Prilenia Therapeutics and Ferrer have begun the confirmatory Phase III PRECISE-HD clinical trial assessing pridopidine as a potential treatment for Huntington’s disease (HD).
The randomised, double-blind, placebo-controlled trial aims to further evaluate the efficacy and safety of the investigational drug pridopidine, an oral capsule taken twice daily.
Recruitment has started in the US, and additional countries are scheduled to begin enrolment later this year.
The trial aims to assess pridopidine’s clinical impact on cognition, disease progression, functional capacity, motor function, quality of life, and speech in HD patients.
The data generated will support potential registration of the treatment.
PRECISE-HD plans to enrol 400 participants living with early to mid-stage HD, as defined by a Total Functional Capacity score between 7 and 13, a Total Motor Score of 20 or higher, and an Independence Scale score of 90% or less.
Up to 75 sites in Canada, the EU, the UK, and the US will conduct the study, integrating patient and regulatory input.
University of Alabama movement disorders division director Victor Sung is serving as the trial's steering committee member.
Sung said: “Across prior studies, pridopidine has shown meaningful clinical effects in specific circumstances, and this study has been carefully designed to confirm those effects and uniquely incorporates prior learnings and novel elements specifically designed to provide treatment effect clarity.”
PRECISE-HD features a 52-week placebo-controlled period, followed by a 104-week open-label extension where all eligible participants receive pridopidine.
Outcomes will be compared to external control cohorts from multinational studies. The primary outcome is the change from baseline to week 52 in the combined Unified Huntington’s Disease Rating Scale (cUHDRS) score.
More than 1,600 participants, mainly from HD studies, have received pridopidine, some for up to seven years.
Pridopidine has orphan drug status for HD and amyotrophic lateral sclerosis (ALS) in the US and EU, and fast track designation from the US Food and Drug Administration (FDA) for HD.
In March 2026, Prilenia and Ferrer enrolled the first participant in the pivotal Phase III PREVAiLS clinical trial assessing pridopidine in individuals with rapidly progressive ALS early in disease progression.
