uniQure reports $61.22 million net loss for Q3 2026

Cover page 2 changes

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For the quarterly period ended June September 30, 2026

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As of July 24, November 2, 2026, the registrant had 69,366,412 70,138,860 ordinary shares, par value €0.05, outstanding.

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Exhibits 2 changes

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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 Authorization Authorisation Application for AMT-130 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 AMT-130; 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 AMT-130; ifezuntirgene inilparvovec ; our ability to achieve long-term growth; and developments and projections relating to our competitors in the industry .

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You should not place undue reliance on these forward-looking statements, which speak only as of the date that they were made. Our actual results or experience could differ significantly from those anticipated in the forward-looking statements and from historical results, due to the risks and uncertainties described in this Quarterly Report on Form 10-Q and in our Annual Report , including in "Part I, Item 1A. Risk Factors," as well as others that we may consider immaterial or do not anticipate at this time. These cautionary statements should be considered in connection with any written or oral forward-looking statements that we may make in the future or may file or furnish with the SEC. We do not undertake any obligation to release publicly 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.

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Financial statements 45 changes

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Non-current investment securities

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Ordinary shares, € 0.05 par value: 100,000,000 shares authorized at June September 30, 2026, and 80,000,000 at December 31, 2025, and 69,334,839 70,044,739 and 62,336,717 shares issued and outstanding at June September 30, 2026, and December 31, 2025, respectively.

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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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Collaboration revenues

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Foreign currency translation gains / (losses), net

Foreign currency translation (losses) / gains, net

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FOR THE THREE MONTHS ENDED JUNE SEPTEMBER 30, 2026 AND 2025

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Balance at March 31, 2025

Other comprehensive loss, net

Exercise of share options

Restricted share units distributed during the period

Share-based compensation expense

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Balance at March 31, 2026

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Tax benefit related to historical share issuance

Exercises Exercise of share options

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Balance at September 30, 2025

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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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FOR THE SIX NINE MONTHS ENDED JUNE SEPTEMBER 30, 2026 AND 2025

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Follow-on public offering offerings

Tax benefit related to historical share issuance costs

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Restricted and performance share units distributed during the period ( 43 45 )

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Balance at June September 30, 2025

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Exercise of pre-funded warrants

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Balance at June September 30, 2026

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Six months ended June 30,

Cash flows from operating activities

Nine months ended September 30,

Cash flows used in operating activities

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Unrealized foreign exchange losses / (gains), net

Unrealized foreign exchange gains / (losses), net

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Accounts receivable, prepaid expenses, and other current assets and receivables ( 6,761 1,648 )

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Cash flows (used in) / generated from investing activities

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Net cash (used in) / generated from investing activities

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Proceeds from issuance of pre-funded warrants, net of issuance costs

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Currency effect on cash, cash equivalents and restricted cash ( 1,906 1,494 )

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Supplemental disclosure of non-cash investing and financing items:

Issuance of ordinary shares upon exercise of pre-funded warrants

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Notes to the financial statements 13 changes

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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 six nine months ended June 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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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 six nine months ended June September 30, 2026.

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There have been no new accounting pronouncements or changes to accounting pronouncements during the six nine months ended June 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.

Current Investment securities

The following tables summarize the Company's investments in government debt securities as of June September 30, 2026 and December 31, 2025:

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Non-current investments securities:

Government debt securities (held-to-maturity)

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The Company invests in short-term U.S. and European government debt securities with high investment credit ratings. The U.S. and European government bonds are U.S. dollar and euro (€, or EUR) denominated, respectively. Investment securities with original maturities of 90 days or less when purchased are presented within cash and cash equivalents and measured at amortized cost. 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 June September 30, 2026 and December 31, 2025, no expected credit loss was identified with respect to the investment securities.

