uniQure nine-month loss widens to $196M in 2026
In AMT-260’s low-dose cohort, three of six patients had meaningful reductions in disabling seizures during months four through six.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
uniQure N.V. reported a $195.8 million net loss for the nine months ended September 30, 2026, compared with $161.9 million in 2025. Total revenue was $14.6 million, versus $10.5 million; net cash used in operating activities was $114.6 million, versus $100.4 million.
At September 30, 2026, cash, restricted cash and investment securities totaled $770.0 million; management said these resources could fund projected operating expenses into 2030. Up to $100.0 million in additional Hercules loan availability becomes accessible upon BLA approval prior to September 30, 2027, subject to confirmatory-trial requirements remaining on track.
The FDA accepted the AMT-130 BLA under the Accelerated Approval pathway and granted Priority Review, with a target action date of May 1, 2027. The planned randomized confirmatory study is designed to enroll approximately 200 patients in a 2:1 ratio of AMT-130 to standard of care, with Total Function Capacity as the primary endpoint at 36 months; U.S. patient screening was planned by the end of 2026. Five high-dose participants (17%) experienced treatment-related serious adverse events involving central nervous system inflammation; all resolved.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Positive
- Moderate pointNine-month total revenue increased to $14.6 million from $10.5 million.
Negative
- Moderate pointNine-month net loss widened to $195.8 million from $161.9 million.
- Moderate pointNine-month operating cash use increased to $114.6 million from $100.4 million.
Filing Explained
At September 30, 2026, uniQure reported 70,044,739 ordinary shares outstanding, versus 62,336,717 at December 31, 2025; during the nine months it issued 5,686,813 shares in a follow-on offering and 526,314 on warrant exercise. These completed issuances expanded the share base, reducing existing holders’ percentage ownership absent offsetting changes.
Key Figures
Key Terms
Accelerated Approval regulatory
Priority Review regulatory
propensity score-matched external control technical
payable in kind financial
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What were QURE’s revenue and net loss for the first nine months of 2026?
What is the FDA’s target action date for QURE’s AMT-130 application?
What were the results from the first AMT-260 low-dose cohort?
AI-generated analysis. How Rhea-AI works. Not financial advice.
Table of Contents
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington D.C. 20549
FORM
(Mark One)
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 | |
| |
| For the quarterly period ended |
| |
| 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: |
(Exact name of Registrant as specified in its charter)
The (State or other jurisdiction of incorporation or organization) | Not applicable (I.R.S. Employer Identification No.) |
(Address of principal executive offices) (Zip Code)
+
(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 |
| | The Nasdaq Stock Market LLC ( |
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.
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).
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.
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
As of November 2, 2026, the registrant had
Table of Contents
TABLE OF CONTENTS
| | | Page | |
| | PART I – FINANCIAL INFORMATION | | |
| | | | |
Item 1 | | Financial Statements | | 2 |
| | | | |
Item 2 | | Management’s Discussion and Analysis of Financial Condition and Results of Operations | | 20 |
| | | | |
Item 3 | | Quantitative and Qualitative Disclosures About Market Risk | | 37 |
| | | | |
Item 4 | | Controls and Procedures | | 38 |
| | | | |
| | PART II – OTHER INFORMATION | | |
| | | | |
Item 1 | | Legal Proceedings | | 38 |
| | | | |
Item 1A | | Risk Factors | | 39 |
| | | | |
Item 2 | | Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities | | 41 |
| | | | |
Item 3 | | Defaults Upon Senior Securities | | 41 |
| | | | |
Item 4 | | Mine Safety Disclosures | | 41 |
| | | | |
Item 5 | | Other Information | | 41 |
| | | | |
Item 6 | | Exhibits | | 41 |
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 with respect to the Company’s Biologics License Application for ifezuntirgene inilparvovec (also known as AMT-130) to the United States Food and Drug Administration (the “FDA”) and Marketing Authorisation Application for ifezuntirgene inilparvovec 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; our plans to conduct a confirmatory study for ifezuntirgene inilparvovec; the timing of, and our ability to, obtain and maintain regulatory approvals for any of our product candidates; the potential benefits and clinical, therapeutic and commercial potential 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 ifezuntirgene inilparvovec; 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”), our Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, 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 publicly release any revisions to these forward-looking statements after completion of the filing of this Quarterly Report on Form 10-Q to reflect later events or circumstances or to reflect the occurrence of unanticipated events. All forward-looking statements attributable to us are expressly qualified in their entirety by these cautionary statements.
In addition, with respect to all our forward-looking statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
1
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Part I – FINANCIAL INFORMATION
Item 1.Financial Statements
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE SHEETS
| | | | | | |
| | September 30, | | December 31, | ||
| | 2026 | | 2025 | ||
| | (in thousands, except share and per share amounts) | ||||
Current assets | | | | | | |
Cash and cash equivalents | | $ | | | $ | |
Current investment securities | | | | | | |
Accounts receivable | | | | | | |
Prepaid expenses | | | | | | |
Other current assets and receivables | | | | | | |
Total current assets | | | | | | |
Non-current assets | | | | | | |
Property, plant and equipment, net | | | | | | |
Other investments | | | | | | |
Non-current investment securities | | | | | | — |
Operating lease right-of-use assets | | | | | | |
Intangible assets, net | | | | | | |
Goodwill | | | | | | |
Deferred tax assets, net | | | | | | |
Other non-current assets | | | | | | |
Total non-current assets | | | | | | |
Total assets | | $ | | | $ | |
Current liabilities | | | | | | |
Accounts payable | | $ | | | $ | |
Accrued expenses and other current liabilities | | | | | | |
Liability related to pre-funded warrants | | | — | | | |
Current portion of operating lease liabilities | | | | | | |
Total current liabilities | | | | | | |
Non-current liabilities | | | | | | |
Long-term debt | | | | | | |
Liability from royalty financing agreement | | | | | | |
Operating lease liabilities, net of current portion | | | | | | |
Contingent consideration | | | | | | |
Deferred tax liability, net | | | | | | |
Other non-current liabilities, net of current portion | | | | | | |
Total non-current liabilities | | | | | | |
Total liabilities | | | | | | |
Commitments and contingencies | | | | | | |
Shareholders' equity | | | | | | |
Ordinary shares, € | | | | | | |
Additional paid-in capital | | | | | | |
Accumulated other comprehensive loss | | | ( | | | ( |
Accumulated deficit | | | ( | | | ( |
Total shareholders' equity | | | | | | |
Total liabilities and shareholders' equity | | $ | | | $ | |
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 September 30, | | Nine months ended September 30, | ||||||||
| | 2026 | | 2025 | | 2026 | | 2025 | ||||
| | (in thousands, except share and per share amounts) | | (in thousands, except share and per share amounts) | ||||||||
License revenues | | $ | | | $ | | | $ | | | $ | |
Collaboration revenues | | | — | | | | | | — | | | |
Total revenues | | | | | | | | | | | | |
Operating expenses: | | | | | | | | | | | | |
Cost of license revenues | | | ( | | | ( | | | ( | | | ( |
Research and development expenses | | | ( | | | ( | | | ( | | | ( |
Selling, general and administrative expenses | | | ( | | | ( | | | ( | | | ( |
Total operating expenses | | | ( | | | ( | | | ( | | | ( |
Other income | | | | | | | | | | | | |
Other expense | | | ( | | | ( | | | ( | | | ( |
Loss from operations | | | ( | | | ( | | | ( | | | ( |
Interest income | | | | | | | | | | | | |
Interest expense | | | ( | | | ( | | | ( | | | ( |
Foreign currency (losses) / gains, net | | | ( | | | ( | | | ( | | | |
Other non-operating (losses), net | | | ( | | | ( | | | ( | | | ( |
Loss before income tax expense | | $ | ( | | $ | ( | | $ | ( | | $ | ( |
Income tax expense | | | ( | | | ( | | | ( | | | ( |
Net loss | | $ | ( | | $ | ( | | $ | ( | | $ | ( |
Other comprehensive loss: | | | | | | | | | | | | |
Foreign currency translation (losses) / gains, net | | | ( | | | | | | ( | | | ( |
Total comprehensive loss | | $ | ( | | $ | ( | | $ | ( | | $ | ( |
Loss per ordinary share - basic and diluted | | | | | | | | | | | | |
Basic and diluted net loss per ordinary share | | $ | ( | | $ | ( | | $ | ( | | $ | ( |
Weighted average shares - basic and diluted | | | | | | | | | | | | |
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 / (DEFICIT)
FOR THE THREE MONTHS ENDED SEPTEMBER 30, 2026 AND 2025
| | | | | | | | | | | | | | | | | |
| | | | | | | | | Accumulated | | | | | | |||
| | | | | | | Additional | | other | | | | | Total | |||
| | Ordinary shares | | paid-in | | comprehensive | | Accumulated | | shareholders’ | |||||||
| | No. of shares | | Amount | | capital | | gain / (loss) | | deficit | | equity / (deficit) | |||||
| | (in thousands, except share and per share amounts) | |||||||||||||||
Balance at June 30, 2025 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | ( |
Loss for the period | | — | | | — | | | — | | | — | | | ( | | | ( |
Other comprehensive gain, net | | — | | | — | | | — | | | | | | — | | | |
Follow-on public offering | | | | | | | | | | | — | | | — | | | |
Tax benefit related to historical share issuance | | — | | | — | | | | | | — | | | — | | | |
Exercise of share options | | | | | | | | | | | — | | | — | | | |
Restricted share units distributed during the period | | | | | | | | ( | | | — | | | — | | | — |
Share-based compensation expense | | — | | | — | | | | | | — | | | — | | | |
Balance at September 30, 2025 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | |
| | | | | | | | | | | | | | | | | |
Balance at June 30, 2026 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | |
Loss for the period | | — | | | — | | | — | | | — | | | ( | | | ( |
Other comprehensive loss, net | | — | | | — | | | — | | | ( | | | — | | | ( |
Exercises of share options | | | | | | | | | | | — | | | — | | | |
Exercise of pre-funded warrants | | | | | | | | | | | — | | | — | | | |
Restricted share units distributed during the period | | | | | | | | ( | | | — | | | — | | | — |
Issuance of ordinary shares relating to employee stock purchase plan | | | | | — | | | | | | — | | | — | | | |
Share-based compensation expense | | — | | | — | | | | | | — | | | — | | | |
Balance at September 30, 2026 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | |
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uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS’ EQUITY / (DEFICIT)
FOR THE NINE MONTHS ENDED SEPTEMBER 30, 2026 AND 2025
| | | | | | | | | | | | | | | | | |
