What's Happening?
Prilenia Therapeutics and Ferrer have announced that the U.S. Food and Drug Administration (FDA) has cleared the start of a pivotal Phase 3 study, named PREVAiLS, to evaluate the efficacy and safety of pridopidine
in patients with rapidly progressive amyotrophic lateral sclerosis (ALS). The study will involve 500 participants and will be conducted across 60 ALS treatment centers globally, including the U.S., Canada, and Europe. The trial aims to confirm previous Phase 2 data that suggested pridopidine could offer clinically meaningful improvements in disease progression, speech, and survival for ALS patients. Recruitment for the study is expected to begin in early 2026.
Why It's Important?
ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disease with limited treatment options. The initiation of the PREVAiLS study represents a significant step in the search for new therapies that could potentially slow disease progression and improve quality of life for ALS patients. Pridopidine's mechanism as a sigma-1 receptor agonist offers a novel approach to neuroprotection, which could be crucial in managing ALS. The study's outcomes could influence future treatment protocols and provide hope for patients and families affected by this debilitating condition.
What's Next?
Following the FDA's clearance, the PREVAiLS study will begin recruitment in early 2026. The study will include a 48-week double-blind placebo-controlled phase followed by a 48-week open-label extension phase. The primary endpoint will focus on changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores adjusted for mortality. Secondary endpoints will assess speech, respiratory function, and quality of life. The results of this study could pave the way for regulatory approval and broader access to pridopidine for ALS patients.
