More info on the trial from their website:
Phase I/II Clinical Trial of AMT-130
uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington’s disease. Based on interactions with the FDA, it was agreed that data from the Phase I/II studies could be compared to a propensity score-matched external control derived from the Enroll-HD natural history data set, under a prespecified statistical analysis plan, which may serve as the primary basis for a BLA submission.
In the U.S. study, a total of 26 patients with early manifest Huntington’s disease were randomized to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) procedure (n=10). Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment. The European open-label Phase 1b/2 study of AMT-130 enrolled 13 patients with early manifest Huntington’s disease (n=6 low dose; n=7 high dose).
Clinical Results
In September 2025
we announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. For patients receiving the high-dose, the data included (with a data cutoff as of June 30, 2025):
- A statistically significant 75% slowing of disease progression as measured by cUHDRS (p=0.003), which met the primary endpoint of the study. Treated patients had a mean change in cUHDRS from baseline of ‑0.38 compared to a change of ‑1.52 for patients in the propensity score-matched external control.
- A statistically significant 60% slowing of disease progression as measured by TFC (p=0.033), which met a key secondary endpoint of the study. Treated patients had a mean change in TFC from baseline of ‑0.36 compared to a change of ‑0.88 for patients in the propensity score-matched external control.
- Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modality Test (SDMT), Stroop Word Reading Test (SWRT) and Total Motor Score (TMS).
- An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of ‑0.44 compared to a change of ‑3.73 for patients in the propensity score-matched external control.
- A 113% slowing of disease progression as measured by SWRT (nominal p=0.002), with a mean change in SWRT from baseline of 0.88 compared to a change of ‑6.98 for patients in the propensity score-matched external control.
- A 59% slowing of disease progression as measured by TMS (nominal p=0.174), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control.
- A mean reduction from baseline in cerebrospinal neurofilament light protein (CSF NfL) of ‑8.2%. CSF NfL is a well-characterized, supportive biomarker of neurodegeneration that has been shown to be strongly associated with the clinical severity of Huntington’s disease.
AMT-130 was generally well-tolerated, with a manageable safety profile at both doses. As of June 30, 2025, no new drug-related serious adverse events have been observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure, which all resolved.
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