| | | | | | | | | Accumulated | | | | | | |||
| | | | | | | Additional | | other | | | | | Total | |||
| | Ordinary shares | | paid-in | | comprehensive | | Accumulated | | shareholders’ | |||||||
| | No. of shares | | Amount | | capital | | gain / (loss) | | deficit | | equity / (deficit) | |||||
| | (in thousands, except share and per share amounts) | |||||||||||||||
Balance at December 31, 2024 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | ( |
Loss for the period | | — | | | — | | | — | | | — | | | ( | | | ( |
Other comprehensive loss, net | | — | | | — | | | — | | | ( | | | — | | | ( |
Follow-on public offerings | | | | | | | | | | | — | | | — | | | |
Tax benefit related to historical share issuance costs | | — | | | — | | | | | | — | | | — | | | |
Exercises of share options | | | | | | | | | | | — | | | — | | | |
Restricted and performance share units distributed during the period | | | | | | | | ( | | | — | | | — | | | — |
Share-based compensation expense | | — | | | — | | | | | | — | | | — | | | |
Balance at September 30, 2025 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | |
| | | | | | | | | | | | | | | | | |
Balance at December 31, 2025 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | |
Loss for the period | | — | | | — | | | — | | | — | | | ( | | | ( |
Other comprehensive loss, net | | — | | | — | | | — | | | ( | | | — | | | ( |
Follow-on public offering | | | | | | | | | | | — | | | — | | | |
Exercises of share options | | | | | | | | | | | — | | | — | | | |
Exercise of pre-funded warrants | | | | | | | | | | | — | | | — | | | |
Restricted share units distributed during the period | | | | | | | | ( | | | — | | | — | | | — |
Issuance of ordinary shares relating to employee stock purchase plan | | | | | | | | | | | — | | | — | | | |
Share-based compensation expense | | — | | | — | | | | | | — | | | — | | | |
Balance at September 30, 2026 | | | | $ | | | $ | | | $ | ( | | $ | ( | | $ | |
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
| | | | | | |
| | Nine months ended September 30, | ||||
| | 2026 | | 2025 | ||
| | (in thousands) | ||||
Cash flows used in operating activities | | | | | | |
Net loss | | $ | ( | | $ | ( |
Adjustments to reconcile net loss to net cash used in operating activities: | | | | | | |
Depreciation and amortization | | | | | | |
Amortization of discount on investment securities | | | ( | | | ( |
Share-based compensation expense | | | | | | |
Royalty financing agreement interest expense, net of interest paid | | | | | | |
Deferred tax expense | | | | | | |
Changes in fair value of contingent consideration | | | | | | |
Changes in fair value of liability related to pre-funded warrants | | | | | | |
Unrealized foreign exchange gains / (losses), net | | | | | | ( |
Other items, net | | | ( | | | ( |
Changes in operating assets and liabilities: | | | | | | |
Accounts receivable, prepaid expenses, and other current assets and receivables | | | | | | |
Accounts payable | | | | | | ( |
Income tax payable | | | | | | |
Accrued expenses, other liabilities, and operating leases | | | | | | |
Net cash used in operating activities | | | ( | | | ( |
Cash flows (used in) / generated from investing activities | | | | | | |
Proceeds on maturity of debt securities | | | | | | |
Investments in debt securities | | | ( | | | ( |
Purchases of property, plant, and equipment | | | ( | | | ( |
Net cash (used in) / generated from investing activities | | | ( | | | |
Cash flows from financing activities | | | | | | |
Proceeds from follow-on public offering of ordinary shares, net of issuance costs | | | | | | |
Proceeds from issuance of pre-funded warrants, net of issuance costs | | | — | | | |
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans | | | | | | |
Net cash generated from financing activities | | | | | | |
Currency effect on cash, cash equivalents and restricted cash | | | ( | | | |
Net increase in cash, cash equivalents and restricted cash | | | | | | |
Cash, cash equivalents and restricted cash at the beginning of period | | | | | | |
Cash, cash equivalents and restricted cash at the end of period | | $ | | | $ | |
Cash and cash equivalents | | $ | | | $ | |
Restricted cash related to leasehold and other deposits | | | | | | |
Total cash, cash equivalents and restricted cash | | $ | | | $ | |
Supplemental cash flow disclosures: | | | | | | |
Cash paid for interest | | $ | ( | | $ | ( |
Supplemental disclosure of non-cash investing and financing items: | | | | | | |
Issuance of ordinary shares upon exercise of pre-funded warrants | | $ | | | $ | — |
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 25, Amsterdam 1105 BP, the Netherlands and its telephone number is +31 20 240 6000.
The Company’s ordinary shares are listed on the Nasdaq Global Select Market and trade under the symbol “QURE”.
2Summary of significant accounting policies
2.1Basis of preparation
The Company prepared these unaudited consolidated financial statements in compliance with generally accepted accounting principles in the United States (“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 parentheses, 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 and nine months ended September 30, 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; the disclosure of contingent liabilities at the date of the financial statements; and the 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 nine months ended September 30, 2026.
2.5Recent accounting pronouncements
There have been no new accounting pronouncements or changes to accounting pronouncements during the nine months ended September 30, 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 | Investment securities |
The following tables summarize the Company’s investments in government debt securities as of September 30, 2026 and December 31, 2025:
| | | | | | | | | | | | |
| | September 30, 2026 | ||||||||||
| | Amortized cost | | Gross unrealized holding gains | | Gross unrealized holding losses | | Estimated fair value | ||||
| | (in thousands) | ||||||||||
Current investments securities: | | | | | | | | | | | | |
Government debt securities (held-to-maturity) | | $ | | | $ | — | | $ | ( | | $ | |
| | | | | | | | | | | | |
Non-current investments securities: | | | | | | | | | | | | |
Government debt securities (held-to-maturity) | | $ | | | $ | — | | $ | ( | | $ | |
Total | | $ | | | $ | — | | $ | ( | | $ | |
| | | | | | | | | | | | |
| | December 31, 2025 | ||||||||||
| | Amortized cost | | Gross unrealized holding gains | | Gross unrealized holding losses | | Estimated fair value | ||||
| | (in thousands) | ||||||||||
Current investments securities: | | | | | | | | | | | | |
Government debt securities (held-to-maturity) | | $ | | | $ | | | $ | — | | $ | |
Total | | $ | | | $ | | | $ | — | | $ | |
The Company invests in 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. Investment securities with contractual maturities greater than 12 months are presented within non-current investment securities and measured at amortized cost. Inputs to the fair value of the investments are considered Level 2 inputs.
As of September 30, 2026 and December 31, 2025,
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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 December 31, 2025 and September 30, 2026:
| | | | | | | | | | | | | | |
| | Quoted prices | | Significant | | Significant | | Total | | Classification in Unaudited Consolidated | ||||
| | | (in thousands) | | | |||||||||
Balance at December 31, 2025 | | | | | | | | | | | | | | |
Assets: | | | | | | | | | | | | | | |
Cash and cash equivalents | | $ | | | $ | — | | $ | — | | $ | | | Cash and cash equivalents |
Restricted cash | | | | | | — | | | — | | | | | Other non-current assets |
Total assets | | $ | | | $ | — | | $ | — | | $ | | | |
Liabilities: | | | | | | | | | | | | | | |
Liability related to pre-funded warrants | | | — | | | | | | — | | | | | Liability related to pre-funded warrants |
Contingent consideration | | | — | | | — | | | | | | | | Contingent consideration |
Consideration for post-acquisition services | | | — | | | — | | | | | | | | Other non-current liabilities |
Total liabilities | | $ | — | | $ | | | $ | | | $ | | | |
Balance at September 30, 2026 | | | | | | | | | | | | | | |
Assets: | | | | | | | | | | | | | | |
Cash and cash equivalents | | $ | | | $ | — | | $ | — | | $ | | | Cash and cash equivalents |
Restricted cash | | | | | | — | | | — | | | | | Other non-current assets |
Total assets | | $ | | | $ | — | | $ | — | | $ | | | |
Liabilities: | | | | | | | | | | | | | | |
Contingent consideration | | | — | | | — | | | | | | | | Contingent consideration |
Consideration for post-acquisition services | | | — | | | — | | | | | | | | Other non-current liabilities |
Total liabilities | | $ | — | | $ | — | | $ | | | $ | | | |
| a) | Contingent consideration |
The Company is required to pay up to EUR
The fair value of the contingent consideration as of September 30, 2026 was EUR
If, as of September 30, 2026, the Company had assumed a
The following table presents the changes in fair value of the contingent consideration (presented within non-current liabilities) between December 31, 2025 and September 30, 2026:
| | | |
| | Amount of | |
| | contingent | |
| | consideration | |
| | (in thousands) | |
Balance at December 31, 2025 | | $ | |
Unrealized change in fair value (presented within Research and development expenses) | | | |
Currency translation effects | | | ( |
Balance at September 30, 2026 | | $ | |
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The Company classified the total contingent consideration liability as non-current as of September 30, 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 connection with its September 2025 follow-on public offering, the Company issued pre-funded warrants to purchase
In August 2026, the holder exercised all outstanding Pre-Funded Warrants, and the Company issued ordinary shares from its reserve account. Immediately prior to exercise, the liability was remeasured to fair value, and its carrying amount was classified to shareholders' equity upon issuance of the ordinary shares (refer to Note 8, "Shareholders' equity").
| | | |
| | Liability related | |
| | to pre-funded | |
| | warrants | |
| | (in thousands) | |
Balance at December 31, 2025 | | $ | |
Realized increase in fair value (presented within Other non-operating (losses), net) | | | |
Currency translation effects | | | |
Settlement of pre-funded warrant liability through issuance of ordinary shares | | | ( |
Balance at September 30, 2026 | | $ | — |
5 | Accrued expenses and other current liabilities |
Accrued expenses and other current liabilities consisted of the following items as of September 30, 2026 and December 31, 2025:
| | | | | | |
| | September 30, | | December 31, | ||
| | 2026 | | 2025 | ||
| | (in thousands) | ||||
Accruals for goods received from and services provided by vendors, not yet billed | | $ | | | $ | |
Personnel-related accruals and liabilities | | | | | | |
Liability owed to the Purchaser pursuant to the Royalty Financing Agreement (refer to Note 7, "Royalty Financing Agreement") | | | | | | |
Current portion of firm purchase commitment liability and Genezen CSA termination payment | | | | | | |
Other current liabilities | | | | | | |
Total | | $ | | | $ | |
In April 2026, the Company entered into an agreement to terminate the commercial supply agreement (the “Genezen CSA”) with Genezen MA, Inc. (together with Genezen Holdings Inc., “Genezen”). Pursuant to the termination agreement, the Company’s obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA terminated.
