WALTHAM, Mass. — Prilenia Therapeutics and Ferrer announced Thursday that their confirmatory Phase 3 PRECISE-HD clinical trial evaluating the investigational drug pridopidine for Huntington’s disease has received authorization to begin in participating European Union countries.
Patient recruitment in Europe is expected to begin this month, with the study expanding to more than 31 clinical sites across nine EU countries. Enrollment is already underway in the United States and United Kingdom, with additional sites in Canada expected to follow.
The global study is designed to enroll approximately 400 participants and evaluate the effectiveness and safety of pridopidine, an oral medication administered twice daily.
PRECISE-HD will examine the drug’s potential effects on disease progression, functional capacity, motor function, cognition, speech and quality of life. The randomized, double-blind, placebo-controlled trial is intended to generate additional evidence for regulatory evaluation.
“The initiation of PRECISE-HD in Europe marks a milestone for the Huntington’s disease research community. This confirmatory Phase 3 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,” said Dr. Ralf Reilmann, principal investigator and member of the PRECISE-HD Steering Committee.
“As investigators, we look forward to working with participating centers and the HD community to generate robust evidence that can help us better understand the potential role of the study treatment in HD,” Reilmann added.
Huntington’s disease is a rare, inherited neurodegenerative disorder that progressively affects movement, thinking, behavior and the ability to perform everyday activities. The condition is caused by a mutation in the huntingtin gene, and children of an affected parent have a 50% chance of inheriting the mutation.
An estimated 100,000 people worldwide are living with Huntington’s disease, while approximately 300,000 others are considered at risk. Symptoms typically emerge between ages 35 and 55, although the disease can develop earlier or later in life.
There are currently no approved treatments demonstrated to slow the overall progression of Huntington’s disease. Available therapies primarily focus on managing symptoms and providing supportive care.
“For people living with Huntington’s disease and their families, every step toward a better understanding of the disease and potential treatment options matters,” said Astri Arnesen, president of the European Huntington Association.
“We welcome the involvement of European centers in PRECISE-HD and the continued focus on outcomes that reflect the realities and priorities of people living with HD and their families,” Arnesen added.
The PRECISE-HD trial will include patients with early- to mid-stage Huntington’s disease and will be conducted at up to 75 clinical sites worldwide.
The study consists of two consecutive phases. During the initial 52-week period, participants will be randomly assigned to receive either pridopidine or a placebo. That will be followed by a 104-week open-label extension, during which all eligible participants will receive pridopidine.
The trial’s primary endpoint will measure changes in the combined Unified Huntington’s Disease Rating Scale score after 52 weeks, providing an assessment of changes in neurological and functional abilities.
Pridopidine is a highly selective sigma-1 receptor agonist being developed as a potential treatment for Huntington’s disease and amyotrophic lateral sclerosis (ALS). The sigma-1 receptor is involved in cellular processes associated with protecting nerve cells.
According to the companies, clinical studies involving more than 1,600 participants have demonstrated a generally favorable safety and tolerability profile for pridopidine. Some participants have received the investigational treatment for as long as seven years.
The drug has received Orphan Drug Designation for Huntington’s disease and ALS in both the United States and European Union, as well as Fast Track designation from the U.S. Food and Drug Administration for Huntington’s disease.
However, pridopidine remains investigational and has not been approved for commercial use by any regulatory authority. Its safety and effectiveness have not been established.
Prilenia and Ferrer also announced plans to present new clinical analyses and trial updates at the European Huntington’s Disease Network Clinical Research Congress, scheduled for October 22–24 in Kraków, Poland.
Prilenia CEO Dr. Michael R. Hayden will deliver a presentation addressing clinical trial design and the determination of clinically meaningful treatment outcomes.
The companies will also present four scientific posters covering clinically meaningful changes in early-stage Huntington’s disease, placebo responses observed in the earlier PROOF-HD trial, and the designs of the PREVAiLS and PRECISE-HD studies investigating pridopidine in ALS and Huntington’s disease, respectively.
