On Monday 22 March 2021 Roche announced that dosing is being permanently discontinued in the GENERATION HD1 phase III trial evaluating the effect of tominersen, an ASO-based huntingtin lowering drug on the clinical course of HD. Dosing is also being paused on the open label extension study, GEN-EXTEND. This decision is the outcome of a pre-planned review of the risk/benefit profile of the intervention in study participants. Roche emphasized that there were no new safety signals that led to this decision.
While EHDN shares the heartbreak and disappointment that this sobering news brings to the global HD community, it is important to remind ourselves that we must not be discouraged by this setback and that in fact a lot has already been achieved through this ground-breaking trial. The GENERATION HD1 trial is a testament to the potential that can be mobilized when patients, physicians, researchers and industry cooperate efficiently. The trial was realized in record time and against unprecedented, pandemic odds. The thorough analysis of the wealth of novel data collected through GENERATION HD1 will be critical in directly informing other upcoming trials.
It is important to note that while the dosing on these two studies is stopped or on hold the studies continue so we strongly recommend that all participants remain in close contact with their sites for further guidance on what happens next.
Roche update on tominersen programme in manifest Huntington’s disease: Press Release