6Long-term debt
In July 2026, the Company entered into an amendment to its venture debt loan facility with Hercules Capital, Inc. (as amended, the "2026 Amended Facility"). The amendment extended the period during which the Company may draw the $
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The total principal outstanding under the 2026 Amended Facility as of September 30, 2026 was $
The foreign currency loss on the 2026 Amended Facility in the three and nine months ended September 30, 2026 was $
During the nine months ended September 30, 2026, the covenants under the 2026 Amended Facility were amended to include a new United Kingdom entity. Other than this amendment and as described above, there were no material changes to the terms, available borrowing tranches, covenants, or collateral arrangements of the 2026 Amended Facility as described in Note 11, "Long-term debt," to the consolidated financial statements included in the Annual Report. As of September 30, 2026, the Company was in compliance with all applicable covenants under the 2026 Amended Facility.
7Royalty Financing Agreement
During the nine months ended September 30, 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 Annual Report.
The following table presents the changes in the liability from the Royalty Financing Agreement for the period from December 31, 2025 to September 30, 2026:
| | | |
| | Amount of liability | |
| | (in thousands) | |
Balance as of December 31, 2025 (includes $ | | $ | |
Royalty payments to the Purchaser | | | ( |
Interest expense for the period | | | |
Balance as of September 30, 2026 (includes $ | | $ | |
The Company records the debt at amortized cost using the effective interest method based on projected cash flows. As of September 30, 2026, the effective interest rate is expected to be within a range of
8Shareholders’ equity
In June 2026, the Company received net proceeds of $
In June 2026, the Company's shareholders approved an amendment to the Articles of Association increasing the Company's authorized share capital from
In August 2026, all outstanding Pre-Funded Warrants were exercised by the holder, and the Company issued
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9Share-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 September 30, 2026, a total of
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
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 September 30, | | Nine months ended September 30, | ||||||||
| | 2026 | | 2025 | | 2026 | | 2025 | ||||
| | (in thousands) | | (in thousands) | ||||||||
Research and development expenses | | | | | | | | | | | | |
Selling, general and administrative expenses | | | | | | | | | | | | |
Total | | $ | | | $ | | | $ | | | $ | |
Share-based compensation expense recognized by award type for the 2014 Plans as well as the ESPP was as follows:
| | | | | | | | | | | | |
| | Three months ended September 30, | | Nine months ended September 30, | ||||||||
| | 2026 | | 2025 | | 2026 | | 2025 | ||||
| | (in thousands) | | (in thousands) | ||||||||
Award type/ESPP | | | | | | | | | | | | |
Share options | | $ | | | $ | | | $ | | | $ | |
Restricted share units | | | | | | | | | | | | |
Performance share units | | | | | | ( | | | | | | |
ESPP | | | | | | | | | | | | |
Total | | $ | | | $ | | | $ | | | $ | |
As of September 30, 2026, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans was:
| | | | | |
| | Unrecognized | | Weighted average | |
| | share-based | | remaining | |
| | compensation | | period for | |
| | expense | | recognition | |
| | (in thousands) | | (in years) | |
Award type | | | | | |
Share options | | $ | | | |
Restricted share units | | | | | |
Performance share units | | | | | |
Total | | $ | | | |
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.
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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
The following table summarizes option activity under the 2014 Plans for the nine months ended September 30, 2026:
| | | | | |
| | Options | |||
| | Number of ordinary shares | | | Weighted |
| | | | | |
Outstanding at December 31, 2025 | | | | | |
Granted | | | | $ | |
Forfeited | | ( | | $ | |
Expired | | ( | | $ | |
Exercised | | ( | | $ | |
Outstanding at September 30, 2026 | | | | $ | |
Thereof, fully vested, and exercisable on September 30, 2026 | | | | $ | |
Thereof, outstanding and expected to vest after September 30, 2026 | | | | $ | |
Outstanding and expected to vest after December 31, 2025 | | | | $ | |
| | | | | |
Total weighted average grant date fair value of options issued during the period (in $ millions) | | | | $ | |
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 September 30, | | Nine months ended September 30, | ||||
| | 2026 | | 2025 | | 2026 | | 2025 |
Assumptions | | | | | | | | |
Expected volatility | | | | | ||||
Expected terms | | | | | ||||
Risk-free interest rate | | | | | ||||
Expected dividend yield | | | | | ||||
RSUs
The following table summarizes the RSU activity for the nine months ended September 30, 2026:
| | | | | |
| | RSUs | |||
| | | | Weighted average | |
| | Number of | | grant-date fair | |
| | ordinary shares | | value | |
Non-vested at December 31, 2025 | | | | $ | |
Granted | | | | $ | |
Vested | | ( | | $ | |
Forfeited | | ( | | $ | |
Non-vested at September 30, 2026 | | | | $ | |
| | | | | |
Total weighted average grant date fair value of RSUs granted during the period (in $ millions) | | | | $ | |
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
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PSUs
The Company has granted PSUs to certain employees of the Company that represent shares potentially issuable in the future. The 2026 PSUs will be earned upon Company management's assessment of the level of achievement of agreed upon performance targets, subject to the grantee's continued employment.
The fair value of PSUs is determined on the grant date by reference to the market price of the Company's ordinary shares, and the Company recognizes the related compensation cost to the extent it considers achievement of the milestones to be probable.
As of September 30, 2026, certain 2026 PSUs were assessed as probable of achieving the requisite performance condition. During the nine months ended September 30, 2026, the performance target applicable to the 2024 PSUs was not met within the contractually defined performance period and the outstanding 2024 PSUs were forfeited.
The following table summarizes the PSU activity for the nine months ended September 30, 2026:
| | | | | |
| | PSUs | |||
| | | | Weighted average | |
| | Number of | | grant-date fair | |
| | ordinary shares | | value | |
Non-vested at December 31, 2025 | | | | $ | |
Granted | | | | $ | |
Vested | | — | | $ | — |
Forfeited | | ( | | $ | |
Non-vested at September 30, 2026 | | | | $ | |
ESPP
During the nine months ended September 30, 2026,
10 | 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
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.
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| | | | | | | | | | | | |
| | Three months ended September 30, | | Nine months ended September 30, | ||||||||
| | 2026 | | 2025 | | 2026 | | 2025 | ||||
| | (in thousands) | | (in thousands) | ||||||||
Revenue | | $ | | | $ | | | $ | | | $ | |
Less: | |
| | | | | |
| | | | |
Employee related expenses | |
| ( | |
| ( | |
| ( | |
| ( |
Laboratory and development expenses | | | ( | | | ( | | | ( | | | ( |
Professional fees | | | ( | | | ( | | | ( | | | ( |
Information technology system costs(2) | | | ( | | | ( | | | ( | | | ( |
Facility expenses | | | ( | | | ( | | | ( | | | ( |
Other segment items(1) (2) | | | ( | | | ( | | | ( | | | ( |
Segment operating loss | | | ( | | | ( | | | ( | | | ( |
Reconciliation | | | | | | | | | | | | |
Depreciation and amortization expense | | | ( | | | ( | | | ( | | | ( |
Share-based compensation expense | | | ( | | | ( | | | ( | | | ( |
Fair value (loss) / gain - contingent consideration | | | ( | | | ( | | | ( | | | ( |
Fair value loss - Liability related to pre-funded warrants | | | ( | | | ( | | | ( | | | ( |
Foreign currency (losses) / gains, net | | | ( | | | ( | | | ( | | | |
Interest income | | | | | | | | | | | | |
Interest expense - Royalty Financing Agreement | | | ( | |
| ( | | | ( | |
| ( |
Interest expense - Hercules loan facility | | | ( | | | ( | | | ( | | | ( |
Other reconciling items | | | ( | | | ( | | | ( | | | ( |
Consolidated loss before income tax expense | | $ | ( | | $ | ( | | $ | ( | | $ | ( |
| (1) |
| (2) | The comparative amounts for the three and nine months ended September 30, 2025 have been adjusted to reflect the level of detail at which the CODM reviews segment operating expenses in 2026, with information technology system costs presented as a separate line item and consumables included within other segment items for all periods presented. |
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11 | Other investments and concentration of credit risk |
The following table summarizes the Company’s Other investments as of September 30, 2026 and December 31, 2025:
| | | | | | |
| | September 30, | | December 31, | ||
| | 2026 | | 2025 | ||
| | (in thousands) | ||||
Carrying amount of equity investments | | $ | | | $ | |
Genezen convertible note receivable | | | | | | |
Total | | $ | | | $ | |
Carrying amount of equity investments
The Company holds investments in non-publicly traded equity securities without readily determinable fair values. These investments are accounted for at cost, less impairment and adjusted to fair value if there are observable price changes in orderly transactions for identical or similar investments using the measurement alternative in accordance with ASC 321, Investments – Equity Securities.