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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 June 30, 2026 and December 31, 2025 : and September 30, 2026:

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Balance at June September 30, 2026

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Liability related to pre-funded warrants

Liability related to pre-funded warrants

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Note A: Contingent consideration 8 changes

The Company is required to pay up to EUR 143.1 million (or $ 163.0 162.4 million based on the foreign exchange rate on June September 30, 2026) to the former shareholders of uniQure France SAS (formerly Corlieve Therapeutics SAS) upon the achievement of the remaining contractually defined milestones related to the development of AMT-260 in connection with the Company's acquisition of uniQure France SAS.

The fair value of the contingent consideration as of June September 30, 2026 was EUR 15.9 16.5 million ($ 18.1 18.7 million) (December 31, 2025: EUR 15.9 million ($ 18.7 million)) using discount rates of approximately 10.1 9.7 % to 11.8 % (December 31, 2025: 11.8 %).

If, as of June September 30, 2026, the Company had assumed a 100 % likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 51.3 53.2 million ($ 58.4 60.4 million). If, as of June September 30, 2026, the Company had assumed that it would discontinue development of the AMT-260 program, then the contingent consideration would have been released to income.

The following table presents the changes in fair value of the contingent consideration (presented within non-current liabilities) between December 31, 2025 and June September 30, 2026:

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Unrealized change in fair value

Unrealized change in fair value (presented within Research and development expenses)

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Balance at June September 30, 2026

The Company classified the total contingent consideration liability as non-current as of June 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.

Note B: Liability related to pre-funded warrants 72 changes

In September 2025, the Company completed a follow-on public offering which included the issuance of pre-funded warrants to purchase 526,316 of the Company's ordinary shares at the public offering price of $ 47.50 per ordinary share, less a $ 0.0001 per ordinary share exercise price for each pre-funded warrant ("Pre-Funded Warrant(s)"). The obligation to issue ordinary shares upon the exercise of the Pre-Funded Warrants is classified as a liability related to pre-funded warrants.

The Pre-Funded Warrants are not exchange-quoted and are measured using a valuation technique whose significant inputs are observable; therefore, the fair value measurement is classified within Level 2 of the fair value hierarchy.

In connection with its September 2025 follow-on public offering, the Company issued pre-funded warrants to purchase 526,316 ordinary shares at a price per warrant equal to the public offering price of $ 47.50 per ordinary share less the $ 0.0001 per share exercise price ("Pre-Funded Warrants"). The obligation to issue ordinary shares upon exercise was classified as a liability and measured at fair value using a valuation technique whose significant inputs were observable; the fair value measurement was therefore classified within Level 2 of the fair value hierarchy.

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").

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Unrealized Realized increase in fair value (presented within Other non-operating (losses), net)

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Settlement of pre-funded warrant liability through issuance of ordinary shares ( 25,732 )

Balance at June September 30, 2026

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Accrued expenses and other current liabilities include consisted of the following items as of September 30, 2026 and December 31, 2025:

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Personnel-related accruals and liabilities (As of June 30, 2026, $ 7.2 million (December 31, 2025: $ 0.0 million) related to cash proceeds from employees' stock option exercises)

Personnel-related accruals and liabilities

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Current portion of firm purchase commitment liability (including and Genezen CSA termination payment

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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, our the Company's obligation to supply HEMGENIX® ® and any minimum purchase commitments under the Genezen CSA terminate once contractually specified batches have been supplied. In connection with the termination, the Company wrote down the remaining carrying value of the intangible asset for the favorable supply terms under the Genezen CSA. The net effect of these items was recorded in Other expense, and did not have a material impact on the Company's results of operations for the three and six months ended June 30, 2026. terminated.

In July 2026, subsequent to the balance sheet date, 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 $ 100.0 million term loan tranche from June 15, 2027 to September 30, 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility remained unchanged.

The total principal outstanding as of June 30, 2026 under the Company's amended venture debt loan facility with Hercules Capital, Inc. (the "2025 2026 Amended Facility as of September 30, 2026 was $ 50.0 million. The amortized cost was $ 50.7 million as of September 30, 2026 (including $ 0.4 million of interest due presented as part of within Accrued expenses and other current liabilities), was $ 50.5 million as of June 30, 2026, compared to $ 50.1 million as of December 31, 2025, and is recorded net of discount and debt issuance costs.

