June 21, 2023 10:56am

Having unveiled mixed results for its study of a Huntington's disease treatment  

A group of patients who received a low dose of the drug, known as AMT-130, showed ONLY functional improvements after two years. The high-dose group showed similar improvements a year following treatment.

Biomarker results were mixed as the high-dose group experienced increases in troublesome proteins a year following treatment. Six patients received the low dose of the Huntington's disease treatment, while 10 received the high dose.

On average, patients in both groups showed improvements in motor skills, functional capacity and total symptoms compared to the expected natural course of Huntington's disease.


The Bottom Line: The low-dose group saw an average decrease of 12.9% after two years. But the high-dose group experienced a 51.5% increase after a year. Those levels seemingly backed off over time, however.

Patients in the low-dose group experienced an 8.1% decrease in this protein after two years. But the high-dose group had a 39.7% increase compared to a jump of 4.7% for the placebo group.  <source: Allison Gatlin, IBD>

QURE HAD hopes its gene therapy will be a one-time treatment for Huntington's, a progressive disease that causes nerve cells in the brain to break down and die.