PROOF-HD, a Phase 3 clinical trial in Huntington’s Disease (HD), is currently recruiting in Europe
The trial is designed to replicate previous findings of pridopidine demonstrating maintenance of functional capacity in early HD patients and could lead to the registration of pridopidine
PROOF-HD trial is endorsed by the European Huntington's Disease Network
Naarden, NL, 28 January 2021 - Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. The study is being conducted in 60 sites in collaboration with the Huntington Study Group (HSG) in North America and Europe. The first US subjects in the PROOF-HD trial were enrolled in October 2020.
PROOF-HD will take place at 30 sites across nine countries in Europe: Austria, Czech Republic, France, Germany, Italy, the Netherlands, Spain, Poland, and the UK. National approval has been granted in all European countries. The first participant was enrolled at George Huntington Institute (GHI) in Münster, Germany. The innovative protocol design, including virtual visits, will allow the study to continue undisrupted in case of any potential lockdown situations during the ongoing COVID-19 pandemic.
The study is a randomized, double-blind, placebo-controlled, Phase 3 study evaluating the efficacy and safety of pridopidine 45 mg bid in patients with early-stage HD. The primary endpoint is Total Functional Capacity. The global study will enroll a total of 480 participants aged 25 years or older with a clinical diagnosis of adult-onset HD in North America and Europe. The treatment period will last up to 78 weeks and there will be an optional open-label extension.
PROOF-HD is designed to replicate previous findings demonstrating that pridopidine shows a beneficial effect on the maintenance of functional capacity in patients with early HD. The trial treatment dose (45 mg bid) has a favorable safety profile based on over 1,000 patient years in previous HD trials.
Ralf Reilmann, MD, PhD, FAAN, Founding Director of the George-Huntington-Institute and European Principal Investigator of the study, said: “PROOF-HD is currently the only Phase 3 study available to early-HD patients in Europe, opening up an exciting opportunity for the patient community with an orally available drug. Pridopidine has shown a positive safety profile and promising results in earlier studies, making me confident of its potential to have a positive effect on everyday function and quality of life for patients.”
Michael R. Hayden, CEO of Prilenia and world-renowned scientist in Huntington’s Disease research, commented: “The design of the PROOF-HD study is based on strong scientific and clinical data, including
in vivo target engagement for the selected dose, prior clinical efficacy results and extensive long-term safety data in our target population. Enrolling our first patients in Europe is a significant milestone.”
Pridopidine is a first-in-class highly selective Sigma 1-receptor (S1R) agonist developed by Prilenia for the treatment of neurodegenerative disorders such as HD and Amyotrophic Lateral Sclerosis (ALS). Pridopidine binds and activates the S1R, a protein that is expressed at high levels within the brain and regulates key cellular pathways, commonly impaired in neurodegeneration. Recent analyses of previous PRIDE-HD and Open-HART trial results have shown positive data with regards to pridopidine on Total Function Capacity.