The foreign currency loss on the loan 2026 Amended Facility in the three and six nine months ended June September 30, 2026 was $ 0.5 0.2 million and $ 1.6 1.8 million, respectively, compared to a foreign currency gain of $ 4.3 2.8 million and $ 6.4 9.2 million, respectively, during the same periods in 2025. Interest expense during the three and six nine months ended June September 30, 2026 was $ 1.4 million and $ 2.8 4.2 million, respectively, compared to $ 1.8 million and $ 3.6 5.4 million, respectively, during the same periods in 2025.

During the six nine months ended June 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 2025 2026 Amended Facility as described in Note 11, " "Long-term debt," " to the consolidated financial statements included in the Company's Annual Report. on Form 10-K for the year ended December 31, 2025. As of June September 30, 2026, the Company was in compliance with all applicable covenants under the 2026 Amended Facility.

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During the six nine months ended June 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 Company's Annual Report. for the year ended December 31, 2025.

The following table presents the changes in the liability from the Royalty Financing Agreement for the period from December 31, 2025 to June September 30, 2026:

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Balance as of June September 30, 2026 (includes $ 5.8 4.8 million presented as "Accrued expenses and other current liabilities")

The Company records the debt at amortized cost using the effective interest method based on projected cash flows. As of June September 30, 2026, the effective interest rate is expected to be within a range of 10.0 % to 11.5 % per annum (June (September 30, 2025: 12.0 10.5 % to 13.5 12.0 %). Interest expense recorded in connection with the Royalty Financing Agreement was $ 13.0 13.2 million and $ 13.8 million for the three months ended June September 30, 2026 and 2025, respectively, and $ 25.6 38.7 million and $ 27.1 40.9 million for the six nine months ended June September 30, 2026 and 2025, respectively.

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In August 2026, all outstanding Pre-Funded Warrants were exercised by the holder, and the Company issued 526,314 ordinary shares from its reserve account. Upon exercise, the fair value of the liability related to Pre-Funded Warrants of EUR 22.0 million ($ 25.7 million) was reclassified to shareholders' equity.

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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"). At the annual general meeting of shareholders in June 2026, the Company's shareholders authorized an additional 350,000 ordinary shares for issuance under the 2014 Plan. As of June September 30, 2026, a total of 2,815,628 2,698,090 ordinary shares remain available for issuance under the 2014 Plan.

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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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As of June September 30, 2026, the unrecognized share-based compensation expense related to unvested awards under the 2014 Plans was:

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The following table summarizes option activity under the 2014 Plans for the six nine months ended June September 30, 2026:

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Outstanding at June September 30, 2026

Thereof, fully vested, and exercisable on June September 30, 2026

Thereof, outstanding and expected to vest after June September 30, 2026

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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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The following table summarizes the RSU activity for the six nine months ended June September 30, 2026:

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Non-vested at June September 30, 2026

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The Company recognizes the compensation cost related to these grants to the extent it considers achievement of the milestones to be probable. As of June 30, 2026, certain 2026 PSUs were assessed as probable of achieving the requisite performance condition.

As of June 30, 2026, the Company determined that the performance target for the outstanding 2024 PSUs were not met within the contractually defined performance period and were consequently forfeited.

The Company has granted PSUs to certain employees in 2024 and 2026, that, with respect to the 2024 PSUs, would have been earned upon the Board of Directors' assessment of the level of achievement of an agreed upon performance target, and, with respect to 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 in each case, to the grantee's continued employment. The fair value of PSUs granted in 2024 and 2026 was determined on the grant date by reference to the market price of the Company's ordinary shares.

The 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 six nine months ended June September 30, 2026:

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Non-vested at June September 30, 2026

During the six nine months ended June September 30, 2026, 13,939 21,368 ordinary shares were issued under the ESPP compared to nil ordinary shares issued during the same period in 2025. As of June September 30, 2026, 72,773 65,344 ordinary shares remain available for issuance under the ESPP compared to a total of 86,712 ordinary shares as of June September 30, 2025.