The carrying value of these investments as of September 30, 2026 and December 31, 2025 was $
Genezen
The Company held a convertible promissory note issued in July 2024 by Genezen at a nominal amount of $
On August 11, 2026, in connection with the refinancing of Genezen’s senior credit facilities, the Original Note was amended and restated (the "Amended Note"). The Amended Note has a contractually agreed principal amount of $
The amendment was accounted for as a continuation of the Original Note under ASC 310 and did not result in the recognition of a new loan. As a result, the Company's investment in the Amended Note was recorded based on the remaining net investment in the Original Note immediately prior to the amendment. The Amended Note had an amortized cost basis of $
CSL Behring
As of September 30, 2026, and December 31, 2025, accounts receivable from CSL Behring LLC ("CSL Behring") totaled $
12 | Income taxes |
The Company recorded a deferred tax expense of $
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months ended September 30, 2025). In addition, during the three and nine months ended September 30, 2025, the Company recorded $
The effective income tax rate of
13Basic 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 and nine months ended September 30, 2026, 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 and nine months ended September 30, 2026. 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 September 30, 2026 and September 30, 2025, respectively.
The potentially dilutive ordinary shares are summarized below:
| | | | | | | | |
| Three months ended September 30, | | Nine months ended September 30, | |||||
| | 2026 | | 2025 | | 2026 | | 2025 |
Anti-dilutive ordinary share equivalents | | | | | | | | |
Share options under 2014 Plans | $ | | | | $ | | | |
Non-vested RSUs and PSUs | | | | | | | | |
Pre-Funded Warrants | | — | | | | — | | |
ESPP | | | | | | | | |
Total anti-dilutive ordinary share equivalents | | | | | | | | |
The Pre-Funded Warrants were fully exercised in August 2026 (refer to Note 4, "Fair value measurement").
14Commitments 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 certain 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 are not fixed or determinable.
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 (collectively, “Defendants”) in the United States District Court for the Southern District of New York. The complaint purported to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), 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 (the “putative Class Period”). The plaintiff sought to recover damages allegedly caused by purported false and misleading statements and omissions with respect to the Company’s Phase I/II study of ifezuntirgene inilparvovec and the timing of the potential Biologics License Application filing for ifezuntirgene inilparvovec.
On April 13, 2026, the court appointed the Oklahoma Firefighters Pension and Retirement System as lead plaintiff (the “Lead Plaintiff”).
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On July 15, 2026, the Lead Plaintiff filed an amended complaint. Like the earlier-filed complaint, the amended complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5, on behalf of a putative class of investors who purchased or otherwise acquired the Company’s ordinary shares during the putative Class Period. Lead Plaintiff seeks unspecified damages allegedly caused by purported false and misleading statements and omissions with respect to the import and viability of data from the Company’s Phase I/II study of ifezuntirgene inilparvovec and available pathways to regulatory approval. Defendants’ motion to dismiss the amended complaint is currently due November 4, 2026. Management 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. The Company has not recorded any liability for any portion of this matter because the Company believes that liability is not probable and reasonably estimable at this time.
15Subsequent events
None.
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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 (ifezuntirgene inilparvovec)
Ifezuntirgene inilparvovec (also known as 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 ifezuntirgene inilparvovec 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 ifezuntirgene inilparvovec in a fourth cohort to evaluate the safety and efficacy of ifezuntirgene inilparvovec in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.
Regulatory Updates
In September 2026, we announced the submission of a Biologics License Application (“BLA”) to the U.S. Food and Drug Administration (the “FDA”) for the accelerated approval of ifezuntirgene inilparvovec as well as the submission of a marketing authorization application (“MAA”) for ifezuntirgene inilparvovec to the United Kingdom’s (“UK”) Medicines and Healthcare products Regulatory Agency (“MHRA”). Both submissions were supported by the three-year data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database.
In the third quarter of 2026, the MHRA validated the MAA for ifezuntirgene inilparvovec.
On November 2 2026, we announced the FDA has accepted for review the BLA for ifezuntirgene inilparvovec, submitted under the Accelerated Approval pathway. The application has been granted Priority Review with a Prescription Drug User Fee Act (“PDUFA”) target action date of May 1, 2027. In its acceptance letter, the FDA stated that they are tentatively planning to discuss this application at an Advisory Committee meeting. We will be notified once the date and timelines have been finalized.
We and FDA have aligned on key elements of a confirmatory study design. The study is designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Function Capacity (“TFC”) as the primary endpoint at 36 months. We plan to initiate patient screening in the United States by the end of 2026.
Data from Phase I/II Clinical Studies
In September 2026, we announced additional data from the ongoing Phase I/II studies of ifezuntirgene inilparvovec at two timepoints – 36 months and 48 months. We analyzed clinical outcomes for 29 patients treated with
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ifezuntirgene inilparvovec (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 48 months of follow-up and were evaluated at that time point. In addition, the 36-month analysis now includes 15 high-dose and 12 low-dose patients, with three additional high-dose patients having reached that timepoint since the September 2025 analysis.
Outcomes for the 36-month and 48-month analyses were compared to propensity score-matched external controls (n=1,337 for high dose) from an updated ENROLL-HD natural history dataset with a September 2025 cutoff.
Topline clinical data at 36-month and 48-month efficacy results for patients receiving high-dose ifezuntirgene inilparvovec were as follows (data cutoff as of June 30, 2026):
High-Dose Results at 36 Months (n=15)
| ● | composite Unified Huntington’s Disease Rating Scale (“cUHDRS”) showed 80% slowing of disease progression compared to the updated external control (nominal p=0.005). Treated patients had a mean change in cUHDRS from baseline of -0.28 compared to a change of -1.39 for the external control, a favorable treatment difference of 1.12 compared to baseline. |
| ● | TFC showed 67% slowing of disease progression compared to the updated external control (nominal p=0.011). Treated patients had a mean change in TFC from baseline of -0.27 compared to a change of -0.82 for patients in the propensity score-matched external control, a favorable treatment difference of 0.55 compared to baseline. |
| ● | Mean cerebrospinal neurofilament light protein (“CSF NfL”) was 6% below baseline (n=14). |
High-Dose Results at 48 Months (n=12)
| ● | cUHDRS showed a 44% slowing of disease progression compared to the updated external control and did not reach statistical significance (p=0.144). Treated patients had a mean change in cUHDRS from baseline of -0.90 compared to a change of -1.61 for the external control, a favorable treatment difference of 0.71 compared to baseline. |
| ● | TFC showed 61% slowing of disease progression compared to the updated external control (nominal p=0.008). Treated patients had a mean change in TFC from baseline of -0.37 compared to a change of -0.94 for the external control, a favorable treatment difference of 0.57 compared to baseline. |
| ● | In a post-hoc sensitivity analysis using the prior ENROLL-HD external control, the 48-month analysis showed 53.5% slowing based on cUHDRS (nominal p=0.041) and 68.3% based on TFC (nominal p=0.001). |
| ● | Mean CSF NfL was 4% above baseline (n=11). |
Dose Comparison at 48 Months (high dose n=12, low-dose n=12)
| ● | The observed differences between the high and low-doses are consistent with a dose-dependent treatment effect. At 48 months, mean change from baseline in cUHDRS was –0.91 in high-dose patients compared with −1.94 in low-dose patients, a difference of 1.03 in favor of the high dose. The mean change from baseline in TFC was −0.30 in high-dose patients compared with −0.70 in low-dose patients, a difference of 0.40 in favor of the high dose. |
As is common with longitudinal natural history studies, missingness of data increased with duration of follow-up, reaching 53% in the updated ENROLL-HD matched controls at 48 months. Our analysis of the updated control showed that patients discontinuing follow-up were progressing materially faster than those remaining in the control group. We believe these factors likely understated disease progression in the control and the resulting treatment effect of ifezuntirgene inilparvovec at 48 months.
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Ifezuntirgene inilparvovec continues to be 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, and all have resolved. As previously disclosed, five high dose participants (17%) experienced a treatment-related serious adverse event (“SAE”) related to central nervous system inflammation, all of which fully resolved.
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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 is a multicenter, open-label trial with two dosing cohorts of at least six patients each to assess safety, tolerability, and initial efficacy of AMT-260 in patients with refractory MTLE.
In June 2026, we announced preliminary data on the first cohort in GenTLE. As of the May 29, 2026 data cutoff date, three of six patients in the first, low-dose cohort (1x1012 gc/mL) achieved meaningful reductions in disabling seizures during months four through six of follow-up, ranging from a 79% to 100% decline from baseline. The remaining three patients in the low-dose cohort experienced variable changes in disabling seizures during months four through six of follow-up, ranging from a 33% decrease to a 36% increase compared to baseline. As of June 19, 2026, there have been no SAEs related to AMT-260 or the surgical procedure reported. All reported adverse events in the low-dose cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2). No immunosuppression was required.
In the third quarter of 2026, the Company completed enrollment in the second dose cohort of seven patients in the ongoing Phase I/IIa study.
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 June 2026, we presented updated preliminary data from the Phase I/II study of AMT-191 for Fabry disease. The data, based on a March 15, 2026 data cutoff date, included patient follow-up ranging from three months to more than 18 months and consisted of the following:
| ● | Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 1.0- to 16.2-fold above mean normal range (1.38-8.66 nmol; mean normal of 3.57 nmol) at the lowest dose, 14.5- to 229.6-fold at the mid dose, and 58.7- to 143.6-fold at the highest dose. |
| ● | All 11 dosed patients were withdrawn from enzyme replacement therapy (“ERT”). |
| ● | Plasma lyso-Gb3 levels were stable post-dose across all dose cohorts, regardless of ERT status through the cutoff date. |
AMT-191 continued to show a manageable safety profile. No SAEs related to AMT-191 were observed at the 4x1013 gc/kg (“mid-dose”) and 2x1013 gc/kg (“low-dose”) doses. No additional SAEs were observed at the 6x1013 gc/kg (“high-dose”) dose beyond those previously reported in September 2025 in two patients.
In August 2026, enrollment resumed in the mid- and high-dose cohorts of the ongoing Phase I/II study. Per protocol, dosing had previously been paused after observing asymptomatic liver enzyme elevations in two mid-dose patients, which resolved following a course of immunosuppression.
Business Development
Hercules Loan Amendment
In July 2026, we entered into an amendment to the $175.0 million senior secured term loan facility (the “2026 Amended Facility”) with Hercules Capital, Inc. (“Hercules”). The 2026 Amended Facility, among other things, extends the period we can draw the $100.0 million term loan tranche from June 2027 to September 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility remain unchanged.
