A drug that was found to have no dose-limiting side effects in nonhuman primates is being evaluated in a clinical trial for its ability to reduce production of the protein that causes Huntington’s disease (HD).
The new drug, which is an antisense oligonucleotide, was formerly known as ISIS-HTTRx and was renamed IONIS-HTTRx, because its developer changed its name in December 2015 to Ionis Pharmaceuticals from Isis Pharmaceuticals. IONIS-HTTRx acts as a gene silencer to inhibit the production of huntington protein in people with HD. The clinical trial now underway is a randomized, double-blind, placebo-controlled study aimed at evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple ascending doses of IONIS-HTTRx in up to 36 patients with early manifest HD. If the trial is successful, IONIS-HTTRx would be the first drug to modify HD progression in patients, according to an abstract to be presented at the annual meeting of the American Academy of Neurology in Vancouver in April.
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Ionis found the drug to be well tolerated in rodents and nonhuman primates in investigational new drug-enabling toxicology studies, with nonhuman primates having received doses up to 20 mg of the drug intrathecally, according to the trial’s principal investigator Dr. Blair R. Leavitt of the University of British Columbia, Vancouver, and colleagues.
“It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease. Right now we only have treatments that work on the symptoms of the disease.” Dr. Leavitt said in a written statement.
The study is supported by Ionis Pharmaceuticals and is part of Ionis’ collaboration with Roche to develop antisense drugs to treat HD, according to the investigators.