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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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(2) The comparative amounts for the three and six nine months ended June 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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The following table summarizes the Company's Other investments as of June September 30, 2026 and December 31, 2025:

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In 2024, as part of the Company's divestment of its commercial manufacturing operations to Genezen, the Company entered into various service agreements including the Genezen CSA. In April 2026, the Company entered into an agreement to terminate the Genezen CSA. Pursuant to the termination agreement, the Company's obligation to supply HEMGENIX ® and any minimum purchase commitments under the Genezen CSA terminate once contractually specified batches have been supplied.

The Company's prepayments for services to be received amounted to $ 5.7 million as of June 30, 2026, compared to $ 13.9 million as of December 31, 2025. In addition, as of June 30, 2026, the Company holds a convertible note receivable of $ 15.1 million which is due in September 2029 and for which Genezen is the counterparty (December 31, 2025: $ 14.6 million).

These balances represent a concentration of credit risk, and the Company's maximum exposure to loss is the carrying value of these assets. No expected credit losses were recognized during the three and six months ended June 30, 2026 or 2025.

The Company recorded a deferred tax expense of $ 1.8 million and $ 2.3 million related to its U.S. operations during the three and six months ended June 30, 2026 ($ 0.4 million and $ 0.9 million for the three and six months ended June 30, 2025).

Genezen convertible note receivable

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 June 30, 2026 and December 31, 2025 was $ 15.1 million and $ 15.6 million, respectively, and are presented within Other Investments in the Company's Consolidated Balance Sheets.

The carrying value of these investments as of September 30, 2026 and December 31, 2025 was $ 15.1 million and $ 15.6 million, respectively, and is presented within Other investments in the Company's Unaudited Consolidated Balance Sheets. The Company did not recognize any upward adjustments, downward adjustments, or impairments relating to investments carried under the measurement alternative during the three and six nine months ended June September 30, 2026, and 2025.

The Company held a convertible promissory note issued in July 2024 by Genezen at a nominal amount of $ 12.5 million, maturing in October 2029 (the "Original Note"). The original note's nominal interest of 8.0 % per annum was payable in kind and is added to the principal at the end of each quarter. The nominal amount as well as the accumulated interest were convertible into Genezen's common stock at $ 2.20 per share.

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 $ 15.1 million and matures in December 2031. The Amended Note bears nominal interest at 15.0 % per annum, payable in kind, and is added to the principal at the end of each quarter. The nominal amount as well as 8.0 % of the total 15.0 % interest per annum are convertible into Genezen's common stock at the lesser of $ 2.20 and the lowest price per share of Genezen's common stock issued in an equity issuance consummated while the Amended Note is outstanding.

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 $ 16.4 million as of September 30, 2026 (December 31, 2025: $ 14.6 million) and is presented within Other investments. No material allowance for expected credit losses was recognized as of September 30, 2026.

As of June September 30, 2026, and December 31, 2025, accounts receivable from CSL Behring LLC ("CSL Behring") totaled $ 5.8 4.8 million and $ 5.9 million, respectively, in each case related to royalty revenue which is included in License Revenue Revenues in the accompanying Unaudited Consolidated Statements of Operations and Comprehensive Loss.

The Company recorded a deferred tax expense of $ 0.6 million and $ 2.9 million related to its U.S. operations during the three and nine months ended September 30, 2026 ( $ 0.2 million and $ 1.1 million for the three and nine months ended September 30, 2025). In addition, during the three and nine months ended September 30, 2025, the Company recorded $ 7.5 million of current income tax expense and $ 0.9 million of deferred income tax expense related to its 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.

The effective income tax rate of 2.3 1.1 % and 1.8 1.6 % during the three and six nine months ended June September 30, 2026 (three and six nine months ended June September 30, 2025: 1.1 12.0 % and 1.1 6.3 %, respectively) is substantially lower than the enacted rate of 25.8 % in the Netherlands as the Company recorded a valuation allowance against its net deferred tax assets in the Netherlands and a partial valuation allowance against its net deferred tax assets in France, among various other items which impacted the effective rate.

