Safety, tolerability, and efficacy of RIPK1 inhibitor, SAR443820, in amyotrophic lateral sclerosis (HIMALAYA): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.

What was studied

This trial tested SAR443820, an oral drug, against a placebo in people with ALS. The drug blocks RIPK1, a protein linked to inflammation and cell death. It was a phase 2, double-blind trial at 63 sites in 13 countries. 305 adults were randomly assigned, 203 to the drug and 102 to placebo. The main goal was change in the ALSFRS-R score at week 24.

What they found

Scores fell by a similar amount in both groups. There was no significant difference between the groups. Adverse events were more common with the drug. The counts were 171 of 202 and 80 of 102. More people stopped the drug, 28 of 202. Raised liver enzymes were the most common cause. Deaths occurred in both groups, and none was blamed on the drug.

What it means for patients

The authors said SAR443820 did not show clinical benefit. It was linked to higher liver enzyme levels. They said further development of the drug in ALS is not warranted.

Why it is not certain yet

The trial was ended early. Some people were left out of the main analysis because baseline scores were missing. The blinded period lasted 24 weeks. A phase 2 trial is an early study, so it cannot settle long-term questions.

Source

Safety, tolerability, and efficacy of RIPK1 inhibitor, SAR443820, in amyotrophic lateral sclerosis (HIMALAYA): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.

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