March 30, 2026
PREVAiLS is a pivotal, global, 500-participant Phase 3 study to evaluate efficacy and safety of pridopidine, an investigational drug, in slowing ALS progression (ALSFRS-R) in early, rapidly progressive participants
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The design of the potentially registrational pivotal Phase 3 study is informed by, and seeks to confirm, subgroup analysis data from the Phase 2 program in a similar rapid and progressive patient population
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PREVAiLS, believed to be the only currently recruiting Phase 3 ALS study, is set to take place in up to 60 leading ALS treatment centers across 13 countries. 11 sites are already, or will imminently be, initiated, with recruitment at additional sites in the US, Canada, EU, UK and Israel expected to commence in the coming weeks and months
Ferrer and Prilenia Therapeutics 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 (“PREVAiLS”, NCT07322003).
The first participant was enrolled at the Mass General Brigham (MGB), under the supervision of Sabrina Paganoni, MD, PhD, Co-Director of the MGH Neurological Clinical Research
Institute, and PREVAiLS principal investigator, who said: “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 (HD). 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.”
Oscar Pérez, Chief Scientific Officer at Ferrer, noted: “The initiation of the PREVAiLS trial marks a highly significant step in our mission to deliver transformative solutions for people living with rare and severe diseases, particularly those with a high unmet medical need. We work every day with a deep commitment to patients and their families, and collaborating
with Prilenia in this clinical study represents an opportunity to contribute to advancing
research in ALS.”
PREVAiLS is informed by peer-reviewed and published data from a subgroup analysis of similar participants with rapidly progressive ALS early in their disease course (pridopidine: n
= 37; shared placebo: n = 35), from the randomized, double-blind, placebo-controlled Phase 2 HEALEY ALS Platform Trial. The HEALEY trial did not meet its primary or secondary endpoints in the full population; pre-specified and additional analyses showed effects in rapid progressive patients that we seek to confirm in PREVAiLS.
Prof. Dr. Leonard van den Berg, Chair of The European Research Initiative to find a Cure for ALS (TRICALS), Professor of Neurology at the University Medical Centre Utrecht, and European member of the PREVAiLS Steering Committee declares: “The PREVAiLS study represents a shared commitment to advancing rigorous clinical research. Having been closely involved in the start-up and preparation phases, it is rewarding to see the study officially underway. We look forward to the continued global execution of the PREVAiLS
study.”
Ruben van Eijk, PhD, Associate professor at the University Medical Centre Utrecht, European member of the PREVAiLS Steering Committee, and medical statistician at TRICALS states: “We strongly believe in the power of close collaboration between Prilenia and Ferrer to successfully deliver a high-quality clinical trial. TRICALS is pleased to contribute to this important milestone through our ongoing support and dedication to generating robust and meaningful clinical evidence.”.
The PREVAiLS study is set to take place in up to 60 leading ALS treatment centers across 13 countries globally. Eleven sites are already, or will imminently be, initiated, with recruitment at additional sites in the US, Canada, EU, UK and Israel expected to commence in the coming weeks and months.
Evy Reviers, Chairwoman of the European Organisation of Professionals and People with ALS (EUpALS) comments: “EUpALS welcomes that Ferrer and Prilenia joined forces to initiate the PREVAiLS Phase 3 Study as it represents continued progress in rigorous clinical research in ALS. We greatly appreciate the commitment of both companies to involve people with ALS at different steps in the process and look forward to following the PREVAiLS study as it progresses.”
About PREVAiLS (Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS) PREVAiLS is a 48-week randomized (3:2 pridopidine:placebo), placebo-controlled study, with a 48-week open label extension phase to follow. The study seeks to enroll participants with definite or probable ALS (El Escorial Criteria) who are within 18 months from first onset of disease symptoms. The primary endpoint of PREVAiLS is the change from baseline in ALSFRS-R adjusted for mortality at 48 weeks. Secondary and exploratory endpoints include survival and measures of speech, respiratory function, bulbar function and quality of life, as well as patient-reported outcomes of communication and plasma biomarkers.
More details on PREVAiLS can be found on ClinicalTrials.Gov (NCT07322003) / EU CT Number: 2025-524002-16-00.
About pridopidine
Pridopidine (45 mg twice daily) is an investigational selective, orally administered sigma-1 receptor (S1R) agonist. S1R has been shown to play a role in stimulating multiple neuroprotective pathways impaired in neurodegenerative diseases, such as ALS and Huntington’s disease (HD).
In clinical studies to date, it has demonstrated a favorable safety and tolerability profile, with data from more than 1,600 people (mostly from HD studies), some of whom have received active treatment for, up to seven years.
In addition to ALS, Prilenia and Ferrer are planning to initiate a potentially registrational pivotal Phase 3 study in HD. The study is expected to start recruitment in the first half of 2026.
Pridopidine has Orphan Drug designation in HD and ALS in the US and EU, and FDA Fast Track designation for the treatment of HD.
Pridopidine is an investigational drug not approved by any regulatory authority. Its role in ALS and other neurological conditions is currently under clinical investigation.
Source: Press release Ferrer and Prilenia