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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 six nine months ended June 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 six nine months ended June 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 June September 30, 2026 and June September 30, 2025, respectively.

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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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Stock Share options under 2014 Plans

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The Pre-Funded Warrants were fully exercised in August 2026 (refer to Note 4, "Fair value measurement").

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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 3 changes

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 AMT-130 ifezuntirgene inilparvovec and the timing of the potential Biologics License Application filing for AMT-130. ifezuntirgene inilparvovec.

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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 AMT-130 ifezuntirgene inilparvovec and available pathways to regulatory approval. Defendants' motion to dismiss the amended complaint is currently due September 14, November 4, 2026. Management intends to vigorously defend against the claims in this action.

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In July 2026, subsequent to the balance sheet date, the Company entered into an amendment to its loan facility with Hercules Capital, Inc. (as amended, the "2026 Amended Facility"). The amendment extended the period during which the Company may draw the $ 100.0 million term loan tranche from June 15, 2027 to September 30, 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility remained unchanged.

MD&A 183 changes

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Business Developments

In June 2026, we received net proceeds of $242.7 million, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 5.7 million ordinary shares, at a public offering price of $45.50 per ordinary share.

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 otherwise remain unchanged.

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Huntington's disease program (AMT-130) (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 AMT-130 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 high dose of AMT-130 ifezuntirgene inilparvovec in a fourth cohort to evaluate the safety and efficacy of AMT-130 ifezuntirgene inilparvovec in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study .

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.

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● Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test ("SDMT"), Stroop Word Reading Test ("SWRT") and Total Motor Score ("TMS").

o An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of -0.44 compared to a change of -3.73 for patients in the propensity score-matched external control.

o A 113% slowing of disease progression as measured by SWRT (nominal p=0.002), with a mean change in SWRT from baseline of 0.88 compared to a change of -6.98 for patients in the propensity score-matched external control.

o A 59% slowing of disease progression as measured by TMS (nominal p=0.174), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control.

A mean reduction from baseline in cerebrospinal neurofilament light protein ("CSF NfL") of -8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies. CSF NfL is a well-characterized, supportive biomarker of neurodegeneration. Elevation in CSF NfL has been shown to be strongly associated with greater clinical severity of Huntington's disease.

We believe that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the high-dose group, compared to the variable trends observed in the low-dose group, reflect a dose-dependent response to AMT-130.

Various other supportive analyses of the results from the AMT-130 high-dose treatment group, including those using a propensity score-weighted external control and comparisons to the TRACK-HD and PREDICT-HD datasets, were consistent with the primary analysis.

From November 2024 through April 2025, we held three Type B meetings with the U.S. Food and Drug Administration (the "FDA") . As part of these interactions, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis of a Biologics License Application (" BLA") submission under the FDA's accelerated approval pathway. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and reductions in CSF NfL may serve as supportive evidence of therapeutic benefit in the application for such accelerated approval.

In October 2025, we met with the FDA at a pre-BLA meeting to discuss the application for AMT-130. In December 2025, we announced that in the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 were currently unlikely to provide the primary evidence to support a BLA submission.

In January 2026, we met with the FDA at a Type A meeting to discuss AMT-130. In March 2026, following receipt of the final meeting minutes from the Type A meeting, we announced that the FDA stated that it could not agree that data from the Phase I/II studies, compared to an external control, were sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.

In March 2026, we held a successful pre-submission meeting with the United Kingdom's ("UK") Medicines and Healthcare products Regulatory Agency ("MHRA") regarding AMT-130, and the regulatory submission is progressing as planned for the third quarter of 2026.

