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A new phase 3 trial is enrolling 500 patients to see if pridopidine, an oral sigma-1 receptor agonist, can slow functional decline and boost survival in early, rapidly progressing ALS. The PREVAiLS trial builds on promising — though not definitive — signals from a phase 2 study. Results could reshape treatment options for one of neurology's most devastating diseases.
A major new clinical trial is now enrolling patients in the fight against ALS. The PREVAiLS phase 3 trial is a randomized, double-blind, placebo-controlled study testing pridopidine — an oral, selective sigma-1 receptor agonist developed by Prilenia Therapeutics and Ferrer — in patients with early, rapidly progressing amyotrophic lateral sclerosis (ALS). The trial, highlighted at the 2026 NEALS Annual Meeting, received FDA clearance to proceed in December 2025 and is being conducted across approximately 60 ALS treatment centers worldwide.
The study is enrolling 500 participants who will receive either pridopidine 45 mg twice daily or placebo (on top of standard care) for 48 weeks, followed by a 48-week open-label extension. The primary endpoint is change in ALSFRS-R scores adjusted for mortality — a rigorous measure of functional decline. The trial's design was shaped by encouraging subgroup data from the phase 2 HEALEY ALS Platform Trial, which showed slower functional and respiratory decline, improved speech metrics, and a trend toward longer survival with pridopidine in early-stage patients.
Key Takeaways:
Why it matters: ALS remains a disease with very limited treatment options, especially for patients with rapid progression. If PREVAiLS confirms earlier signals, pridopidine could become a meaningful new tool for neurologists treating this devastating condition.
