Prilenia Therapeutics and Ferrer have launched the global Phase 3 PRECISE‑HD trial (NCT07609108) of pridopidine in early to mid‑stage Huntington’s disease, enrolling ~400 patients worldwide to generate pivotal efficacy and safety data for regulatory evaluation.
Written By: Nalam Karthik, PharmD
Reviewed By: Pharmacally Editorial Team
Prilenia Therapeutics and Ferrer have launched the global Phase 3 PRECISE-HD trial (NCT07609108) evaluating the investigational oral therapy pridopidine in people with early to mid-stage Huntington’s disease (HD). Patient recruitment has begun in the United States, with additional sites across Europe, the United Kingdom, and Canada expected to open later this year.
The randomized, double-blind, placebo-controlled study will enroll approximately 400 participants at up to 75 sites worldwide. The trial will generate confirmatory efficacy and safety data to support future regulatory evaluation of pridopidine, an oral capsule taken twice daily.
Study design targets disease progression
PRECISE-HD incorporates findings from earlier clinical studies together with input from researchers, patient organizations, and regulatory authorities. Eligible participants must have early to mid-stage Huntington’s disease, defined by a Total Functional Capacity (TFC) score of 7-13, Total Motor Score (TMS) of at least 20, and an Independence Scale score of 90% or lower. This population has measurable motor impairment and functional decline, enabling a clearer assessment of treatment effects on disease progression.
Participants will be randomized 1:1 to receive pridopidine or placebo during a 52-week double-blind phase, followed by a 104-week open-label extension in which all eligible participants will receive pridopidine. Investigators will evaluate long-term outcomes for up to three years using matched external control cohorts from multinational observational Huntington’s disease studies.
The primary endpoint is the change from baseline to Week 52 in the combined Unified Huntington’s Disease Rating Scale (cUHDRS). Secondary endpoints include functional capacity, motor function, cognition, speech, quality of life, disease progression, and long-term safety.
Sigma-1 receptor agonist in late-stage development
Pridopidine is an investigational, highly selective sigma-1 receptor (S1R) agonist, a target involved in multiple neuroprotective pathways disrupted in neurodegenerative diseases. The therapy is also in late-stage development for amyotrophic lateral sclerosis (ALS) through the pivotal Phase 3 PREVAiLS trial (NCT07322003).
Across previous clinical studies involving more than 1,600 participants, primarily individuals with Huntington’s disease, pridopidine has demonstrated a generally favorable safety and tolerability profile. Some patients have remained on treatment for up to seven years, although the therapy remains investigational and has not been approved by any regulatory authority.
Experts highlight unmet need
Katie Jackson, President and CEO of Help 4 HD International, said families affected by Huntington’s disease continue to face an urgent need for treatments that slow disease progression rather than simply relieve symptoms. She emphasized the importance of completing the study quickly so its clinical value can be fully evaluated.
Victor Sung, MD, Director of the Division of Movement Disorders at the University of Alabama and a PRECISE-HD Steering Committee member, said the trial builds on encouraging findings from previous studies while incorporating methodological improvements intended to provide clearer evidence of pridopidine’s clinical effects.
Regulatory Path Forward
Huntington’s disease is a rare, inherited, autosomal dominant neurodegenerative disorder caused by mutations in the HTT gene. It progressively impairs motor function, cognition, behavior, and independence over 15 to 20 years, ultimately leading to death. Approximately 100,000 people worldwide are living with the disease, with another 300,000 individuals at genetic risk. Current therapies provide symptomatic relief, but none has demonstrated an ability to slow overall disease progression.
Pridopidine has received Orphan Drug designation for Huntington’s disease and ALS in both the United States and European Union, along with FDA Fast Track designation for Huntington’s disease. As enrollment expands globally, PRECISE-HD is expected to provide the pivotal evidence needed to determine whether pridopidine can become a disease-modifying treatment for Huntington’s disease.
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About the Writer
Nalam Karthik (LinkedIn) is a healthcare writer and PharmD graduate with interests in pharmacovigilance, drug safety, clinical data analysis, and quality assurance. He is passionate about translating clinical and pharmaceutical knowledge into accessible healthcare content while staying engaged with advancements in drug development and patient safety initiatives.