Pipeline &
Clinical Studies
Pipeline
Pridopidine (45 mg twice daily) is a potent and highly selective, orally administered sigma-1 receptor (S1R) agonist designed to regulate key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS.
In April 2025, Prilenia and Ferrer entered into a collaboration and license agreement for the commercialization and co-development of pridopidine in Europe and other select international markets. Prilenia retains full commercialization and development rights to pridopidine in North America, Japan and Asia Pacific.
Prilenia and Ferrer are working hard to bring pridopidine to HD and ALS patients as quickly as possible. Our global confirmatory Phase 3 studies of pridopidine in ALS and HD have both started enrolling participants, as we continue discussions with regulatory authorities globally on the pathway to approval.
We also continue our early research on pridopidine, S1R and its neuroprotective role in multiple other CNS indications.
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Vanishing white matter disease*
Potential additional rare neurodegenerative indications
Potential additional common neurodegenerative indications
Pridopidine is an investigational product and has not been approved by the US Food & Drug Administration or other ex-US regulatory agencies. Its safety and efficacy have not yet been determined.
Phase 3 Clinical Trials in ALS and HD
ALS Clinical Trials
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
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
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 at ClinicalTrials.Gov (NCT07322003) / EU CT Number: 2025-524002-16-00.
HD Clinical Trials
The confirmatory PRECISE-HD study (Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease (HD)) is focused on further evaluating the efficacy and safety of pridopidine, an investigational medicinal product (taken as an oral capsule twice a day), and the generation of data to support registration. PRECISE-HD has commenced recruitment in the US, with other countries expected to follow shortly.
The approximately 400-participant randomized, double-blind, placebo-controlled PRECISE-HD study will assess the clinical impact of pridopidine on disease progression, functional capacity, motor function, cognition, speech and quality of life (QoL).
Incorporating learnings from prior research and advice and input from the HD patient and research community and regulatory authorities, the study will focus on people living with HD, from early to mid-stage disease (defined as Total Functional Capacity (TFC) score of 7-13), a Total Motor Score (TMS) ≥20 and Independence Scale score ≤ 90%, meaning people who have experienced some motor changes or impact on independent functioning. This will allow for appropriate assessment of any potential effect of the therapy on disease progression in comparison to placebo. PRECISE-HD will be undertaken in up to 75 sites globally, including the US, EU, UK and Canada.
PRECISE-HD is comprised of two consecutive stages: A 52-week placebo-controlled stage with participants randomized to receive either pridopidine or placebo on a 1:1 basis. This will be followed by a 104-week open-label extension stage, in which all eligible participants will receive pridopidine, allowing researchers to assess any potential effect on disease progression for up to three years in total, compared to matched external control cohorts from longitudinal, multinational observational studies.
Further details on the PRECISE-HD study can be found at: www.clinicaltrials.gov NCT07609108.
Expanded Access
We understand that, in some cases, patients with serious diseases may not be eligible to participate in a clinical study, and there may not be any alternative treatment options for their disease. We are deeply committed to the patient community, and under certain circumstances, we will consider making pridopidine available to individual patients through an expanded access program (also referred to as early access or compassionate use).