ALS study launches as drug aims to slow progression

The PREVAiLS phase 3 trial has enrolled its first participant to evaluate pridopidine in early, rapidly progressing ALS; the global study plans about 500 participants across up to 60 centers in 13 countries.

Researchers have enrolled the first participant in PREVAiLS, a global phase 3 study testing the investigational drug pridopidine for early, rapidly progressing ALS. The trial follows phase 2 HEALEY platform findings that did not meet the main goal overall but showed positive signals in some patients who were early in disease and declining rapidly. Pridopidine is described by researchers as a sigma-1 receptor agonist that may engage neuroprotective pathways. The study will evaluate safety and whether the drug can slow disease progression in the selected patient group. What we know: - The first participant was enrolled at Mass General Brigham under lead investigator Sabrina Paganoni, MD, PhD. - PREVAiLS plans to enroll about 500 participants at up to 60 ALS treatment centers across 13 countries. - Phase 2 HEALEY results did not meet the primary endpoint overall but showed subgroup signals; the phase 2 safety profile was generally similar to placebo, with common events overlapping ALS symptoms. Summary: The phase 3 PREVAiLS study aims to determine whether pridopidine can provide consistent benefits for people with early, rapidly progressing ALS, building on subgroup findings from phase 2. Definitive conclusions will await completion and full analysis of the phase 3 trial. Undetermined at this time.