New York, NY, December 11, 2017 – Today, the Huntington’s disease community achieved a goal that would not have been possible without the support and dedication of the families, physicians and researchers who passionately pursue a treatment for Huntington’s disease (HD). Ionis’ announcement of the completion of the Phase 1/2a study of IONIS-HTTRX is a historic moment in the fight against HD as it represents the successful completion of the first trial to treat the underlying cause of Huntington’s disease, the genetic mutation itself.
The fact that levels of mutant huntingtin were reduced in correlation to the dose of IONIS-HTTRx that was given is significant, and the fact that participants in this first Phase 1/2a study are able to continue on the drug through open label extension gives us optimism regarding its safety.
As the next phase of clinical study of IONIS-HTTRx gets underway, we look forward to continuing to partner with Roche and the many dedicated clinicians and scientists who work tirelessly to bring greater help and hope to Huntington’s families everywhere. We know families will have many questions about what happens next. We always recommend you speak with your doctor about any medical questions you may have. HDTrialFinder.org and HDSA.org will continue to provide the timeliest information regarding development of meaningful treatments for HD.