Uniqure SEC Filings

uniQure N.V. filed its Annual Report describing a broad gene therapy pipeline and major 2025–2026 developments. The lead Huntington’s disease program AMT‑130 showed favorable 36‑month high‑dose data and received FDA Breakthrough, RMAT, Orphan Drug and Fast Track designations.

However, after pre‑BLA and Type A meetings, the FDA said current Phase I/II data using external controls are unlikely to provide primary evidence for approval and strongly recommended a prospective, randomized, sham‑surgery‑controlled Phase III study. Early‑stage programs saw mixed outcomes, with promising AMT‑260 epilepsy data but safety‑driven pauses in the SOD1‑ALS trial (AMT‑162) and higher‑dose Fabry cohorts (AMT‑191).

To fund these efforts, uniQure completed two follow‑on equity offerings in 2025 raising net proceeds of about $404.2 million and entered a new $175.0 million term loan facility with Hercules, providing substantial capital but adding debt obligations through 2030.

uniQure N.V. filed its Annual Report describing a broad gene therapy pipeline and major 2025–2026 developments. The lead Huntington’s disease program AMT‑130 showed favorable 36‑month high‑dose data and received FDA Breakthrough, RMAT, Orphan Drug and Fast Track designations.

However, after pre‑BLA and Type A meetings, the FDA said current Phase I/II data using external controls are unlikely to provide primary evidence for approval and strongly recommended a prospective, randomized, sham‑surgery‑controlled Phase III study. Early‑stage programs saw mixed outcomes, with promising AMT‑260 epilepsy data but safety‑driven pauses in the SOD1‑ALS trial (AMT‑162) and higher‑dose Fabry cohorts (AMT‑191).

To fund these efforts, uniQure completed two follow‑on equity offerings in 2025 raising net proceeds of about $404.2 million and entered a new $175.0 million term loan facility with Hercules, providing substantial capital but adding debt obligations through 2030.

uniQure N.V. reported 2025 results and a key regulatory update on its Huntington’s disease program AMT-130. The company ended December 31, 2025 with $622.5 million in cash, cash equivalents and current investment securities, up from $367.5 million a year earlier, largely after raising about $404.2 million through public offerings of ordinary shares and pre-funded warrants.

Full-year 2025 revenue was $16.1 million, down from $27.1 million in 2024, mainly due to lower collaboration and contract manufacturing revenue, partly offset by higher license revenue. The net loss narrowed to $199.0 million, or $3.46 per share, compared with a $239.6 million loss, or $4.92 per share, in 2024. Shareholders’ equity improved to $198.9 million from a deficit of $6.8 million. Management expects the year-end cash position to fund operations into the second half of 2029.

On AMT-130, the U.S. FDA told uniQure that Phase I/II data using an external control are not sufficient as primary evidence of effectiveness to support a marketing application and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study. uniQure plans to seek a Type B FDA meeting in the second quarter of 2026 to discuss Phase III design. The company also highlighted progress in other gene therapy programs, including AMT-260 for refractory mesial temporal lobe epilepsy and AMT-191 for Fabry disease, and completed enrollment of the first AMT-260 Phase I/IIa cohort.

uniQure N.V. reported 2025 results and a key regulatory update on its Huntington’s disease program AMT-130. The company ended December 31, 2025 with $622.5 million in cash, cash equivalents and current investment securities, up from $367.5 million a year earlier, largely after raising about $404.2 million through public offerings of ordinary shares and pre-funded warrants.

Full-year 2025 revenue was $16.1 million, down from $27.1 million in 2024, mainly due to lower collaboration and contract manufacturing revenue, partly offset by higher license revenue. The net loss narrowed to $199.0 million, or $3.46 per share, compared with a $239.6 million loss, or $4.92 per share, in 2024. Shareholders’ equity improved to $198.9 million from a deficit of $6.8 million. Management expects the year-end cash position to fund operations into the second half of 2029.

On AMT-130, the U.S. FDA told uniQure that Phase I/II data using an external control are not sufficient as primary evidence of effectiveness to support a marketing application and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study. uniQure plans to seek a Type B FDA meeting in the second quarter of 2026 to discuss Phase III design. The company also highlighted progress in other gene therapy programs, including AMT-260 for refractory mesial temporal lobe epilepsy and AMT-191 for Fabry disease, and completed enrollment of the first AMT-260 Phase I/IIa cohort.

uniQure N.V. CEO and Managing Director Matthew C. Kapusta reported an open-market sale of 12,378 ordinary shares of the company at a weighted average price of $23.86 per share. The shares were sold automatically upon the vesting of restricted share units solely to cover estimated withholding taxes, under pre-set sale instructions, and not as a discretionary trade. After this transaction, Kapusta directly held 639,076 ordinary shares of uniQure.

uniQure N.V. Chief Financial Officer Christian Klemt reported an open-market sale of 6,217 ordinary shares of the company. The shares were sold at a weighted average price of $23.86 per share and were linked to the vesting of restricted share units.

According to the disclosure, the shares were sold solely to cover estimated withholding taxes under automatic sale instructions in the relevant restricted share unit agreement, meaning the transaction was not a discretionary trade by the executive. After this tax-related sale, Klemt still directly owns 211,513 ordinary shares.

uniQure N.V. (QURE) received an amended Schedule 13G/A showing that investment firm EcoR1 Capital, LLC, its fund EcoR1 Capital Fund Qualified, L.P., and Oleg Nodelman now report 0 ordinary shares beneficially owned, representing 0% of the class as of 12/31/2025.

All three reporting persons state they have no sole or shared voting or dispositive power over uniQure ordinary shares. They also indicate they own 5 percent or less of the class and disclaim group status and broader beneficial ownership beyond any pecuniary interest. The certifications emphasize that any securities were acquired and held in the ordinary course of business and not for the purpose of changing or influencing control of uniQure.

Avoro Capital Advisors LLC and Behzad Aghazadeh report beneficial ownership of 6,150,000 ordinary shares of uniQure N.V., equal to 9.87% of the company’s ordinary shares. This percentage is based on 62,291,663 shares outstanding as of November 6, 2025.

The shares were acquired solely for investment purposes on behalf of Avoro Life Sciences Fund LLC, with Aghazadeh acting as portfolio manager and controlling person of Avoro. The filers state the holdings are in the ordinary course of business and not for changing or influencing control of uniQure.

RTW Investments, LP and Roderick Wong, M.D. report a significant ownership stake in uniQure N.V. in this amended Schedule 13G filing. They disclose beneficial ownership of 3,711,462 ordinary shares, representing 6.0% of the class, based on 62,291,663 shares outstanding as of November 6, 2025.

The shares are held by RTW-managed funds, with RTW Investments and Dr. Wong sharing voting and dispositive power over the same 3,711,462 shares. They certify the position is held in the ordinary course of business and not for the purpose of changing or influencing control of uniQure.