A new dawn for Tominersen

Some mornings are better than others and Tuesday January 18th was definitely one of the really good ones as I woke up to a beautiful sunrise and the news that Roche has decided to do another Phase II trial with Tominersen. 

The decision is based upon the analyses made from data collected in the Generation HD 1 trial.  As most of you may remember this trial stopped dosing with Tominersen in April 2021 because the preliminary results didn’t show any effect. Actually, some of the participants who received Tominersen had a more rapid worsening of symptoms than the ones on placebo and this was the reason to stop dosing.

 

Thorough digging into all the information that has been collected from all the 800 trial participants have shown that there are subgroups that seems to benefit from being treated with Tominersen.  This is exactly one of the possible perspectives that was outlined in the Roche webinar in April 2021

The planned Phase II trial will including younger people with less disease burden ( earlier in the disease progression) than in Generation HD1.  The dosing will also be less frequent.  It’s important to point out that there are still a lot of uncertainty to whether Tominersen can be helpful or not, but nevertheless, the data indicates that there may be an effect and this should be explored in a new trial.  For more information read the Community letter from Roche

 

We should use this opportunity to say a big, big THANK YOU to all who participated in the Roche trials and other previous and ongoing trials.  We owe you so much for providing a lot of valuable data and thus knowledge about HD progression and potential treatments.  I wish I could give you all a warm and sincere hug and a gold medal.   

If you want to learn more about the details in the data findings from Generation HD1 you can attend the EHDN webinar (aimed towards scientists and clinicians) 

or if you prefer things presented in lay terms join the EHA webinar

 

– An article by Astri Arnesen.