Great news for the HD community towards the end of 2025

This week Roche announced that recruitment has started for a new trial called POINT-HD.  40 participants will be included in this Phase I trial which means that the main objective is to test if the drug is safe and tolerated.  Nevertheless, the trial will be ongoing for two years or more, and efficacy will be of course also be measured.  

The trial will be done in multiple clinics and countries, and New Zealand and Australia are the first ones to start recruiting.  Argentina will soon follow.  Additional countries and clinics will be added when the respective regulatory and practical issues are solved until the total number of participants is reached.  Roche can not give us answer to whether clinics in Europe will be involved.  It’s a small study and the most important is to have the total number of participants recruited and starting as soon as possible.  

People may wonder why Roche is starting a new trial when they already have the Generation HD2 trial ongoing.  It is clear to us that the company is investing a lot in HD and  want to explore additional approaches for treatment.  HD will most probably not be stopped or cured with only one therapy.  Multiple approaches is needed to combat a complex disease like HD. 

The drug that will be tested in POINT-HD is called RG6496 (RG representing Roche Genentech). RG6496 is designed to pinpoint a genetic variation on the faulty huntingtin gene copy and to leave the healthy more or less unaffected.  This is similar to what Wave has done in their trial PRECISION-HD2 but with a different genetic variation.   It is assumed that around 40% of the HD gene carriers has the genetic variation targeted by RG6496.  

It is very good news that the trial is moving forward and start recruiting and we hope that the HD community in New Zealand, Australia and Argentina find this to be a good opportunity to support research and help us find effective therapies as soon as possible.  

You can read more about the trial here in the community letter shared by Roche. 

– Article written by Astri Arnesen

FIND MORE INFORMATION HERE:

  • Read the community letter here.