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Financial Overview
Key components of our results of operations include the following:
| | | | | | | | | | | | | |
| | Three months ended September 30, | | Nine months ended September 30, | | ||||||||
| | 2026 | | 2025 | | 2026 | | 2025 | | ||||
| | (in thousands) | | (in thousands) | | ||||||||
Total revenues | | $ | 5,196 | | $ | 3,701 | | $ | 14,599 | | $ | 10,530 | |
Cost of license revenues | | | (236) | | | (398) | | | (805) | | | (1,251) | |
Research and development expenses | | | (35,517) | | | (34,366) | | | (98,657) | | | (105,889) | |
Selling, general and administrative expenses | | | (23,918) | | | (19,438) | | | (61,353) | | | (43,846) | |
Net loss | | | (61,217) | | | (80,529) | | | (195,811) | | | (161,885) | |
As of September 30, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $768.4 million and $622.5 million, respectively. We had a net loss of $61.2 million and $195.8 million in the three and nine months ended September 30, 2026, compared to a net loss of $80.5 million and $161.9 million for the same periods in 2025. As of September 30, 2026 and December 31, 2025, we had accumulated deficits of $1,524.7 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 nine months ended September 30, 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. |
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:
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| ● | 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.
In 2024, as part of the divestment of our commercial manufacturing operations to Genezen Holdings Inc. and its subsidiary Genezen MA, Inc. (together “Genezen”), we entered into various service agreements, including a commercial supply agreement (the “Genezen CSA”). In April 2026, we entered into an agreement to terminate the Genezen CSA. Pursuant to the termination agreement, our obligation to supply HEMGENIX® and any minimum purchase commitments under the Genezen CSA terminated in October 2026.
Our other expenses up to September 30, 2026, consisted of costs incurred under the Genezen CSA. These include costs related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring LLC (“CSL Behring”), amortization of the intangible asset recorded with respect to the favorable supply terms under the Genezen CSA, and release of liabilities related to expected net losses associated with the remaining minimum purchase commitments under the Genezen CSA.
Through the termination of the Genezen CSA in October 2026, we treated costs related to the purchase of HEMGENIX® from Genezen and revenue from the sales of HEMGENIX® to CSL Behring as an agent, and presented them on a net basis, together with income related to the release of liabilities associated with expected net losses, in Other expense within the Company's Consolidated Statements of Operations and Comprehensive Loss.
Additionally, other expenses also consist of expenses we incur in relation to our subleasing income.
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Results of Operations
Comparison of the three months ended September 30, 2026 and 2025
The following table presents a comparison of our results of operations for the three months ended September 30, 2026 and 2025:
| | | | | | | | | |
| | Three months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
License revenues | | $ | 5,196 | | $ | 3,582 | | $ | 1,614 |
Collaboration revenues | | | — | | | 119 | | | (119) |
Total revenues | | | 5,196 | | | 3,701 | | | 1,495 |
Operating expenses: | | | | | | | | | |
Cost of license revenues | | | (236) | | | (398) | | | 162 |
Research and development expenses | | | (35,517) | | | (34,366) | | | (1,151) |
Selling, general and administrative expenses | | | (23,918) | | | (19,438) | | | (4,480) |
Total operating expenses | | | (59,671) | | | (54,202) | | | (5,469) |
Other income | | | 1,690 | | | 1,510 | | | 180 |
Other expense | | | (195) | | | (2,044) | | | 1,849 |
Loss from operations | | | (52,980) | | | (51,035) | | | (1,945) |
Non-operating (expense) / income, net | | | (7,561) | | | (20,868) | | | 13,307 |
Net loss before income tax expense | | $ | (60,541) | | $ | (71,903) | | $ | 11,362 |
Income tax expense | | | (676) | | | (8,626) | | | 7,950 |
Net loss | | $ | (61,217) | | $ | (80,529) | | $ | 19,312 |
License revenues
We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the three months ended September 30, 2026 and 2025, we recognized $5.2 million and $3.6 million of license revenues, respectively.
R&D expense
R&D expenses for the three months ended September 30, 2026 were $35.5 million, compared to $34.4 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.
| | | | | | | | | |
| | Three months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Huntington's disease (ifezuntirgene inilparvovec) | | $ | 7,693 | | $ | 11,801 | | $ | (4,108) |
Temporal lobe epilepsy (AMT-260) | | | 3,723 | | | 2,245 | | | 1,478 |
Fabry disease (AMT-191) | | | 1,372 | | | 1,709 | | | (337) |
Amyotrophic lateral sclerosis (AMT-162) | | | 572 | | | 1,638 | | | (1,066) |
Programs in preclinical development and platform related expenses | | | 3,152 | | | 1,419 | | | 1,733 |
Total direct research and development expenses | | $ | 16,512 | | $ | 18,812 | | $ | (2,300) |
| | | | | | | | | |
Employee and contractor-related expenses | | | 10,743 | | | 8,566 | | | 2,177 |
Facility expenses | | | 3,253 | | | 3,716 | | | (463) |
Share-based compensation expense | | | 2,081 | | | 695 | | | 1,386 |
Fair value changes related to contingent consideration | | | 698 | | | 1,822 | | | (1,124) |
Information technology costs | | | 590 | | | 27 | | | 563 |
Other expenses | | | 1,640 | | | 728 | | | 912 |
Total other research and development expenses | | $ | 19,005 | | $ | 15,554 | | $ | 3,451 |
| | | | | | | | | |
Total research and development expenses | | $ | 35,517 | | $ | 34,366 | | $ | 1,151 |
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Direct research and development expenses
Huntington’s disease (ifezuntirgene inilparvovec)
In the three months ended September 30, 2026 and 2025, we incurred costs related to the development of ifezuntirgene inilparvovec of $7.7 million and $11.8 million respectively. The decrease of $4.1 million was primarily related to lower manufacturing process validation costs, which were $0.7 million in the current year period, compared to $5.3 million in the prior year period.
Temporal lobe epilepsy (AMT-260)
In the three months ended September 30, 2026 and September 30, 2025, we incurred costs related to the development of AMT-260 of $3.7 million and $2.2 million, respectively. The three-month period ended September 30, 2026 included $3.2 million of clinical expenses and $0.5 million of CMC expenses, respectively, compared to $2.1 million and $0.1 million, respectively, in the three-month period ended September 30, 2025.
Fabry disease (AMT-191)
In the three months ended September 30, 2026 and September 30, 2025, we incurred costs related to the development of AMT-191 of $1.4 million and $1.7 million, respectively. These costs were primarily related to our Phase I/II trial.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the three months ended September 30, 2026 and September 30, 2025, we incurred costs related to the development of AMT-162 of $0.6 million and $1.6 million, respectively. These costs were primarily related to our Phase I/IIa clinical trial.
Preclinical programs & platform development
In the three months ended September 30, 2026 and September 30, 2025, we incurred costs related to research and technology projects of $3.2 million and $1.4 million, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Other research & development expenses
| ● | We incurred $10.7 million in personnel and contractor-related expenses in the three months ended September 30, 2026, compared to $8.6 million for the same period in 2025. The $2.2 million increase was primarily due to higher personnel-related expenses in the current year period; |
| ● | We incurred $3.3 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended September 30, 2026, compared to $3.7 million in the same period in 2025; |
| ● | We incurred $2.1 million in share-based compensation expenses in the three months ended September 30, 2026, compared to $0.7 million for the same period in 2025. The increase of $1.4 million was primarily driven by new awards granted during the current year period; |
| ● | We incurred a $0.7 million loss related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the three months ended September 30, 2026, compared to a $1.8 million loss for the same period in 2025. The $1.1 million favorable change was primarily attributable to changes in discount rates applied to the remaining milestone payments in the current period; |
| ● | We incurred $0.6 million in information technology costs in the three months ended September 30, 2026, compared to less than $0.1 million in the same period in 2025; and |
| ● | We incurred $1.6 million of other expenses for the three months ended September 30, 2026, compared to $0.7 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 September 30, 2026 were $23.9 million, compared to $19.4 million for the same period in 2025.
| | | | | | | | | |
| | Three months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Employee and contractor-related expenses | | $ | 9,499 | | $ | 7,055 | | $ | 2,444 |
Professional fees | | | 7,074 | | | 6,362 | | | 712 |
Share-based compensation expense | | | 3,365 | | | 2,827 | | | 538 |
Information technology costs | | | 960 | | | 310 | | | 650 |
Intellectual property fees | | | 789 | | | 582 | | | 207 |
Depreciation and facility costs | | | 528 | | | 466 | | | 62 |
Other expenses | | | 1,703 | | | 1,836 | | | (133) |
Total selling, general and administrative expenses | | $ | 23,918 | | $ | 19,438 | | $ | 4,480 |
| ● | We incurred $9.5 million in personnel and contractor-related expenses in the three months ended September 30, 2026, compared to $7.1 million in the same period in 2025. The $2.4 million increase was primarily driven by a higher headcount following employee recruitment to support the potential commercial launches of ifezuntirgene inilparvovec; |
| ● | We incurred $7.1 million in professional fees in the three months ended September 30, 2026, compared to $6.4 million in the same period in 2025. The $0.7 million increase was primarily a result of higher costs in connection with the potential commercial launches of ifezuntirgene inilparvovec; |
| ● | We incurred $3.4 million in share-based compensation expenses in the three months ended September 30, 2026, compared to $2.8 million in the same period in 2025. The $0.5 million increase was primarily driven by new awards granted during the current year period; |
| ● | We incurred $1.0 million in information technology costs in the three months ended September 30, 2026, compared to $0.3 million in the same period in 2025. The increase was primarily related to expenses incurred to support the preparation of a potential commercialization of ifezuntirgene inilparvovec; |
| ● | We incurred $0.8 million in intellectual property fees in the three months ended September 30, 2026, compared to $0.6 million in the same period in 2025; |
| ● | We incurred $0.5 million in depreciation and facility costs in the three months ended September 30, 2026, compared to $0.5 million in the same period in 2025; and |
| ● | We incurred $1.7 million in other expenses in the three months ended September 30, 2026, compared to $1.8 million in the same period in 2025. |
Other income and expense
| | | | | | | | | |
| | Three months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Research and development grants from Dutch authorities | | $ | 1,073 | | $ | 1,012 | | $ | 61 |
Sublease income, net | | | 309 | | | 31 | | | 278 |
Supply of HEMGENIX® to CSL Behring | | | 71 | | | (1,586) | | | 1,657 |
Other income, net | | | 42 | | | 9 | | | 33 |
Total other items, net | | $ | 1,495 | | $ | (534) | | $ | 2,029 |
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Other non-operating items, net
Our other non-operating items, net, for the three months ended September 30, 2026 and September 30, 2025 were as follows:
| | | | | | | | | |
| | Three months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Interest income | | $ | 8,114 | | $ | 3,503 | | $ | 4,611 |
Interest expense - Royalty Financing Agreement | | | (13,164) | | | (13,806) | | | 642 |
Interest expense - Hercules loan facility | | | (1,406) | | | (1,794) | | | 388 |
Interest expense - other | | | (6) | | | (1,121) | | | 1,115 |
Foreign currency (losses) / gains, net | | | (217) | | | (428) | | | 211 |
Changes in fair value of liability related to pre-funded warrants | | | (882) | | | (5,722) | | | 4,840 |
Issuance costs - Pre-funded warrants | | | — | | | (1,500) | | | 1,500 |
Total non-operating items, net | | $ | (7,561) | | $ | (20,868) | | $ | 13,307 |
We recognize interest income associated with our cash and cash equivalents, investment securities and convertible note. We recognized $8.1 million in interest income in the three months ended September 30, 2026, compared to $3.5 million in the same period in 2025. The $4.6 million increase was primarily due to higher interest rates as well as higher average balances of our investment securities held during the current year period, compared to the prior year period.
