BARCELONA, Spain, NAARDEN, The Netherlands and WALTHAM, Mass. – March 30, 2026 – Ferrer and Prilenia Therapeutics B.V.1 today announced the inclusion of the first participant in the PREVAiLS, a pivotal Phase 3, randomized, placebo-controlled, global study with 500 participants, designed to evaluate pridopidine in people with rapidly progressing early Amyotrophic Lateral Sclerosis (ALS) ("PREVAiLS", NCT07322003).
The first participant was included in the Mass General Brigham (MGB), under the supervision of Dr. Sabrina Paganoni, co-director of the MGH Neurological Clinical Research Institute and principal investigator of PREVAiLS, who stated:
"Pridopidine is an activator of the sigma-1 receptor (S1R). S1R has been shown to play a role in stimulating multiple neuroprotective pathways that are altered in neurodegenerative diseases such as ALS and Huntington's disease (HD)ii. The inclusion of the first participant in this confirmatory study is a milestone in the search for new therapeutic options that can help slow disease progression, preserve function, maintain speech and prolong survival, key goals in the early treatment of ALS."
Oscar Pérez, Chief Scientific Officer at Ferrer, said: "The start of the PREVAiLS trial is a very important step in our purpose to offer transformative solutions for people living with rare and severe diseases, especially those with a high unmet medical need. At Ferrer 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 responsibly to the advancement of ALS research."
PREVAiLS is based on peer-reviewed, published data from a subgroup analysis of participants with early-stage rapid progression ALS (pridopidine: n = 37; placebo sharing: n = 35) from the Phase 2 randomized, double-blind, placebo-controlled HEALEY ALS Platform trial. The HEALEY trial did not achieve its primary or secondary endpoints in the global population; pre-specified and additional analyses showed effects in rapidly progressing patients that are intended to be confirmed 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, states: "The PREVAiLS study represents a shared commitment to advancing rigorous clinical research. Having been closely involved in the preparation and commissioning phases makes it especially gratifying to see the study officially underway. We look forward to the continuation of the global implementation of the PREVAiLS study."
Dr. Mónica Povedano, coordinator of the Functional Unit of Motor Neuron Diseases and head of the Neurophysiology Section, Neurology Service, of the Bellvitge-IDIBELL University Hospital, "PREVAiLS is an example of the commitment and collaboration that Ferrer and Prilenia maintain to advance in the research of new treatments that can modify the course of ALS. In addition, thanks to the scientific rigour provided by TRICALS, the study reinforces Catalonia's international leadership in clinical research and innovation in rare neurodegenerative diseases."
The PREVAiLS study will be conducted at up to 60 leading ALS treatment centers in 13 countries globally. Eleven centers have already been launched, or will be initiated, and the inclusion of participants in other centers in the United States, Canada, the European Union, the United Kingdom, and Israel is expected to begin in the coming weeks and months.
Fernando Martín Pérez, president of the National Confederation of ALS Entities (ConELA) adds: "ConELA values the start of PREVAiLS in Europe and Spain: rigour, transparency and responsible communication, without unfounded expectations, putting people at the centre."
More information about PREVAiLS can be found at ClinicalTrials.gov (NCT07322003) / EU Clinical Trial Number: 2025-524002-16-00.
[This press release is for informational purposes only. Pridopidine is an investigational medicine and is not approved for commercial use by any regulatory authority. Their safety and efficacy have not been established. The information contained herein does not constitute medical advice. Patients should consult their healthcare professional for guidance on diagnosis or treatment options. Local regulations may vary; This release is not intended to promote or advertise any product.]
About PREVAiLS (Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS)
PREVAiLS is a randomized (3:2 pridopidine:placebo), placebo-controlled, 48-week study, followed by a 48-week open-label extension phase. The study seeks to include participants with a diagnosis of definitive or probable ALS (El Escorial criteria) and with less than 18 months from the onset of symptoms. The primary endpoint is change from baseline in the mortality-adjusted ALSFRS-R scale 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 and plasma biomarkersiii.
More details about PREVAiLS in ClinicalTrials.Gov (NCT07322003) / EU CT Number: 2025-524002-16-00.
About pridopidine
Pridopidine (45 mg twice daily) is an investigational oral, selective sigma-1 receptor (S1R) agonist. S1R has been shown to play a role in stimulating multiple altered neuroprotective pathways in neurodegenerative diseases such as ALS and HD. In clinical studies conducted to date, it has shown a favourable safety and tolerability profile, with data from more than 1,600 people (mainly from HD studies), some of whom have received active treatment for up to seven yearsiv.
In addition to ALS, Prilenia and Ferrer plan to initiate a pivotal phase 3 potentially registry-based study in HD. Recruitment is expected to begin in the first half of 2026.
Pridopidine has orphan drug designation for 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 diseases is currently under clinical investigation.
About ALS
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease or motor neuron disease (MND), is a progressive neurodegenerative disease, meaning it is a chronic condition that worsens over time as damage occurs to parts of the nervous systemv. It affects a specific type of neurons, the motor neurons of the brain and spinal cord, and leads to a gradual loss of muscle function that eventually results in paralysis and deathvi. ALS affects about 500,000 people worldwidevii, and is more common in men than in womenviii. Median survival is 2 to 5 yearsix. The exact cause of ALS is not fully known, but it is thought to be a combination of genetic and environmental factors.
ALS usually appears between the ages of 40 and 70x and presents with a variety of symptoms, including muscle weakness and atrophy, mobility problems such as lack of coordination and balance, muscle spasms, difficulty speaking clearly (dysarthria), swallowing problems (dysphagia), fatigue, and emotional and cognitive changesxi. Symptoms worsen over time and significantly affect the quality of life of patients and their families by making it difficult to perform independently daily activities. Current treatment options are limited.
S1R dysfunction has been associated with multiple forms of ALS, and maintaining its functionality may be key to protecting neuronal function.
