Post by UCL News

A global clinical trial for a new Huntington’s disease treatment has posted positive results today, announced by trial sponsor uniQure and UCL scientists. The researchers found that patients receiving the treatment experienced 75% less progression of the disease overall, compared to a matched cohort of people with Huntington’s who were not receiving the treatment. This is the first time a drug trial has reported continuing, statistically significant slowing of Huntington’s progression. uniQure plans to submit an application to the US Food and Drug Administration early next year requesting accelerated approval to market the drug, with applications in the UK and Europe to follow. Professor Sarah Tabrizi, MD PhD FMedSci FRS from UCL Faculty of Brain Sciences, lead scientific advisor on the trial, said: "l am thrilled that this study of AMT-130 showed statistically significant effects on disease progression at 36 months. These groundbreaking data are the most convincing evidence in the field to date and underscore the disease-modifying effect in Huntington's disease, where an urgent need persists. For patients, AMT-130 has the potential to preserve daily function, keep them in work longer, and meaningfully slow disease progression.” Professor Edward Wild, principal investigator of the UCL Huntington’s Disease Centre trial site at UCL and University College London Hospitals NHS Foundation Trust, said: “This result changes everything. On the basis of these results it seems likely AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff." Watch Professor Tabrizi featured on BBC News at One, and read more here: https://lnkd.in/eKSypsmD

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