In essence the data indicate that there was no effect on disease progression for the participants being treated with Tominersen versus those who received placebo. This lack of effect happens even though the huntingtin levels are considerably reduced in the CSF (Cerebral Spinal Fluid).
When the dosing was stopped in March the participants had been in the trial for an average of 17 months.
In the trial there are 3 groups. 1 group received the drug every 2-month, another group got Tominersen every 4 month, and the 3rd group got placebo. From the data presented, it seems that the group that was dosed every 2-month got worse faster than the other two groups. The group that was dosed every 4 months had a disease progression quite similar to the placebo group.
Dr Schobel also told us that Roche will continue to analyse data from the study. They strongly encourage participants to stay in the study for the planned trial duration as this will provide more valuable information.