Prilenia and Ferrer Enroll First Participant in ALS Drug Trial

Pivotal Phase 3 study to evaluate pridopidine's potential to slow disease progression in early, rapidly progressive ALS

Mar. 30, 2026 at 11:53am

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Prilenia Therapeutics B.V. and Ferrer have announced the first enrollment in the pivotal, 500-participant, randomized, placebo-controlled Phase 3 study of pridopidine in people with rapidly progressive Amyotrophic Lateral Sclerosis (ALS) early in their disease course. The study, called PREVAiLS, is set to take place in up to 60 leading ALS treatment centers across 13 countries.

Why it matters

ALS is a devastating neurodegenerative disease with limited treatment options, and the ALS community urgently needs new therapies that can delay the disease's relentless progression. The PREVAiLS study aims to confirm the potential of pridopidine, a sigma-1 receptor agonist, to slow ALS progression in patients with early, rapid disease based on promising subgroup analysis data from a previous Phase 2 trial.

The details

PREVAiLS is a 48-week randomized (3:2 pridopidine:placebo), placebo-controlled study, with a 48-week open label extension phase. The study will enroll participants with definite or probable ALS who are within 18 months from first onset of disease symptoms. The primary endpoint is the change from baseline in ALSFRS-R (a measure of ALS progression) adjusted for mortality at 48 weeks. Secondary and exploratory endpoints include survival, speech, respiratory function, bulbar function, quality of life, and biomarkers.

  • The first participant was enrolled at Mass General Brigham on March 30, 2026.

The players

Prilenia Therapeutics B.V.

A private biopharmaceutical company driven by a commitment to developing transformative medicines for people affected by devastating neurodegenerative diseases like HD and ALS.

Ferrer

A Barcelona-based pharmaceutical company that focuses on transformative solutions for life-threatening diseases and has an increasing focus on pulmonary vascular, interstitial lung, and rare neurological disorders.

Sabrina Paganoni, MD, PhD

Co-Director of the MGH Neurological Clinical Research Institute and principal investigator for the PREVAiLS study.

Kuldip Dave, Ph.D.

Senior Vice President of Research at the ALS Association.

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What they’re saying

“Pridopidine is a sigma-1 receptor (S1R) agonist. The S1R has been shown to play a role in stimulating multiple neuroprotective pathways impaired in neurodegenerative diseases, such as ALS and Huntington's disease. Enrolling the first participant in this confirmatory study is a milestone in our search for potential new therapeutic options that may help to slow disease progression, preserve function, maintain speech and prolong survival – key aims of early ALS therapy.”

— Sabrina Paganoni, MD, PhD, Co-Director of the MGH Neurological Clinical Research Institute, PREVAiLS principal investigator

“The ALS community urgently needs new treatment options that can delay the disease's relentless progression, and awaits the outcome of this study. The earlier we can diagnose and treat ALS, the greater the potential to preserve function and maintain quality of life for longer, which are key to making ALS livable until we can cure it.”

— Kuldip Dave, Ph.D., Senior Vice President of Research, ALS Association

What’s next

The PREVAiLS study is expected to recruit participants at additional sites in the US, Canada, EU, UK and Israel in the coming weeks and months.

The takeaway

This pivotal Phase 3 trial of pridopidine represents a promising new approach to potentially slow the progression of ALS, a devastating disease with limited treatment options. If successful, pridopidine could offer a much-needed new therapy to help preserve function and quality of life for ALS patients.