Following announcements earlier this week, Roche has presented further information on their planned Phase II trial of tominersen, and the findings from the Phase III GENERATION-HD1 study which have led to the development of this new trial. The webinar was hosted by the European Huntington’s Disease Network on Thursday, January 20, 2022. The following are key takeaways from the HDYO research team.

When dosing of tominersen stopped in March 2021 due to its benefit/risk profile, data analysis was undertaken to determine if there was a subgroup of participants who responded positively to treatment. In the GENERATION-HD1 study, participants were assigned to one of three groups – placebo, treatment with tominersen every 8 weeks or treatment with tominersen every 16 weeks. It was found that in participants treated with tominersen less frequently (every 16 weeks), response was associated with the participant’s age, disease burden and drug exposure.

To investigate this further, the data from participants were split into subgroups for analysis, depending on their age and disease burden. Results from a subgroup of younger participants (age <48) with less disease burden (CAP* <500) were found to warrant further investigation. Consequently, Roche has decided to continue the development program with a new Phase II clinical. This trial, currently in the early planning stages, will explore the safety and efficacy of different doses of tominersen in a younger adult patient population with less disease burden. More details of this new trial are expected to be shared at future scientific conferences.

• CAP - a research tool calculated using a person’s age and CAG repeat number