uniQure reports 48-month AMT-130 data for Huntington's disease gene therapy
The company presented updated Phase I/II results and safety findings on September 29, 2026, ahead of planned BLA and MAA submissions.
uniQure N.V. disclosed in an 8-K filed September 29, 2026 that it had presented a program update on AMT-130 (ifezuntirgene inilparvovec), its gene therapy for Huntington's disease, and issued an accompanying press release. On September 29, 2026, uniQure presented an update of its ongoing clinical trials of AMT-130 and issued a press release summarizing the presentation.1
The materials describe a 36-month statistical analysis, based on a data cut through June 30, 2025, intended as part of the basis for BLA and MAA submissions. In that analysis, high-dose AMT-130 showed a statistically significant 60% slowing based on TFC at 36 months (p=0.033) and a statistically significant 75% slowing based on cUHDRS at 36 months (p=0.003)1, measured against a propensity-matched Enroll-HD external control.
A separate 48-month primary analysis, using data as of June 30, 2026 and an updated Enroll-HD B external control of 1,337 participants, found a 44% slowing of disease progression at 48 months on cUHDRS (p=0.144, nominal) and a 61% slowing on TFC (p=0.008, nominal)1. The company said effects on secondary measures including symbol digit modalities, Stroop word reading, and total motor score were also assessed against the external control1.
On neurofilament light chain, a marker of neurodegeneration, uniQure said CSF NfL levels stayed near baseline starting at Month 18 through four years, with mean CSF NfL 4% above baseline in the high-dose group and 8% below baseline in the low-dose group at Month 481, compared with an untreated annual rise cited from external literature.
On safety, the company said no new treatment-related serious adverse events had been reported in Cohorts 1 and 2 since September 2025, while a Cohort 4 patient experienced a treatment-related serious adverse event of CNS inflammation that fully resolved after a short course of corticosteroids, and one low-dose patient died by suicide roughly five years after treatment, assessed as unrelated to treatment1. Overall, the company said AMT-130 continues to be generally well-tolerated1.
Written by readthrough’s AI from the linked primary sources and fact-checked against them automatically before publishing. Not investment advice.