Phase I/II Huntington’s Data From uniQure Draw Caution On Biomarker

Updated data from uniQure N.V.’s Phase I/II trial of the gene therapy AMT-130 in Huntington’s disease has shown further evidence of improvement on functional and clinical endpoints and the biomarker of neurofilament light chain (NfL) in the cerebral spinal fluid (CSF), but continued inconclusive results on another biomarker, mutant Huntingtin protein (mHTT), which drew caution from some analysts about durability. Still, the company suggested the measurement of mHTT in the CSF was not representative of the protein’s pharmacodynamics in the brain, citing assay limitations and the way AMT-130 is administered.

The biotech announced the updated data from 39 patients in the study on 19 December, stating that patients treated with AMT-130 had continued to show evidence of preserved neurological function as well as potential