IONIS-HTTRX successfully lowers the toxic mutant huntingtin protein (mHTT) in people with Huntington disease (HD), and it does so safely, according to a press release issued by Ionis Pharmaceuticals, Inc. The drug will be licensed by Roche following the completion of its Phase 1/2a randomized, placebo-controlled, dose escalation study. IONIS-HTTRX is the ﬁrst therapy in clinical development designed to target the underlying cause of HD by reducing the production of mHTT.
“The results of this trial are of ground-breaking importance for Huntington disease patients and families,” said Dr. Sarah Tabrizi, professor of clinical neurology, director of the University College London’s Huntington Centre, and the global lead investigator on the Phase 1/2a study, in the press release. “For the ﬁrst time, a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated. The key now is to move quickly to a larger trial to test whether IONIS-HTTRX slows disease progression.”
Ionis and Roche plan to present results from this study at medical conferences in the ﬁrst half of 2018 and to submit the study results for publication in a peer-reviewed medical journal. In addition, Ionis and Roche recently have initiated an open-label extension (OLE) study for patients who completed the Phase 1/2a study.
An article by HD Buzz, calls IONIS-HTTRX “one of the biggest breakthroughs in Huntington’s disease since the discovery of the HD gene in 1993.” The article notes that “The success of this ﬁrst trial sets the stage for a larger study in hundreds of HD patients, as soon as possible…Until we conduct the next trial, we won’t know if this reduces the impact of HD.”