The final stage, phase III trial, called MINDSET, began in 2015 after the drug showed promising signs in a smaller phase II trial. However, the headline result of this final stage trial showed that the drug did not lead to a benefit in memory and thinking skills or improve the ability for people to cope with activities of daily life.
Like existing treatments for Alzheimer’s disease, RVT-101 was designed to be a symptomatic drug, treating the symptoms of Alzheimer’s disease by boosting levels of chemical messengers in the brain.
The unsuccessful trial involved 1,315 people with mild-to-moderate Alzheimer’s disease who received either a daily pill containing 35mg of RVT-101 or a placebo pill, alongside the current Alzheimer’s treatment, donepezil. After 24 weeks of treatment, people who took the daily dose of RVT-101 did not show signs of a benefit compared to people who took donepezil alone.
As a result of the findings, Axovant will not be submitting the drug for regulatory approval for the treatment of mild-to-moderate Alzheimer’s. However, the drug is currently involved in a separate trial as a treatment for dementia with Lewy bodies, a form of dementia that affects around 100,000 people in the UK. The results of that study are set to come out next year.
Dr David Reynolds, Chief Scientific Officer at Alzheimer’s Research UK, said:
“There are around half a million people in the UK living with Alzheimer’s and many more working tirelessly to care for loved ones with the disease. This news is clearly a set-back for those affected by Alzheimer’s, and it highlights the ongoing importance of drug discovery and the challenges we face in our fight against dementia.”
“RVT-101 was designed to compensate for the processes that cause Alzheimer’s, rather than tackle the underlying causes of the disease. While existing Alzheimer’s treatments show that symptomatic drugs can work, this drug was not able to produce benefits over and above current treatment options. Only headline results were published today, so we will need to wait to see the full data to understand why RVT-101 did not show benefits and how researchers can learn from the findings.
“It’s vital we continue to discover more about the biological processes underlying Alzheimer’s to identify new opportunities for treatments that can affect these processes and transform people’s lives. Alzheimer’s Research UK is committed to bringing about new ways to help people affected by dementia by supporting millions of pounds of research taking place in laboratories around the country.
“The 15-year wait for a new Alzheimer’s drug does not end today. In recent years science has made huge leaps forward in tackling diseases like cancer and HIV/AIDS, and despite set-backs like this, we will be able to tackle diseases like Alzheimer’s if we continue to invest in research.“