SAR443820

What does the drug do?

SAR443820 works by suppressing an enzyme known as RIPK1 (receptor-interacting protein kinase 1). The over-activation of this enzyme is associated with abnormal microglia activity and nerve cell death. Microglia are immune cells involved in the central nervous system, brain and spinal cord. RIPK1 levels are known to be significantly increased in the spinal cord of people living with MND. It is hoped that suppressing RIPK1 will help to ease disease progression.

Phase 2 (HIMALAYA)

The Phase 2 clinical trial will look at the safety and efficacy of taking SAR443820. The trial will have two phases, the first being a 24-week long double blind, placebo controlled trial, where participants will be randomised in a 2:1 ratio of treatment:placebo. At the end of the 24-week period, the participants will able to enter the second phase which is open-label. This means that everyone in the trial will be given the treatment up to week 106. The trial will recruit 261 participants across the US, Germany, the Netherlands, Belgium, France and the UK. You can find out more about the trial here.

Last updated: 31/07/2023