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 of $0.2 million during the three months ended September 30, 2026, compared to a net loss of $0.4 million during the same period in 2025. The foreign currency movements relate to our Hercules loan facility, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.
In connection with our September 2025 follow-on public offering, we issued pre-funded warrants that were classified as a liability and measured at fair value each reporting period. In August 2026, the holder exercised all outstanding pre-funded warrants and we recognized a loss of $0.9 million in the three months ended September 30, 2026, reflecting the change in fair value of the liability through the exercise date, compared to a loss of $5.7 million for the same period in 2025.
Income tax expense
We recognized $0.6 million of deferred tax expense in the three months ended September 30, 2026, and $0.2 million of deferred tax expense for the same period in 2025 related to our U.S. operations. In addition, during the three months ended September 30, 2025, we recorded $7.5 million of current income tax expense and $0.9 million of deferred tax related to our Dutch operations as a result of a revised assessment of the Dutch corporate income tax treatment of the $375.0 million upfront payment received in 2023 under the Royalty Financing Agreement.
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Results of Operations
Comparison of the nine months ended September 30, 2026 and 2025
The following table presents a comparison of our results of operations for the nine months ended September 30, 2026 and 2025:
| | | | | | | | | |
| | Nine months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
License revenues | | $ | 14,599 | | $ | 10,411 | | $ | 4,188 |
Collaboration revenues | | | — | | | 119 | | | (119) |
Total revenues | | | 14,599 | | | 10,530 | | | 4,069 |
Operating expenses: | | | | | | | | | |
Cost of license revenues | | | (805) | | | (1,251) | | | 446 |
Research and development expenses | | | (98,657) | | | (105,889) | | | 7,232 |
Selling, general and administrative expenses | | | (61,353) | | | (43,846) | | | (17,507) |
Total operating expenses | | | (160,815) | | | (150,986) | | | (9,829) |
Other income | | | 4,920 | | | 12,413 | | | (7,493) |
Other expense | | | (9,607) | | | (6,188) | | | (3,419) |
Loss from operations | | | (150,903) | | | (134,231) | | | (16,672) |
Non-operating (expense) / income, net | | | (41,903) | | | (18,107) | | | (23,796) |
Loss before income tax expense | | $ | (192,806) | | $ | (152,338) | | | (40,468) |
Income tax expense | | | (3,005) | | | (9,547) | | | 6,542 |
Net loss | | $ | (195,811) | | $ | (161,885) | | $ | (33,926) |
License revenues
We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the nine months ended September 30, 2026 and 2025, we recognized $14.6 million and $10.4 million of license revenues, respectively.
R&D expense
R&D expenses for the nine months ended September 30, 2026 were $98.7 million, compared to $105.9 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.
| | | | | | | | | |
| | Nine months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Huntington's disease (ifezuntirgene inilparvovec) | | $ | 21,953 | | $ | 30,548 | | $ | (8,595) |
Temporal lobe epilepsy (AMT-260) | | | 10,718 | | | 6,275 | | | 4,443 |
Fabry disease (AMT-191) | | | 5,088 | | | 5,151 | | | (63) |
Amyotrophic lateral sclerosis (AMT-162) | | | 3,969 | | | 4,567 | | | (598) |
Programs in preclinical development and platform related expenses | | | 6,169 | | | 3,104 | | | 3,065 |
Total direct research and development expenses | | $ | 47,897 | | $ | 49,645 | | $ | (1,748) |
| | | | | | | | | |
Employee and contractor-related expenses | | | 28,914 | | | 28,248 | | | 666 |
Facility expenses | | | 9,274 | | | 12,119 | | | (2,845) |
Share-based compensation expense | | | 5,946 | | | 5,916 | | | 30 |
Information technology costs | | | 2,646 | | | 1,861 | | | 785 |
Fair value changes related to contingent consideration | | | 616 | | | 5,293 | | | (4,677) |
Other expenses | | | 3,364 | | | 2,807 | | | 557 |
Total other research and development expenses | | $ | 50,760 | | $ | 56,244 | | $ | (5,484) |
| | | | | | | | | |
Total research and development expenses | | $ | 98,657 | | $ | 105,889 | | $ | (7,232) |
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Direct research and development expenses
Huntington’s disease (ifezuntirgene inilparvovec)
In the nine months ended September 30, 2026 and 2025, we incurred costs related to the development of ifezuntirgene inilparvovec of $22.0 million and $30.5 million, respectively. The decrease of $8.6 million was primarily driven by lower manufacturing costs of $4.6 million in the current period, compared to $11.4 million in the prior year period, and lower costs related to clinical trials of $14.5 million in the current period, compared to $17.1 million in the prior year period. These decreases were partially offset by higher regulatory costs of $1.8 million, compared to $1.1 million in the prior year period, associated with the preparation of BLA and MAA submissions for ifezuntirgene inilparvovec in the United States and the United Kingdom, respectively.
Temporal lobe epilepsy (AMT-260)
In the nine months ended September 30, 2026 and September 30, 2025, we incurred costs related to the development of AMT-260 of $10.7 million and $6.3 million, respectively. The current period included costs of $9.4 million related to clinical trials and $1.0 million related to CMC development, compared to $5.4 million and $0.7 million, respectively, in the prior year period.
Fabry disease (AMT-191)
In the nine months ended September 30, 2026 and September 30, 2025, we incurred costs related to the development of AMT-191 of $5.1 million and $5.2 million, respectively. These costs primarily related to the Phase I/II trial.
Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)
In the nine months ended September 30, 2026 and September 30, 2025, we incurred costs related to the development of AMT-162 of $4.0 million and $4.6 million, respectively. These costs primarily related to the Phase I/II trial.
Preclinical programs & platform development
In the nine months ended September 30, 2026 and September 30, 2025, we incurred costs related to research and technology projects of $6.2 million and $3.1 million, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.
Other research & development expenses
| ● | We incurred $28.9 million in personnel and contractor-related expenses in the nine months ended September 30, 2026, compared to $28.2 million for the same period in 2025; |
| ● | We incurred $9.3 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the nine months ended September 30, 2026, compared to $12.1 million in the same period in 2025. The decrease of $2.8 million was primarily driven by a revision to the estimated useful lives of certain assets, resulting in higher depreciation recognized during the prior year period; |
| ● | We incurred $5.9 million in share-based compensation expenses in the nine months ended September 30, 2026, compared to $5.9 million for the same period in 2025; |
| ● | We incurred $2.6 million in information technology costs in the nine months ended September 30, 2026, compared to $1.9 million for the same period in 2025; |
| ● | We incurred a $0.6 million loss related to an increase in the fair value of contingent consideration associated with the acquisition of uniQure France SAS in the nine months ended September 30, 2026, compared to a $5.3 million loss for the same period in 2025. The $4.7 million favorable change was primarily driven by larger fair value increases in the prior year period, reflecting accelerated expected milestone timing and lower discount rates, whereas in the current year period a delay in expected milestone timing was more than offset by the lower discount rates; and |
| ● | We incurred $3.4 million of other expenses for the nine months ended September 30, 2026, compared to $2.8 million for the same period in 2025. |
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Selling, general and administrative expenses
Selling, general and administrative expenses for the nine months ended September 30, 2026 were $61.4 million, compared to $43.8 million for the same period in 2025.
