LEXINGTON, Mass. and AMSTERDAM — November 10, 2025 — Leads & Copy — Gene therapy company uniQure N.V. (NASDAQ: QURE) announced its third-quarter 2025 financial results and recent business updates, including a key shift in FDA feedback regarding its Huntington’s disease treatment, AMT-130.
According to uniQure, preliminary feedback from the FDA at a pre-Biologics License Application (BLA) meeting indicated a shift from previous regulatory communications. The company said it plans to interact with the FDA urgently to determine the next steps for AMT-130.
In a statement, uniQure CEO Matt Kapusta said the third quarter was a defining moment, as the company presented topline three-year data of AMT-130, demonstrating a statistically significant slowing of disease progression. He added that while recent FDA feedback was unexpected and has introduced uncertainty into the timing of its BLA submission, the company strongly believes that AMT-130 has the potential to bring substantial benefit for patients with Huntington’s disease, and that uniQure is fully committed to working with the FDA to determine the most expeditious path forward to rapidly bring AMT-130 to patients and their families in the U.S.
In September, uniQure announced positive topline data from the Phase I/II study for AMT-130 in treating Huntington’s disease. The results include a statistically significant 75% slowing in disease progression measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), meeting the primary endpoint compared to a propensity score-matched external control (p=.003). Additionally, a key secondary endpoint of Total Functional Capacity (TFC) demonstrated a statistically significant 60% slowing of disease progression compared to a propensity score-matched external control (p=0.033).
The data also showed a mean reduction from baseline in cerebrospinal neurofilament light protein (NfL) of -8.2% at 36 months in the high-dose group. The company also reported that AMT-130 was generally well-tolerated in the Phase I/II studies with a manageable safety profile across both doses, with no new drug-related serious adverse events observed since December 2022.
Other company highlights include:
- Advanced enrollment of the Phase I/IIa study of AMT-260 in mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026.
- Initial data from the Phase I/IIa study of AMT-191 in Fabry disease showed sustained increases in α-gal enzyme activity. Additional clinical data is expected in the first half of 2026.
- Approximately $323.7 million was raised in net proceeds from an upsized public follow-on offering, resulting in cash, cash equivalents, and current investment securities of $694.2 million as of September 30, 2025.
In September 2025, following an IDMC review of preliminary safety and efficacy data, the Company voluntarily paused enrollment in the AMT-162 study as a dose limiting toxicity, which resulted in a serious adverse event determined to be related to AMT-162, was observed in one patient in the second cohort. The Company will continue to collect and evaluate data from the five patients treated in the Phase I/II EPISOD1 study.
As of September 30, 2025, uniQure held cash, cash equivalents, and current investment securities of $694.2 million, compared to $367.5 million as of December 31, 2024. The company expects that its cash, cash equivalents and investment securities will be sufficient to fund operations into 2029.
Chiara Russo
Direct: 781-491-4371
Mobile: 617-306-9137
c.russo@uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile:339-223-8541
t.malone@uniQure.com
Source: uniQure
