UniQure announces positive interim data in slowing Huntington's disease progression 

This morning, uniQure released a press statement presenting interim data from patients participating in the ongoing Phase I/II clinical trials of AMT-130, [HD Gene TRX-1 and HD Gene TRX2], for Huntington’s disease treatment in the United States and Europe. The analysis includes follow-up periods of up to 24 months and also introduces an external control group. The major updates are as follows:

  • A significant, dose-dependent, slowing in disease progression measured by cUHDRS was observed through 24 months in patients receiving the high dose of AMT-130.
  • Trends in measurements of motor and cognitive function showed near-baseline stability throughout the 24 months of follow-up in patients receiving the high dose of AMT-130.
  • A statistically significant reduction of NfL in cerebral spinal fluid (CSF) was observed in patients treated with AMT-130.
    Based on data observed to date, AMT-130 remains generally well-tolerated, with a manageable safety profile at both doses. There were no new AMT-130-related serious adverse events reported.

 AMT-130 is an experimental huntingtin-lowering gene therapy. 

Walid Abi-Saab, M.D., chief medical officer of uniQure stated,

We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months.

Read the press release