Roche has announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD).
The decision was based on the results of a pre-planned review of the data from the Phase III study conducted by an unblinded Independent Data Monitoring Committee (iDMC). The iDMC made its recommendation based on the investigational therapy’s potential benefit/risk profile for study participants. No new or emerging safety signals were identified for tominersen in the review of the data from this study. Roche intends to continue following participants for safety and clinical outcomes, without the dosing of the investigational medicine or placebo. Once full data from the Phase III study are available and analysed, Roche will share learnings and future plans with the HD community.
Dosing will be paused in the open-label extension study (GEN-EXTEND) of tominersen while data are carefully analysed to inform next steps on this study. The Phase I PK/PD study (GEN-PEAK) of tominersen and the observational Roche HD Natural History Study will continue.
Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development said: “This is very unfortunate news to deliver on the tominersen Phase III study and we know it will be especially difficult for people with Huntington’s disease to hear. The HD community currently has no treatments to stop or slow the progression of this rare neurodegenerative disease that impacts families across generations. GENERATION HD1 is the largest clinical trial in Huntington’s disease to date and we do know that the data generated will significantly advance our understanding of huntingtin-lowering as a potential treatment approach. We would like to thank all of the individuals and families participating in the study for their contribution, as well as the broader HD community for their commitment and collaboration.”