This week, Ionis Pharmaceuticals shared data from the successful safety trial for their huntingtin-lowering drug. The data confirms what was announced in December 2017: the drug safely lowers mutant huntingtin. No one taking the drug had a serious side effect, and no one dropped out of the trial – these are great signs of a safe and promising therapy. The next step is a larger trial that will be carried out by Roche Pharmaceuticals to see whether the drug has any effect on symptoms. There has not yet been an announcement about when and where this will take place, or who is eligible, but HDSA will continue to share the news as soon as updates are available.
For more info, check out HDSA’s responses to Frequently Asked Questions about the trial.
The full results from the Phase 1b/2a trial were presented at the annual CHDI Huntington’s disease therapeutics conference in Palm Springs and also during a live webcast on the Ionis webpage. The materials were directed at scientists and investors, but HDSA shared a community statement from Ionis and Roche when the results were announced. If you are interested in seeing the data slides from these presentations, pdf copies are available for a limited time on the Ionis website.
A great narrative article about Huntington’s disease, the past and present of HD research, and the Ionis/Roche trial was published this week in Mosaic. Gene Veritas wrote an informative blog post about the results at the CHDI conference.