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.
The Phase 2 (HIMALAYA) clinical trial was investigating SAR443820 over a 24-week period. Denali Therapeutics reported that the trial did not meet its primary endpoint of change in ALSFRS-R, suggesting there was no significant change in ALSFRS-R (from baseline to 24-weeks) for those who received the treatment compared to those who received the placebo (dummy drug). Further details on the trial outcome are expected in the coming months.
Previous Clinical Trials
Phase 2 (HIMALAYA)
The Phase 2 clinical trial looked at the safety and efficacy of taking SAR443820. The trial had two phases, the first being a 24-week long double blind, placebo controlled trial, where participants were randomised in a 2:1 ratio of treatment:placebo. At the end of the 24-week period, the participants were able to enter the second phase which is open-label. This allows everyone in the trial will be given the treatment up to week 106. The trial aimed to recruit 261 participants across the US, Germany, the Netherlands, Belgium, France and the UK. You can find out more about the trial here.
February 2024 - Denali Therapeutics reported that SAR443820 did not meet its primary endpoint of change in the ALSFRS-R. Further details about the outcome of the trial are expected in the coming months.
July 2023 - The trial closed to recruitment.
May 2022 - The trial began recruiting. You can find out more here.
Last updated: 20/02/2024