RIPK1 Inhibitor SAR443820 Fails to Slow ALS Progression in Phase 2 Trial

The HIMALAYA phase 2 trial randomised 305 adults with ALS to the oral RIPK1 inhibitor SAR443820 or placebo over 24 weeks. The trial reported no statistically significant difference in ALSFRS-R functional decline between groups, while the SAR443820 group had more adverse events, more treatment discontinuations, and higher rates of elevated hepatic enzymes; the trial was terminated early and the authors state further clinical development of SAR443820 in ALS is not warranted.

Most relevant for: Adults with possible, probable, or definite amyotrophic lateral sclerosis being considered for investigational RIPK1-targeted therapies.

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