposes, CSL shall submit a written proposal to uniQure detailing the use of such vials, and such use shall be subject to uniQure’s prior written approval. In connection with the foregoing, CSL has notified uniQure about its desire to [***]. [***] CSL agrees to provide, and will provide [***]. |
iii. | For clarity, nothing in this Termination Agreement represents or is intended to represent any concession or agreement of uniQure Bio or its Affiliates that it is or can be liable for [***]. |
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(b) | Covenant Not to Sue. CSL, on behalf of itself and its Releasors, agrees not to bring or commence any claim, complaint, demand, cause of action, request, or demand for arbitration, or other proceeding of any kind or nature against uniQure Bio and its Releasees in respect of any Released Claim. |
(c) | No Outstanding or Known Future Claims/Causes of Action; Unknown Claims. |
i. | CSL affirms that neither it nor any other Releasors has filed with any Governmental Authority any type of action, suit, or proceeding with respect to a Released Claim. |
ii. | With respect to the releases set forth in clause (a) of this Section 7 of this Termination Agreement, CSL, on behalf of itself and the other Releasors, (x) expressly understands, acknowledges, and assumes the risk that, with respect to the Released Claims, claims may exist as of the Termination Execution Date but currently be unknown, that claims may be suspected but currently be undetermined, that claims may be known but not have been asserted, or that losses resulting from any such claims may be currently unknown or overestimated or underestimated in amount or severity and (y) acknowledges that it may discover facts in addition to or different from those now known or believed to be true with respect to the settled Released Claims, but that it is CSL’s intention, on behalf of itself and the other Releasors, to hereby completely, fully, finally, and forever compromise, settle, release, discharge, and extinguish any and all claims known or unknown, suspected or unsuspected, which now exist, or heretofore existed, or may hereafter exist, and without regard to the subsequent discovery or existence of additional or different facts, in each case, with respect to the Released Claims. |
iii. | With respect to any and all Released Claims, CSL, and each of its Releasors, shall be deemed to have waived, relinquished, and released any and all provisions, rights, and benefits conferred by any law of the United States or any state or territory of the United States or other jurisdiction, or principle of common law or foreign law, which is similar, comparable, or equivalent to Cal. Civ. Code § 1542, which provides: A GENERAL RELEASE DOES NOT EXTEND TO CLAIMS THAT THE CREDITOR OR RELEASING PARTY DOES NOT KNOW OR SUSPECT TO EXIST IN HIS OR HER FAVOR AT THE TIME OF EXECUTING THE RELEASE AND THAT, IF KNOWN BY HIM OR HER, WOULD HAVE MATERIALLY AFFECTED HIS OR HER SETTLEMENT WITH THE DEBTOR OR RELEASED PARTY. |
8. | Termination and Amendment of Genezen Agreements. CSL acknowledges that in connection with the Manufacturing Responsibility Transfer, (a) uniQure Bio is amending its Development and Other Manufacturing Services Agreement with Genezen executed as of July 22, 2024 (the “Genezen DMSA”) to exclude the Product and services with respect to the Product and (b) uniQure US is terminating the Genezen CSA relating to the supply of Product by Genezen to uniQure US for further supply to CSL under the CSA ((a) and (b), the “Genezen-uniQure Terminations”). CSL, as a direct and intended third party beneficiary of the Genezen DMSA with respect to all obligations reasonably related to the Manufacture and Manufacturing |
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Process of the Products and Genezen CSA, hereby consents to the Genezen-uniQure Terminations.
9. | CSL-Genezen Agreement. Based on uniQure Bio’s review of the draft CSL-Genezen Agreement, uniQure agrees to Genezen’s designation under Section 5.3.2 (Transfer of Manufacturing Responsibility) as CSL’s CMO under the CLA as part of the Manufacturing Responsibility Transfer and uniQure consents to the grant of a sublicense by CSL to Genezen under uniQure Manufacturing Technology pursuant to Section 2.2.1 (Right to Sublicense) under the CLA. CSL represents and warrants that the draft agreement provided to uniQure for review prior to the Termination Execution Date is the CSL-Genezen Agreement in full, subject to permitted redactions and to the completion of immaterial matters (such as the insertion of an effective date) at execution, and that the terms of the CSL-Genezen Agreement comply with the requirements of Section 2.2 of the CLA. |
10. | Termination Amount. In partial consideration for CSL’s entry into this Termination Agreement, uniQure Bio, Genezen and CSL have entered into that certain Payment Agreement in the form attached hereto in Appendix C as of the Termination Execution Date, under which CSL will receive [***] (such consideration, the “Termination Amount”). |
11. | [***]. As of the Termination Execution Date, uniQure Bio represents and warrants that [***]. |
12. | Quality Agreements. CSL acknowledges and agrees that in connection with the termination of the Genezen CSA and the Manufacturing Responsibility Transfer, that certain quality agreement by and among CSL, CSL Behring GmbH, Genezen and uniQure US effective on or about October 27, 2025 (the “Three-Way QA”) is terminated as of the Termination Effective Date pursuant to Section 5 thereof but the (a) pharmacovigilance and adverse event reporting and management obligations, including under Section 4.5 and Annex 3 of the Three-Way QA Agreement and (b) obligations relating to complaints, recalls and retention of samples and documentation in accordance with and for the time period set forth in Section 6 of the Three-Way QA (and no other Sections or Articles of the Three-Way QA), in each case ((a) and (b)), will survive solely with respect to Products supplied pursuant to the CSA, including the Final CSA Batches. |
