Topline results announced

On 20th February 2024 Denali Therapeutics reported that the phase 2 clinical trial of SAR443820 (HIMALAYA) did not meet its primary endpoint. This suggests that 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. You can read more about SAR443820 here.

A Phase 2 Multicentre, Randomized, Double-blind, Placebo-controlled, 3 arm Study to Evaluate the Efficacy, Safety and Tolerability of SAR443820 in Adult Patients with Amyotrophic Lateral Sclerosis Followed by a Long-term Safety Extension 

UK Chief Investigator

Dr Tom Lambert

Research summary

SAR443820 is an investigational drug that is being developed to slow down the disease progression of ALS. SAR443820 is a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor. RIPK1 is protein found within cells, involved in the regulation of inflammation, cytokine release, and cell death. Both preclinical and clinical studies have shown that RIPK1 is a key mediator of programmed cell death and inflammatory pathways in ALS. SAR443820, an inhibitor of RIPK1 that can penetrate the central nervous system, has the potential to modify the course of neurodegenerative processes and slow disease progression in patients with ALS. The purpose of this study is to see if the drug SAR443820 works to improve the symptoms of adults with ALS compared to a group receiving placebo (a dummy substance), with both groups continuing to receive their current treatment for ALS. Additional purposes of the study are to find out the pharmacokinetics (PK), and pharmacodynamics (PD) of SAR443820 and how safe and tolerable the drug is.

Inclusion / exclusion criteria

Key inclusion criteria:

Key exclusion criteria:

Current status

Study completed


Information about study sites

Contact details

Stoke: Katrina Parkinson research@uhnm.nhs.uk

Plymouth: jonanderson2@nhs.net

Recruitment group(s)

Patients with MND

Recruitment target(s)

Up to 261 participants globally (8 participants in the UK)

Key dates

Recruitment open date: 18 May 2022

Recruitment end date: 14 June 2023 (earlier than planned - global recruitment target reached)





Study design

Interventional, randomised controlled trial

Intervention (if applicable)

Phase (if applicable)


Outcome measures

Primary outcome measures

Secondary outcome measures

Publications / Results reports

Links will be provided when papers are published.