Update on tominersen clinical trials
GENERATION HD1 (NCT03761849) was a randomised, multicentre, double-blind, Phase III, placebo-controlled trial designed to evaluate the safety and efficacy of tominersen in adults with manifest Huntington’s disease. In March 2021, dosing was stopped in GENERATION HD1 following a recommendation from the independent data monitoring committee. Post hoc analyses from GENERATION HD1 suggested that tominersen at lower exposures may have potential benefit in younger adult individuals with less disease burden. This hypothesis will be investigated in the new Phase II study of tominersen, GENERATION HD2.