| | | | | | | | | |
| | Nine months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Employee and contractor-related expenses | | $ | 28,232 | | $ | 17,937 | | $ | 10,295 |
Professional fees | | | 12,321 | | | 11,693 | | | 628 |
Share-based compensation expense | | | 9,606 | | | 7,100 | | | 2,506 |
Intellectual property fees | | | 2,644 | | | 1,146 | | | 1,498 |
Information technology costs | | | 1,869 | | | 784 | | | 1,085 |
Depreciation and facility costs | | | 1,373 | | | 1,424 | | | (51) |
Other expenses | | | 5,308 | | | 3,762 | | | 1,546 |
Total selling, general and administrative expenses | | $ | 61,353 | | $ | 43,846 | | $ | 17,507 |
| ● | We incurred $28.2 million in personnel and contractor-related expenses in the nine months ended September 30, 2026, compared to $17.9 million in the same period in 2025. The $10.3 million increase was primarily driven by higher headcount following employee recruitment to support the potential commercial launches of ifezuntirgene inilparvovec; |
| ● | We incurred $12.3 million in professional fees in the nine months ended September 30, 2026, compared to $11.7 million in the same period in 2025. The increase was primarily related to professional fees incurred to support the preparation of a potential commercialization of ifezuntirgene inilparvovec; |
| ● | We incurred $9.6 million in share-based compensation expenses in the nine months ended September 30, 2026, compared to $7.1 million in the same period in 2025. The $2.5 million increase was primarily driven by new awards granted during the current year period; |
| ● | We incurred $2.6 million in intellectual property fees in the nine months ended September 30, 2026, compared to $1.1 million in the same period in 2025. The $1.5 million increase was primarily driven by higher registration and professional fees. The increase was primarily related to expenses incurred to support the preparation of a potential commercialization of ifezuntirgene inilparvovec. |
| ● | We incurred $1.9 million in information technology costs in the nine months ended September 30, 2026 compared to $0.8 million in the same period in 2025. The increase was primarily related to expenses incurred to support the preparation of a potential commercialization of ifezuntirgene inilparvovec; |
| ● | We incurred $1.4 million in depreciation and facility costs in the nine months ended September 30, 2026 compared to $1.4 million in the same period in 2025; and |
| ● | We incurred $5.3 million in other expenses in the nine months ended September 30, 2026, compared to $3.8 million in the same period in 2025. |
Other income and expense
| | | | | | | | | |
| | Nine months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Supply of HEMGENIX® to CSL Behring | | | (8,809) | | | (4,842) | | | (3,967) |
Research and development grants from Dutch authorities | | | 3,195 | | | 4,123 | | | (928) |
Sublease income, net | | | 915 | | | 402 | | | 513 |
Other income, net | | | 12 | | | 542 | | | (530) |
Sale of critical reagents to Genezen | | $ | — | | $ | 6,000 | | $ | (6,000) |
Total other items, net | | $ | (4,687) | | $ | 6,225 | | $ | (10,912) |
In April 2026, we entered into an agreement to terminate the Genezen CSA. In connection with the termination, we recorded a write-down of $5.9 million related to the favorable supply intangible asset in Other expense.
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Other non-operating items, net
Our other non-operating items, net, for the nine months ended September 30, 2026 and September 30, 2025 were as follows:
| | | | | | | | | |
| | Nine months ended September 30, | |||||||
| | 2026 | | 2025 | | 2026 vs 2025 | |||
| | (in thousands) | |||||||
Interest income | | | 18,394 | | | 11,154 | | | 7,240 |
Interest expense - Royalty Financing Agreement | | | (38,748) | | | (40,885) | | | 2,137 |
Interest expense - Hercules loan facility | | | (4,219) | | | (5,415) | | | 1,196 |
Interest expense - other | | | (6) | | | (1,121) | | | 1,115 |
Changes in fair value of liability related to pre-funded warrants | | | (13,083) | | | (5,722) | | | (7,361) |
Foreign currency (losses) / gains, net | | | (4,241) | | | 25,382 | | | (29,623) |
Issuance costs - Pre-Funded Warrants | | | — | | | (1,500) | | | 1,500 |
Total Other non-operating items, net | | $ | (41,903) | | $ | (18,107) | | $ | (23,796) |
We recognize interest income associated with our cash and cash equivalents, investment securities and convertible note. We recognized $18.4 million in interest income in the nine months ended September 30, 2026, compared to $11.2 million in the same period in 2025. The $7.2 million increase was primarily due to higher average balances of investment securities held during the current year period, compared to the prior year period.
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 $38.7 million and $40.9 million in the nine months ended September 30, 2026 and 2025, respectively.
We recognized interest expense related to the Hercules loan facility of $4.2 million in the nine months ended September 30, 2026, compared to $5.4 million in the same period in 2025. In July 2026, the Company entered into an amendment to its venture debt loan facility with Hercules Capital, Inc. (as amended, the "2026 Amended Facility"). Except as provided for in the amendment, the terms of borrowing remained unchanged. The $1.2 million decrease was primarily due to more favorable terms following the amendment of the facility in September 2025, as well as a decrease in market interest rates.
In connection with our September 2025 follow-on public offering, we issued pre-funded warrants that were classified as a liability and measured at fair value each reporting period. In August 2026, the holder exercised all outstanding pre-funded warrants and we recognized a loss of $13.1 million in the nine months ended September 30, 2026, reflecting the change in fair value of the liability through the exercise date, compared to a loss of $5.7 million for the same period in 2025.
We recognized a net foreign currency loss of $4.2 million in the nine months ended September 30, 2026, compared to a net gain of $25.4 million in the same period in 2025. The foreign currency movements related to our borrowings from Hercules, the Royalty Financing Agreement, cash and cash equivalents, investment securities, and intercompany loans within the uniQure group.
Income tax expense
We recognized $2.9 million of deferred tax expense in the nine months ended September 30, 2026, compared to $1.1 million in the same period in 2025 related to our U.S. operations. In addition, during the nine months ended September 30, 2025, we recorded $7.5 million of current income tax expense and $0.9 million of deferred tax expense related to our Dutch operations as a result of a revised assessment of the Dutch corporate income tax treatment of the $375.0 million upfront payment received in 2023 under the Royalty Financing Agreement.
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Financial Position, Liquidity and Capital Resources
As of September 30, 2026, we had cash and cash equivalents, restricted cash and investment securities of $770.0 million. We believe these resources will be sufficient to fund our projected operating expenses into 2030, including costs associated with the commercial launches and confirmatory study of ifezuntirgene inilparvovec, ongoing clinical trials of ifezuntirgene inilparvovec, AMT-191 and AMT-260, and potential investments to advance certain pipeline candidates into late-stage development. The amount and timing of our actual expenditures may vary significantly depending on the commencement and design of any new clinical studies 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.
This includes up to $100.0 million in additional availability under our Hercules senior secured term loan facility, which becomes accessible upon BLA approval for ifezuntirgene inilparvovec prior to September 30, 2027, subject to confirmatory trial requirements remaining on track. 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 September 30, 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 ifezuntirgene inilparvovec is approved prior to October 1, 2028. The interest-only period will be further extended to October 1, 2030 if certain commercial milestones are met prior to March 31, 2029. Future contractual interest payments (assuming repayments commence on October 1, 2028) associated with the loan are $14.9 million, with $4.8 million payable within the next 12 months.
Leases
We have entered into lease arrangements for facilities, including corporate, laboratories and office space. As of September 30, 2026, we had fixed lease payment obligations of $18.3 million, with $4.5 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 September 30, 2026, we remain obligated under a guarantee of lease payments of $12.8 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 September 30, 2026, our remaining commitment amounts include EUR 160.0 million ($181.7 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 September 30, 2026 of $1.14/€1.00. As of September 30, 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 nine months ended September 30, 2026 and 2025:
| | | | | | |
| | Nine months ended September 30, | ||||
| | 2026 | | 2025 | ||
| | (in thousands) | ||||
Cash, cash equivalents and restricted cash at the beginning of the period | | $ | 81,801 | | $ | 160,329 |
Net cash used in operating activities | | | (114,646) | | | (100,394) |
Net cash (used in) / generated from investing activities | | | (116,912) | | | 119,751 |
Net cash generated from financing activities | | | 251,223 | | | 412,949 |
Foreign exchange impact | | | (1,494) | | | 5,914 |
Cash, cash equivalents and restricted cash at the end of period | | $ | 99,972 | | $ | 598,549 |
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 $61.2 million and $195.8 million in the three and nine months ended September 30, 2026, compared to a net loss of $80.5 million and $161.9 million during the same period in 2025. As of September 30, 2026, we had an accumulated deficit of $1,524.7 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®.
In June 2026, we received aggregate net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, following the completion of a follow-on public offering of 5.7 million ordinary shares at a public offering price of $45.50 per ordinary share.
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.
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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.
Net cash used in operating activities
Net cash used in operating activities was $114.6 million for the nine months ended September 30, 2026 and consisted of a net loss of $195.8 million, adjusted for non-cash items including depreciation and amortization expense of $13.3 million, amortization of the discount on investment securities of $13.6 million, share-based compensation expense of $15.6 million, $23.4 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $2.9 million, changes in the fair value of contingent consideration of $0.6 million, changes in the fair value of the liability related to pre-funded warrants of $13.1 million, and unrealized foreign exchange losses of $5.1 million. Net cash used in operating activities also included favorable changes in operating assets and liabilities of $24.4 million. There was a net decrease in accounts receivable, accrued income, prepaid expenses, and other current assets and receivables of $10.1 million. There was a net increase in accounts payable, accrued expenses, and operating leases of $14.3 million.
Net cash used in operating activities was $100.4 million for the nine months ended September 30, 2025 and consisted of net loss of $161.9 million adjusted for non-cash items, including depreciation and amortization expense of $11.8 million, amortization of the discount on investment securities of $6.1 million, share-based compensation expense of $13.0 million, $29.2 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $2.0 million, changes in the fair value of contingent consideration of $5.3 million, changes in the fair value of the liability related to pre-funded warrants of $5.7 million, unrealized foreign exchange gains of $24.5 million, and other items, net, of $3.0 million. Net cash used in operating activities also included favorable changes in operating assets and liabilities of $28.1 million. There was a net decrease in accounts receivable, prepaid expenses, and other current assets and receivables of $2.9 million. There was a net increase in accounts payable, accrued expenses, other liabilities, and operating leases of $25.2 million.
Net cash (used in) / generated from investing activities
In the nine months ended September 30, 2026, we used $116.9 million in investing activities compared to $119.8 million generated during the same period in 2025.
| | | | | | |
| | Nine months ended September 30, | ||||
| | 2026 | | 2025 | ||
| | (in thousands) | ||||
Cash flows from investing activities | | | | | | |
Proceeds from maturity of debt securities | | $ | 466,265 | | $ | 337,195 |
Investment in debt securities | | | (582,554) | | | (217,124) |
Purchases of property, plant, and equipment | | | (623) | | | (320) |
Net cash (used in) / generated from investing activities | | $ | (116,912) | | $ | 119,751 |
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Net cash generated from financing activities
In the nine months ended September 30, 2026, net cash generated from financing activities was $251.2 million, compared to net cash generated from financing activities of $412.9 million in the same period in 2025.
| | | | | | |
| | Nine months ended September 30, | ||||
| | 2026 | | 2025 | ||
| | (in thousands) | ||||
Cash flows from financing activities | | | | | | |
Proceeds from follow-on public offering of ordinary shares, net of issuance costs | | $ | 242,682 | | $ | 380,737 |
Proceeds from issuance of pre-funded warrants, net of issuance costs | | | — | | | 23,499 |
Proceeds from issuance of ordinary shares related to employee stock options and purchase plans | | | 8,541 | | | 8,713 |
Net cash generated from financing activities | | $ | 251,223 | | $ | 412,949 |
Funding requirements
Our future capital requirements will depend on many factors, including but not limited to:
| ● | activities to prepare for the potential commercialization of ifezuntirgene inilparvovec for Huntington’s disease; |
| ● | investments required to support the MAAs of ifezuntirgene inilparvovec or generate confirmatory evidence in conjunction with obtaining accelerated approval; |
| ● | investments in the late stage development of AMT-191 and AMT-260; |
| ● | earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to achieving 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 nine months ended September 30, 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.
