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InternationalResearch and clinical trials

Experimental oral RIPK1 inhibitor showed no benefit in ALS in a phase 2 trial

Peer-reviewed publication

A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.

The HIMALAYA study, published in The Lancet Neurology, examined an experimental drug in patients with ALS (amyotrophic lateral sclerosis). The drug is a RIPK1 inhibitor taken by mouth. A total of 305 patients in 13 countries took part. The decline in the function score at 24 weeks was similar in the two groups. Raised liver enzymes were seen more often in those taking the drug.

What does it mean for patients and families?

The researchers do not recommend developing this drug for ALS. Negative results also protect patients from ineffective treatment.

Source

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

Scientific paper: Safety, tolerability, and efficacy of RIPK1 inhibitor, SAR443820, in amyotrophic lateral sclerosis (HIMALAYA): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. The Lancet Neurology, 2026. DOI: 10.1016/S1474-4422(26)00277-2 · PubMed 42716044

The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).

This page is translated from the Turkish original.

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This page was translated from Turkish by the Doktorclub editorial team.