In June 2026, we held a Type B meeting with the FDA. Official meeting minutes received in July 2026 confirmed that we and the FDA reached alignment that a BLA submission under the accelerated approval pathway for AMT-130, based on the existing clinical data, is reasonable. In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of a randomized standard-of-care control design instead of a sham procedure. The FDA also stated that, in accordance with the FDA's draft public guidance for accelerated approvals, the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway, and potentially fully enrolled, at the time of accelerated approval. Discussions with the FDA to align on the confirmatory study design and analysis are underway, and we expect to submit a BLA in the third quarter of 2026.

In September 2025, 2026, we announced positive topline additional data from the three-year analysis of cohorts one and two of the ongoing Phase I/II studies of AMT-130 for the treatment of Huntington's disease. ifezuntirgene inilparvovec at two timepoints – 36 months and 48 months. We analyzed clinical outcomes for 29 patients treated with AMT-130 ifezuntirgene inilparvovec (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 36 48 months of follow-up and were evaluated at that time point. Outcomes for each dose group were compared to a propensity score-matched external control drawn from In addition, the Enroll-HD natural history data set (n=940 for 36-month analysis now includes 15 high-dose n=626 for 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 AMT-130 ifezuntirgene inilparvovec were as follows (data cutoff as of June 30, 2025): 2026):

High-Dose Results at 36 Months (n=15)

● A statistically significant 75% slowing of disease progression as measured by composite Unified Huntington's Disease Rating Scale ("cUHDRS") (p=0.003), which met the primary endpoint showed 80% slowing of disease progression compared to the study. updated external control (nominal p=0.005). Treated patients had a mean change in cUHDRS from baseline of -0.38 -0.28 compared to a change of -1.52 -1.39 for patients in the propensity score-matched external control, a favorable treatment difference of 1.12 compared to baseline.

● A statistically significant 60% TFC showed 67% slowing of disease progression as measured by Total Function Capacity ("TFC") (p=0.033), which met a key secondary endpoint of compared to the study. updated external control (nominal p=0.011). Treated patients had a mean change in TFC from baseline of -0.36 -0.27 compared to a change of -0.88 -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.

AMT-130 was Ifezuntirgene inilparvovec continues to be generally well-tolerated in the Phase I/II studies, with a manageable safety profile at both doses. There have been five drug-related serious adverse events ("SAEs") reported across all cohorts, and 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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In September 2025, we completed enrollment of the first three patients in the first cohort administering AMT-260 to patients with lesions in the non-dominant hemisphere of the brain. Following a review by the independent data monitoring committee ("IDMC"), we expanded the first cohort into MTLE in the dominant hemisphere and initiated a second cohort. We completed enrollment of six patients into the first cohort in 2025. We also initiated enrollment of a second cohort in 2025.

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 (1x10 12 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 the 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 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.

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Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity. These elevations resolved as of the end of May 2026 following a course of immunosuppression as per the study protocol.

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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Six months ended June 30,

Three months ended June September 30,

Nine months ended September 30,

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As of June September 30, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $810.3 $768.4 million and $622.5 million, respectively. We had a net loss of $81.1 $61.2 million and $134.6 $195.8 million in the three and six nine months ended June September 30, 2026, compared to a net loss of $37.7 $80.5 million and $81.4 $161.9 million for the same periods in 2025. As of June September 30, 2026 and December 31, 2025, we had accumulated deficits of $1,463.5 $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.

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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 six nine months ended June September 30, 2026.

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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 a greement (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 terminate once contractually specified batches have been supplied. terminated in October 2026.

Our other expenses consist 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.

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Comparison of the three months ended June September 30, 2026 and 2025

The following table presents a comparison of our results of operations for the three months ended June September 30, 2026 and 2025:

Three months ended June September 30,

Collaboration revenues

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We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX ® sales, when earned. For the three months ended June September 30, 2026 and 2025, we recognized $5.8 $5.2 million and $5.3 $3.6 million of license revenues, respectively.

