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EAN 2021 | Huntingtin-lowering strategies for Huntington’s disease

Anne Rosser, PhD, FRCP, Cardiff University, Cardiff, UK, outlines the future of huntingtin-lowering strategies following the decision to terminate the Phase III GENERATION HD1 study (NCT03761849) of tominersen in Huntington’s disease. The GENERATION HD1 study was stopped after an analysis of trial data by an independent data monitoring committee that found the treatment did not show a favorable benefits/risk profile. In the arm that was given this more frequent dosage, tominersen was considered unfavorable compared to placebo, and there were more adverse events in that group as well. There are various possible reasons for it, but the mechanisms responsible are not known at this point. Although the decision to stop the GENERATION HD1 trial is a setback, huntingtin-lowering approaches are still viable in Huntington’s disease. This interview took place during the European Academy of Neurology 2021 congress.