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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 September 30, 2026. Based on such evaluation, our CEO and CFO concluded that as of September 30, 2026, our disclosure controls and procedures were effective to ensure that information required to be disclosed by us 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, 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 (collectively, “Defendants”) in the United States District Court for the Southern District of New York. The complaint purported to assert claims pursuant to Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), 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 (the “putative Class Period”). The plaintiff sought to recover damages allegedly caused by purported false and misleading statements and omissions with respect to our Phase I/II study of ifezuntirgene inilparvovec and the timing of the potential BLA filing for ifezuntirgene inilparvovec.
On April 13, 2026, the court appointed the Oklahoma Firefighters Pension and Retirement System as lead plaintiff (the “Lead Plaintiff”).
On July 15, 2026, the Lead Plaintiff filed an amended complaint. Like the earlier-filed complaint, the amended complaint purports to assert claims pursuant to Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5, on behalf of a putative class of investors who purchased or otherwise acquired our ordinary shares during the putative Class Period. Lead Plaintiff seeks unspecified damages allegedly caused by purported false and misleading statements and omissions with respect to the import and viability of data from our Phase I/II study of ifezuntirgene inilparvovec and available pathways to regulatory approval. Defendants’ motion to dismiss the amended complaint is currently due November 4, 2026. Management intends 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. We have not recorded any liability for any portion of this matter because we believe that liability is not probable and reasonably estimable at this time.
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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 year ended December 31, 2025, as supplemented by “Item 1A. Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2026 and the risk factor set forth below. 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, as supplemented by our Quarterly Report on Form 10-Q for the quarter ended June 30, 2026 and the risk factor set forth below.
Risks Related to Our Business and the Development of Our Clinical Product Candidates
We are dependent on the success of our lead clinical product candidate, ifezuntirgene inilparvovec, for the treatment of Huntington’s disease. A failure of ifezuntirgene inilparvovec in clinical development, including inability to demonstrate sufficient safety or efficacy, or challenges associated with its regulatory approval, manufacturing or commercialization could adversely affect our business.
We have invested a significant portion of our development efforts and financial resources in the development of our lead clinical product candidate, ifezuntirgene inilparvovec, for the treatment of Huntington’s disease.
We submitted our BLA for ifezuntirgene inilparvovec to the FDA in August 2026 and the FDA accepted the application for review in October 2026, with a PDUFA target action date of May 1, 2027. In its acceptance letter, the FDA stated that they are tentatively planning to discuss our application at an Advisory Committee meeting. In the third quarter of 2026, we submitted our MAA for ifezuntirgene inilparvovec to the MHRA, which was subsequently validated by the MHRA.
We can provide no assurance that our marketing application will result in approval of ifezuntirgene inilparvovec in Huntington’s disease by the FDA or any other regulatory authorities, that the FDA or any other regulatory authorities will be able to make approval decisions by their respective target action dates, that the FDA or any other regulatory authorities will agree that we have met their respective requirements, if any, for a confirmatory study, or that the FDA or any other regulatory authorities will not require advisory committees to be convened as part of their review of respective applications.
There are numerous other factors that could impede or otherwise negatively impact our further development of ifezuntirgene inilparvovec, including, but not limited to: potential patient safety issues; our failure to demonstrate sufficient clinical efficacy or durability of response to warrant approval by the FDA or any other regulatory authority; our failure to demonstrate that our regulatory submissions otherwise meet the requirements for approval; shifting regulatory standards for approval; regulatory authorities may disagree with our interpretation of the data; our current beliefs and understanding of the communications with regulatory authorities regarding the further development of and approval pathway for ifezuntirgene inilparvovec could be incorrect, and the success of our efforts to address such communications and interactions; the results from future interim or topline data analyses and readouts from our ongoing Phase I/II trials, including as additional patient data becomes available; any requirement for additional studies to obtain approval, including a confirmatory study; our ability to fund and enroll a confirmatory study, including engaging sites that may be needed to enroll participants in a confirmatory study; the timing, cost and resources associated with our planned marketing applications; our ability to successfully commercialize ifezuntirgene inilparvovec should we choose to do so without a partner; challenges with potential development or commercial partners, should we choose to pursue further development or commercialization of ifezuntirgene inilparvovec with a partner; and our ability to fund the further development and commercialization of the ifezuntirgene inilparvovec program.
Any one or combination of these factors could force us to halt or discontinue the ongoing clinical trials of ifezuntirgene inilparvovec or related commercialization efforts or could prevent us from obtaining marketing approval within the predicted timeframes or at all. Certain of these risks are heightened in the context of drug development for rare diseases like Huntington’s disease and novel investigational products like gene therapies in which non-traditional study designs may be utilized to demonstrate efficacy and safety, including open-label studies, single arm studies, studies utilizing active comparators or natural history data, biomarkers or other forms of surrogate endpoints, which
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may be utilized due to the challenges inherent in designing and conducting clinical trials for severe diseases that progress slowly and that affect small patient populations.
The FDA has broad discretion with regard to licensure, including through the Accelerated Approval Program, and the FDA may require additional studies and trials beyond those that we currently contemplate. Furthermore, even if the FDA reviews a BLA seeking approval, including accelerated approval, there can be no assurance that licensure will be granted on a timely basis, or at all. The FDA may disagree that the design of, or results from, our studies and statistical analysis plan (“SAP”) support accelerated approval. Additionally, the FDA may require us to conduct further studies or trials prior to granting licensure of any type, including by determining that licensure through the Accelerated Approval Program is not appropriate and that our clinical trials and SAP for ifezuntirgene inilparvovec may not be used to support licensure through the conventional pathway. We might not be able to fulfill the FDA’s requirements in a timely manner, which would cause delays, or licensure might not be granted because our submission is deemed incomplete by the FDA. Furthermore, the feedback or requests we receive from the FDA, or other non-U.S. regulatory authority, may be difficult or impossible to implement. There also can be no assurance that after subsequent FDA feedback we will continue to pursue licensure, including through the Accelerated Approval Program or otherwise. A failure to obtain licensure for ifezuntirgene inilparvovec could delay or prevent our ability to launch and commercialize ifezuntirgene inilparvovec in the U.S., could result in a longer time period to pursue licensure of our other clinical product candidates or increase the cost of development of our other clinical product candidates, and could significantly harm our financial position and competitive position in the marketplace.
The applications submitted to the FDA and MHRA predated the subsequently reported 48-month topline results, and therefore the 48-month topline results were not part of the BLA or MAA. In the 12 high-dose patients with 48 months of follow-up, the pre-specified primary endpoint for the 48-month analysis, cUHDRS, did not reach statistical significance. Results on other measures, including TFC and our updated 36-month analysis in all 15 high-dose patients, are based on nominal p-values that are not adjusted for multiplicity. External controls have inherent limitations, including missing data and selection bias, and the FDA or other regulatory authorities may consider the 48-month topline results in their reviews and may disagree with our interpretation of them or of the limitations of the external control.
We cannot be certain that ifezuntirgene inilparvovec, or any of our clinical product candidates, will be successful in clinical trials or ultimately receive regulatory approval. If we were required, or if we chose, to discontinue development of ifezuntirgene inilparvovec or any other current or future product candidate, or if any of them were to fail to receive regulatory approval or achieve sufficient market acceptance, we could be prevented from or significantly delayed in achieving profitability and our business would be adversely affected.
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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 September 30, 2026, no
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
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3.1* | Amended Articles of Association of uniQure N.V. |
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10.1* | First Amendment to Lease related to 1 Hartwell Place, Lexington, Massachusetts, dated as of July 31, 2026, by and between uniQure, Inc. and NRL 91 Hartwell LLC. |
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10.2+ | Amendment No. 4 to Third Amended and Restated Loan and Security Agreement, dated July 27, 2026, by and among uniQure biopharma, B.V., uniQure, Inc., uniQure IP B.V., uniQure N.V. and Hercules Capital, Inc. (incorporated by reference to Exhibit 10.2 of the Company’s Quarterly Report on Form 10-Q for the period ended June 30, 2026 (file no. 001-36294) filed with the SEC on July 29, 2026). |
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31.1* | Certification of Chief Executive Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the Securities and Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002. |
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31.2* | Certification of Chief Financial Officer pursuant to Rule 13a-14(a) or 15d-14(a) of the Securities and Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002. |
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32.1± | Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002. |
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101* | The following financial information from our Quarterly Report on Form 10-Q for the period ended September 30, 2026, filed with the Securities and Exchange Commission on November 4, 2026, is formatted in Inline Extensible Business Reporting Language (“iXBRL”): (i) Consolidated Balance Sheets; (ii) Consolidated Statements of Operations and Comprehensive Loss; (iii) Consolidated Statements of Shareholders’ Equity; (iv) Consolidated Statements of Cash Flows; and (v) Notes to Consolidated Financial Statements (tagged as blocks of text). |
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104* | The cover page from our Quarterly Report on Form 10-Q for the period ended September 30, 2026, filed with the Securities and Exchange Commission on November 4, 2026, is formatted in Inline Extensible Business Reporting Language (“iXBRL”). |
* Filed herewith.
± Furnished herewith.
+Portions of this exhibit have been omitted pursuant to Item 601(b)(10)(iv) of Regulation S-K.
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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| UNIQURE N.V. |
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| By: /s/ Matthew Kapusta Matthew Kapusta Chief Executive Officer (Principal Executive Officer) |
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| By: /s/ Christian Klemt Christian Klemt Chief Financial Officer (Principal Financial and Accounting Officer) |
Date: November 4, 2026
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