13. | Miscellaneous. |
(a) | The Appendices to this Termination Agreement are incorporated herein by reference and will be deemed a part of this Termination Agreement. |
(b) | Upon and after the Termination Execution Date, each reference in the CLA to “this Agreement”, “hereunder”, “hereof” or words of like import referring to the CLA shall mean and be a reference to the CLA as modified and amended hereby. |
(c) | Each Party hereby represents and warrants to the other Party, as of the Termination Execution Date, that it has full right, power and authority to enter into this Termination Agreement and that the signatories executing this Termination Agreement are authorized to sign on behalf of their respective Party. |
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(d) | The term of this Termination Agreement shall commence on the Termination Execution Date and continue until and unless terminated by mutual written agreement of the Parties. The Parties agree that the CLA, as specifically amended by this Termination Agreement, continues to remain in full force and effect. In the event of any conflict between the CLA and this Termination Agreement, this Termination Agreement shall control. |
(e) | Each Party will perform all further acts and things and execute and deliver such further documents as may be necessary or as another Party may reasonably be required to implement or give effect to this Termination Agreement. |
(f) | The interpretation and construction of this Termination Agreement shall be governed by the laws of the State of New York excluding any conflicts or choice of law rule or principle that might otherwise refer construction or interpretation of this Termination Agreement to the substantive law of another jurisdiction. |
(g) | The following Article and Sections of the CLA shall apply mutatis mutandis to this Termination Amendment as if set forth in full in this Termination Amendment, with the references to “Agreement” in each of these Sections being deemed to be references to this Termination Amendment: Article 14 (Dispute Resolution), Section 16.9 (Headings), Section 16.10 (Independent Contractors), Section 16.16 (Further Actions), Section 16.17 (Construction), and Section 16.18 (Language; Translations). |
(h) | This Termination Agreement may be executed in two (2) or more counterparts, each of which will be deemed an original and all of which will together be deemed to constitute one agreement. The Parties agree that the execution of this Termination Agreement by industry standard electronic signature software or by exchanging PDF signatures shall have the same legal force and effect as the exchange of original signatures, and that in any proceeding arising under or relating to this Termination Agreement, each Party hereby waives any right to raise any defense or waiver based upon execution of this Termination Agreement by means of such electronic signatures or maintenance of the executed agreement electronically. |
[Signature Page Follows]
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IN WITNESS WHEREOF, each of the Parties has caused this Termination Agreement to be executed as of the date hereof by a duly authorized corporate officer.
| CSL Behring LLC | ||
| | ||
| By | /s/ Michael Deem | |
| | Name: | Michael Deem |
| | Title: | Head of Global Operations |
| | ||
| | ||
| By | /s/ Michael O’Connor | |
| | Name: | Michael O’Connor |
| | Title: | AGC, US & Plasma, CSL Behring |
| | ||
| uniQure biopharma B.V. | ||
| | ||
| By | /s/ Christian Klemt | |
| | Name: | Christian Klemt |
| | Title: | CFO |
Signature Page to Termination Agreement (CSL and uniQure)
Appendix A
[***]
Appendix B
[***]
Appendix C
[***]
Appendix 1
[***]
Appendix 2
[***]
Exhibit 31.1
Certification of Chief Executive Officer
I, Matthew Kapusta, certify that:
1. I have reviewed this Quarterly Report on Form 10-Q of uniQure N.V.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation;
(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting;
5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
| By: | /s/ MATTHEW KAPUSTA |
|
|
|
|
| Matthew Kapusta |
|
| Chief Executive Officer |
| | (Principal Executive Officer) |
| M | May 5, 2026 |
Exhibit 31.2
Certification of Chief Financial Officer
I, Christian Klemt, certify that:
1. I have reviewed this Quarterly Report on Form 10-Q of uniQure N.V.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation;
(d) Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting;
5. The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
| By: | /s/ CHRISTIAN KLEMT |
|
|
|
|
| Christian Klemt |
|
| Chief Financial Officer |
| | (Principal Financial Officer) |
|
| May 5, 2026 |
Exhibit 32.1
CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350
AS ADOPTED PURSUANT TO SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002
In connection with this Quarterly Report of uniQure N.V. (the “Company”) on Form 10-Q for the period ended March 31, 2026, as filed with the Securities and Exchange Commission on the date hereof (the “Report”), the undersigned, Matthew Kapusta, Chief Executive Officer, and Christian Klemt, Chief Financial Officer of the Company, hereby certify, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that:
1 the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and
2 the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company.
| By: | /s/ MATTHEW KAPUSTA |
|
|
|
|
| Matthew Kapusta |
|
| Chief Executive Officer |
| | (Principal Executive Officer) |
|
| |
|
| May 5, 2026 |
| By: | /s/ CHRISTIAN KLEMT |
|
|
|
|
| Christian Klemt |
|
| Chief Financial Officer |
| | (Principal Financial Officer) |
|
| |
|
| May 5, 2026 |
A signed original of this written statement required by Section 906 has been provided to uniQure N.V. and will be retained by uniQure N.V. and furnished to the SEC or its staff upon request.