AMT-162

Nov. 10, 2025: uniQure N.V. (NASDAQ: QURE) reported Q3 2025 results and provided pipeline/regulatory updates led by AMT-130 in Huntington’s disease. The company reiterated September 2025 topline 36‑month Phase I/II “pivotal” data (cutoff June 30, 2025) showing statistically significant slowing vs propensity score–matched external control: 75% slowing on cUHDRS (primary endpoint; p=0.003) and 60% slowing on TFC (key secondary; p=0.033). CSF NfL mean change was -8.2% at 36 months; safety was generally well-tolerated with no new drug-related SAEs since Dec. 2022. Data were presented at the 2025 Huntington’s Disease Clinical Research Congress (Oct. 10–13, 2025). A fourth high-dose cohort (6 patients with lower striatal volumes) was initiated and fully recruited in Oct. 2025; dosing expected to complete before year-end 2025.

Regulatory: At an Oct. 2025 pre-BLA FDA meeting, uniQure said FDA feedback suggested the agency may no longer agree that the Phase I/II external-control comparison is adequate as primary evidence for a BLA; BLA timing is unclear. Final minutes are expected within 30 days; uniQure plans urgent FDA interactions to define a path toward accelerated approval.

Other programs: AMT-260 (refractory MTLE) advanced after IDMC review; updated data expected 1H 2026. AMT-191 (Fabry) initial data in 4 patients showed 27–208x α‑Gal A activity vs mean normal range; stable lyso‑Gb3 and ERT withdrawal; updated results expected 1H 2026. AMT-162 (SOD1 ALS) enrollment was voluntarily paused after an IDMC-cited dose-limiting toxicity/related SAE in one patient.

Financing: Raised ~$323.7M net in an upsized follow-on; cash/investments $694.2M as of Sept. 30, 2025; runway into 2029. Debt refinanced with potential additional tranches tied to an AMT-130 regulatory milestone.