uniQure N.V.

Previous wording

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.

Current wording

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.

Previous wording

We are currently conducting Phase I/II clinical trials of AMT-130 in the U.S. and Europe. We completed the enrollment of all 26 patients in the first two cohorts of our U.S. study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023. In 2025, we completed enrollment of all 12 patients in the third cohort, and we treated six patients with the high-dose of AMT-130 in a fourth cohort to evaluate the safety and efficacy of AMT-130 in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.

Current wording

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.

Previous wording

In September 2025, we announced positive topline 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. We analyzed clinical outcomes for 29 patients treated with AMT-130 (n=17 high-dose; n=12 low-dose) of which 12 patients per dose group had attained 36 months of follow-up and were evaluated at that time point. Outcomes for each dose group were compared to a propensity score-matched external control drawn from the Enroll-HD natural history data set (n=940 for high-dose; n=626 for low-dose).

Current wording

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.

Previous wording

Topline 36-month efficacy results for patients receiving high-dose AMT-130 were as follows (data cutoff as of June 30, 2025):

Current wording

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):

Previous wording

AMT-130 was 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, andthe most common adverse events in the treatment groups were related to the administration procedure.

Current wording

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.

Previous wording

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 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 cohort were classified as mild or moderate in severity, with the most common adverse event being headache (N=2). No immunosuppression was required.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

As of June 30, 2026 and December 31, 2025, we had cash and cash equivalents and investment securities of $810.3 million and $622.5 million, respectively. We had a net loss of $81.1 million and $134.6 million in the three and six months ended June 30, 2026, compared to a net loss of$37.7 million and $81.4 million for the same periods in 2025. As of June 30, 2026 and December 31, 2025, we had accumulated deficits of $1,463.5 million and $1,328.9 million, respectively. See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above.

Current wording

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.

Previous wording

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 months ended June 30, 2026.

Current wording

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.

Previous wording

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, includinga 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 terminate once contractually specified batches have been supplied.

Current wording

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, includinga 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.

Previous wording

Our other expenses consist 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.

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

In the three months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $6.3 million and $10.5 million respectively. The decrease of $4.2 million was primarily related to lower manufacturing process validation costs, which were $0.8 million in the current year period, compared to $4.0 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.

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $2.2 million and $1.3 million, respectively. These costs were primarily related to our Phase I/II trial.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

In the three months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $1.9 million and $0.4 million, respectively.

Current wording

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.

Previous wording

We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $5.1 million in interest income in the three months ended June 30, 2026, compared to $3.5 million in the same period in 2025. The $1.5 million increase was primarily due to higher average balances of our investment securities held during the current year period, compared to the prior year period.

Current wording

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.

Previous wording

We recognized a net foreign currency loss of $1.7 million during the three months ended June 30, 2026, compared to a net gain of $18.6 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.

Current wording

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.

Previous wording

In connection with our September 2025 follow-on public offering, we issued pre-funded warrants that are classified as a liability and measured at fair value each reporting period. We recognized a $16.0 million loss in the three months ended June 30, 2026, reflecting an increase in the fair value of this liability, compared to nil for the same period in 2025.

Current wording

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.

Previous wording

We recognized $1.8 million of deferred tax expense in the three months ended June 30, 2026, and $0.4 million of deferred tax expense for the same period in 2025.

Current wording

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.

Previous wording

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

Current wording

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

Previous wording

We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned. For the six months ended June 30, 2026 and 2025, we recognized $9.4 million and $6.8 million of license revenues, respectively.

Current wording

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.

Previous wording

R&D expenses for the six months ended June 30, 2026 were $63.1 million, compared to $71.5 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.

Current wording

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.

Previous wording

In the six months ended June 30, 2026 and 2025, we incurred costs related to the development of AMT-130 of $14.3 million and $18.7 million respectively. The decrease of $4.5 million was primarily driven by lower costs related to clinical trials of $9.0 million in the current period, compared to $11.2 million in the prior year period. Additionally, manufacturing process validation costs were decreased in the current year period, partially offset by higher regulatory costs associated with the preparation of BLA and MAA submissions for AMT-130 in the United States and the United Kingdom, respectively.

Current wording

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.

Previous wording

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-260 of $7.0 million and $4.0 million, respectively. The current period included costs of $6.3 million related to clinical trials and $0.5 million related to CMC development, compared to $3.3 million and $0.7 million, respectively, in the prior year period.

Current wording

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.

Previous wording

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-191 of $3.7 million and $3.4 million, respectively. These costs primarily related to the Phase I/II trial.

Current wording

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.

Previous wording

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to the development of AMT-162 of $3.4 million and $2.9 million, respectively. These costs primarily related to the Phase I/II trial.

Current wording

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.

Previous wording

In the six months ended June 30, 2026 and June 30, 2025, we incurred costs related to research and technology projects of $3.0 million and $1.7 million, respectively.

Current wording

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.

Previous wording

Our other non-operating items, net, for the six months ended June 30, 2026 and June 30, 2025 were as follows:

Current wording

Our other non-operating items, net, for the nine months ended September 30, 2026 and September 30, 2025 were as follows:

Previous wording

We recognize interest income associated with our cash and cash equivalents and investment securities. We recognized $10.3 million in interest income in the six months ended June 30, 2026, compared to $7.7 million in the same period in 2025. The $2.6 million increase was primarily due to higher average balances of investment securities held during the current year period, compared to the prior year period.

Current wording

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.

Previous wording

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 $13.0 million and $13.8 million in the three months ended June 30, 2026 and 2025, respectively.

Current wording

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.

Previous wording

We recognized interest expense related to the Hercules loan facility of $2.8 million in the six months ended June 30, 2026, compared to $3.6 million in the same period in 2025. The $0.8 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.

Current wording

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.

Previous wording

We recognized a net foreign currency loss of $4.0 million in the six months ended June 30, 2026, compared to a net gain of $25.8 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.

Current wording

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.

Previous wording

As of June 30, 2026, we had cash and cash equivalents, restricted cash and investment securities of $811.9 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 AMT-130, ongoing clinical trials of AMT-130, 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.

Current wording

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.

Previous wording

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 AMT-130 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.

Current wording

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.

Previous wording

As of June 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 AMT-130 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 $19.5 million, with $5.4 million payable within the next 12 months.

Current wording

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.

Previous wording

We have entered into lease arrangements for facilities, including corporate, laboratories and office space. As of June 30, 2026, we had fixed lease payment obligations of $22.9 million, with $5.2 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 June 30, 2026, we remain obligated under a guarantee of lease payments of $14.0 million, with the maximum potential exposure under the guarantee decreasing over the remaining lease term through May 2029.

Current wording

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.