Verubecestat is an investigational small molecule inhibitor of the beta-site amyloid precursor protein cleaving enzyme 1 (BACE1) that is thought to work by reducing the build-up of toxic amyloid plaques in the brain.
Merck’s decision follows a recommendation from the external Data Monitoring Committee after assessing the overall risks and benefits of the drug during a recent interim safety analysis. The Data Monitoring Committee determined that there was “virtually no chance of finding a positive clinical effect,” but noted that there were no safety concerns. A second trial of Verubecestat in people with prodromal (or pre-symptomatic ) Alzheimer’s disease, called APECS, will continue and results are expected in February 2019.
Dr Doug Brown, Director of Research at Alzheimer’s Society, said:
‘It’s encouraging that there are no concerns over the safety of this drug and that it will continue to be tested in people in the pre-symptomatic stages of disease, where there is more chance that it will prevent the build-up of toxic amyloid plaques.
‘However, we are disappointed that another trial has been stopped because the drug is failing to bring benefits for people with Alzheimer’s. It reiterates the importance of people receiving a dementia diagnosis as early as possible so that drugs have the best possible chance to stop or slow down damage to the brain.
‘Deaths from dementia are rising year on year, with one person developing it every three minutes and one million expected to have the condition by 2021. More than ever, we need to redouble our efforts to meet the G7’s ambition of finding a disease-modifying treatment for dementia by 2025. As we continue to hunt for a cure and new treatments, we must concentrate on finding the best ways to care for people living with dementia now.’