R&D expenses for the three months ended June September 30, 2026 were $34.0 $35.5 million, compared to $35.4 $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 June September 30,

Huntington's disease (AMT-130) (ifezuntirgene inilparvovec)

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Amyotrophic lateral sclerosis (AMT-162)

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Amyotrophic lateral sclerosis (AMT-162)

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Information technology costs

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Information technology costs

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Huntington's disease (AMT-130) (ifezuntirgene inilparvovec)

In the three months ended June September 30, 2026 and 2025, we incurred costs related to the development of AMT-130 ifezuntirgene inilparvovec of $6.3 $7.7 million and $10.5 $11.8 million respectively. The decrease of $4.2 $4.1 million was primarily related to lower manufacturing process validation costs, which were $0.8 $0.7 million in the current year period, compared to $4.0 $5.3 million in the prior year period. Additionally, clinical trials costs decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and Marketing Authorization Application ("MAA") submissions for AMT-130 in the United States and the United Kingdom, respectively.

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Amyotrophic Lateral Sclerosis caused by mutations in SOD1 (AMT-162)

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $2.5 million and $1.1 million, respectively. These costs were primarily related to our Phase I/II clinical trial.

In the three months ended June September 30, 2026 and June September 30, 2025, we incurred costs related to the development of AMT-260 of $4.2 $3.7 million and $2.0 $2.2 million, respectively. The current year three-month period ended September 30, 2026 included $3.7 $3.2 million of clinical expenses and $0.3 $0.5 million of CMC expenses, respectively, compared to $1.7 $2.1 million and $0.2 $0.1 million, respectively, in the prior year three-month period ended September 30, 2025.

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In the three months ended June September 30, 2026 and June September 30, 2025 , we incurred costs related to the development of AMT-191 of $ 2.2 $1.4 million and $1.3 $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.

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In the three months ended June September 30, 2026 and June September 30, 2025, we incurred costs related to research and technology projects of $1.9 $3.2 million and $0.4 $1.4 million, respectively, related primarily to our preclinical activities associated with product candidates for various other research programs and technology innovation projects.

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● We incurred $ 9.1 10.7 million in personnel and contractor-related expenses in the three months ended June September 30 , 2026 , compared to $9.5 $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 $ 2.4 3.3 million in operating and depreciation expenses related to our leased facilities in Amsterdam and Lexington, Massachusetts in the three months ended June September 30, 2026, compared to $3.9 $3.7 million in the same period in 2025;

● We incurred $1.9 $2.1 million in share-based compensation expenses in the three months ended June September 30, 2026, compared to $2.8 $0.7 million for the same period in 2025. The decrease increase of $0.9 $1.4 million was primarily driven by lower costs related to performance stock units new awards granted during the current year period; and

● We incurred a $1.3 $ 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 June September 30, 2026, compared to a $2.3 $1.8 million loss for the same period in 2025. The $1.0 $1.1 million favorable change reflects that the prior period included an adjustment was primarily attributable to changes in discount rates applied to the expected timing of achieving future milestones that did not recur remaining milestone payments in the current year period;

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and 103 more changes in the filing

Market risk 1 change

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Our market risks and exposures to such market risks during the three nine months ended June 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 .

Controls and procedures 1 change

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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 June September 30, 2026. Based on such evaluation, our CEO and CFO concluded that as of June September 30, 2026, our disclosure controls and procedures were effective to ensure that information required to be disclosed by it 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 .

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Risk factors 13 changes

In June 2026, we held a Type B meeting with the FDA. Official meeting minutes received in July 2026 confirmed that we and the FDA reached alignment that a BLA submission under the accelerated approval pathway for AMT-130, based on the existing clinical data, is reasonable. In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of a randomized standard-of-care control design instead of a sham procedure. The FDA also stated that, in accordance with the FDA's draft public guidance for accelerated approvals, the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway, and potentially fully enrolled, at the time of accelerated approval. We expect to submit a BLA in the third quarter of 2026. We can provide no assurance that we will align with the FDA on a confirmatory study design or that our BLA will be accepted for review or, if accepted, that our BLA will result in approval of AMT-130 in Huntington's disease by the FDA, or that the FDA will agree that we have met the FDA's requirements for our confirmatory study at the time of the BLA action date.

