Ionis Pharmaceuticals which is a leader in antisense therapeutics asserted that the positive top-line data from a completed Phase 1/2 study of IONIS-HTTRx
in people with early stage Huntington’s disease at the 13th Annual CHDI HD conference. The data exhibits that This candidate is the first drug development to lower the disease-causing protein in people with HD.
HD is a rare, progressive, neurogenerative disease which is caused by genetic mutation in the mutation in the Huntingtin gene which results in the production of a toxic protein, the mutant huntingtin protein, which is gradually destroying neurons in the brain resulting in deteriaration in mental abilities and physical control. Ionis designed candidate which is a Generation 2+ antisense drug to specifically lessen the production of all forms of huntingtin protein including mHTT.
Dr. Sarah Tabrizi, professor of clinical neurology, director of the University College London’s Huntington’s Disease Centre and the global lead investigator on the study said “I look forward to a longer-term, larger study that can establish the benefit of reducing the toxic mutant huntingtin protein in people with HD.”