Prilenia Therapeutics and Ferrer have secured EU authorisation to begin PRECISE-HD, a confirmatory Phase III trial of pridopidine to treat Huntington’s disease (HD).
Enrolment in the EU is scheduled to start this month, expanding an international clinical programme that is already recruiting patients in the UK and the US, with Canadian centres scheduled to join subsequently.
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The global, randomised, double-blind, placebo-controlled trial will enrol around 400 individuals with early- to mid-stage HD, defined by a total functional capacity (TFC) score of 7 to 13.
To be conducted at up to 75 centres across Canada, the EU, the UK, and the US, the study seeks to generate clinical data for regulatory assessment.
It will assess the effects of oral pridopidine, administered twice daily, on disease progression, motor performance, cognition, functional capacity, speech, and overall quality of life.
PRECISE-HD Steering Committee member and principal investigator Dr Ralf Reilmann said: “The initiation of PRECISE-HD in Europe marks a milestone for the Huntington’s disease research community. This confirmatory Phase III study has been designed to build on learnings from previous research and to rigorously evaluate the efficacy and safety of pridopidine in people living with HD.
“As investigators, we look forward to working with participating centres and the HD community to generate robust evidence that can help us better understand the potential role of the study treatment in HD.”
The trial protocol features two sequential parts: an initial 52-week placebo-controlled phase with 1:1 participant randomisation, and a subsequent 104-week open-label extension period in which all eligible subjects receive active therapy.
Researchers have established the primary endpoint as the change in the combined Unified Huntington’s Disease Rating Scale (cUHDRS) score from baseline to week 52.
Pridopidine is an investigational, highly selective oral sigma-1 receptor agonist that targets neuroprotective pathways affected in amyotrophic lateral sclerosis (ALS) and HD.
The therapy holds orphan drug designations in the EU and the US for both indications, alongside the US Food and Drug Administration (FDA) fast track status for HD.
In March, Prilenia Therapeutics 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.