In March 2026, we held a successful pre-submission meeting with the UK MHRA, and the regulatory submission is progressing as planned for the third quarter of 2026. We can provide no assurance that any such submission will result in approval of AMT-130 in the UK.

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, The information presented below updates, and should be read in conjunction with, the as supplemented by "Item 1A. Risk Factors" disclosed in our Annual Quarterly Report on Form 10-K 10-Q for the year quarter ended December 31, 2025. Except as presented June 30, 2026 and the risk factor set forth below. There have been no material changes from to the risk factors described 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, AMT-130, ifezuntirgene inilparvovec , for the treatment of Huntington's disease. A failure of AMT-130 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, AMT-130 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 AMT-130, ifezuntirgene inilparvovec , including, but not limited to: our inability to reach agreement with the FDA on the design of a confirmatory study prior to a BLA submission; potential patient safety issues; our failure to demonstrate sufficient clinical efficacy or durability of response data to warrant further development or approval by the FDA MHRA 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 AMT-130, which are based on our interpretation of communications and interactions with regulatory authorities to date, 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 AMT-130 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 AMT-130 ifezuntirgene inilparvovec with a partner; and our ability to fund the further development and commercialization of the AMT-130 ifezuntirgene inilparvovec program.

Any one or combination of these factors could force us to halt or discontinue the ongoing clinical trials of AMT-130 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 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 even if we believe that the Accelerated Approval Program is appropriate for AMT-130, 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 AMT-130 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 AMT-130 ifezuntirgene inilparvovec could delay or prevent our ability to launch and commercialize AMT-130 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 AMT-130, 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 AMT-130 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 .

Other information 11 changes

Entry into a Material Definitive Agreement

On July 27, 2026, we and certain of our affiliates entered into Amendment No. 4 to Third Amended and Restated Loan and Security Agreement (the "Amendment") with Hercules, which amended certain terms of the Third Amended and Restated Loan and Security Agreement, dated December 15, 2021 by and among us, certain of our subsidiaries, and Hercules, as amended by that certain Amendment No. 1 to Third Amended and Restated Loan and Security Agreement, dated as of May 12, 2023, Consent and Amendment No. 2 to Third Amended and Restated Loan and Security Agreement, dated as of June 28, 2024, and Amendment No. 3 to Third Amended and Restated Loan and Security Agreement.

The Amendment, among other things, extends the period we can draw the $100.0 million term loan tranche from June 15, 2027 to September 30, 2027. Except as provided for in the amendment, the terms of borrowing under the 2026 Amended Facility otherwise remain unchanged.

The foregoing description of the Amendment does not purport to be complete and is qualified in its entirety by reference to the full text of the Amendment, a copy of which is filed as Exhibit 10.2 hereto and is incorporated herein by reference.

Adoption of 10b5-1 Trading Plans by Our Officers and Directors

During the three months ended June 30, 2026, the following adopted a " Rule 10b5-1 trading arrangement " (as defined in Item 408(a) of Regulation S-K) that is intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act and our policies on insider trading:

Aggregate Number of Ordinary Shares to be Purchased or Sold Pursuant to Trading Arrangement

David Meek , Non-executive Director

(1) Date of adoption of this Rule 10b5-1 trading arrangement is in accordance with applicable SEC rules and regulations. The first trade pursuant to this Rule 10b5-1 trading arrangement will be, in accordance with applicable SEC rules and regulations, on a date after the date of adoption of the Rule 10b5-1 trading arrangement, to the extent triggered under its terms.

(2) The Rule 10b5-1 trading arrangement is scheduled to expire on the date listed in the table, subject to earlier termination upon the sale of all shares subject to the Rule 10b5-1 trading arrangement, or as otherwise provided in the Rule 10b5-1 trading arrangement.

Other than those disclosed above, none of our directors During the three months ended September 30, 2026, no director or officers officer adopted , modified , or terminated a "Rule 10b5-1 trading arrangement" or " non-Rule 10b5-1 trading arrangement ," in each case as defined in Item 408 of Regulation S-K. during the three months ended June 30, 2026.