The Huntington Study Group (“HSG”), a world leader in clinical research for Huntington’s Disease (HD), announces a partnership with Prilenia Therapeutics, a clinical stage biotech company led by Michael R. Hayden, MD, PhD, to conduct PRidopidine Outcome On Function in Huntington’s Disease (PROOF-HD) clinical study. PROOF-HD is a global Phase 3, randomized, double-blind, placebo-controlled, parallel arm, multicenter study evaluating the efficacy and safety of pridopidine in patients with early stage HD.
Pridopidine is a first in class small molecule which is a highly selective Sigma-1 receptor (S1R) agonist. Prior trials of pridopidine in HD have demonstrated good safety and tolerability. The PROOF-HD study seeks to demonstrate that pridopidine slows functional decline over 65 weeks of treatment.
In a prior Phase 2 clinical study, PRIDE-HD, pridopidine administered orally twice a day, was associated with maintenance of functional capacity from baseline compared to placebo at 52 weeks in patients with early HD, as measured by Total Functional Capacity (TFC). Extensive safety data from over 1300 subjects exposed to various oral daily doses of pridopidine demonstrates pridopidine has a favorable safety profile and is well tolerated.
“Slowing the decline in functional capacity in HD patients would be a significant advance in HD care,” said Andrew Feigin, MD, North American Principal Investigator of PROOF-HD. “We are excited to partner with Prilenia to study pridopidine this study directly aligns with HSG’s mission of seeking treatments that make a difference for those affected by HD.”
HSG is also partnering with TFS International AB, a clinical research organization (CRO), to conduct this trial in Europe, and with the Clinical Trials Coordination Center at the University of Rochester to assist with efforts in North America. PROOF-HD will be conducted through HSG Clinical Research, Inc.
“This is an exciting time in Huntington’s Disease research,” added Ralf Reilmann, MD, PhD, the European Principal Investigator of the study. “Prior trials of pridopidine have suggested that it may maintain functional capacity in early HD patients, as measured by Total Functional Capacity (TFC). PROOF-HD will focus on this potential effect.”
The PROOF-HD study plans to enroll 480 participants aged 25 or older with a clinical diagnosis of adult-onset HD in approximately 30 study centers across the U.S. and Canada, and another 30 study centers across Europe. The study will include a screening period, a double-blind placebo-controlled treatment period up to 78 weeks and optional open-label extension.
“There is extensive preclinical evidence that further supports pridopidine’s potential beneficial effect in HD,” said Michael R. Hayden, MD, PhD, Chief Executive Officer at Prilenia. “It is a first-in-class drug candidate, with promising previous clinical results and an established safety profile. We are pleased to be partnering with HSG to explore its impact through the PROOF-HD study.”
About Huntington’s Disease
Huntington’s Disease (HD) is a hereditary neurodegenerative disease characterized by a movement disorder, psychiatric difficulties, and cognitive changes, usually beginning in middle adult life. Additional characteristics of HD include weight loss (probably from a combination of difficulty eating, and calories burned by the involuntary movements), difficulty swallowing, and hard-to-understand speech. About 30,000 people in North America have HD, and another 150,000 are considered “at risk” for inheriting the illness because they have (or had) a parent with HD.
About Huntington Study Group Clinical Research (HSG)
Founded in 1993 in Rochester, NY, the Huntington Study Group (hsglimited.org | huntingtonstudygroup.org) is a not-for-profit organization comprised of the world’s first and largest collaborative network of experts in Huntington’s Disease. HSG Clinical Research, Inc. is a wholly-owned for-profit subsidiary of the HSG, conducts clinical trials to benefit the HSG and its mission of seeking treatments that make a difference for those affected by HD. There are 700 credentialed HD experts at more than 120 HSG credentialed research sites worldwide. The HSG also offers educational services like CME4HD ™ for healthcare professionals and care providers on treating patients with HD.
Prilenia (prilenia.com) is a clinical stage biotech startup founded in 2018 with the purpose of improving the lives of patients and their families by developing treatments for neurodegenerative and neurodevelopmental disorders. Prilenia raised $ 82.5 million thus far and is backed by a group of well-respected investors: Talisman, Forbion, Morningside and Sectoral. The Company is based in Naarden, the Netherlands, Herzliya, Israel and Boston, MA in the U.S.
About TFS International
TFS (tfscro.com) was founded in 1996 and has grown to become the leading global mid-size clinical CRO focusing on small and mid-size Biotech customers. TFS employs nearly 700 professionals throughout 21 countries and currently delivers clinical research services in more than 40 countries. TFS provides end-to-end solutions including full clinical development services, strategic resourcing, and flexible single services. TFS partnering approach with customers is based on our four business principles commitment, flexibility, value creation and global reach. Our core therapeutic specialties are Dermatology, Hematology and Oncology, Ophthalmology as well as Internal Medicine and Neurology.
About the CTCC
The Clinical Trials Coordination Center (CTCC) is part of the University of Rochester Medical Center’s Center for Health + Technology (CHeT). The CTCC specializes in the development, management, and conduct of clinical research studies. Over the past 25 years, the CTCC has managed the conduct of over 100 clinical research studies with 45 sponsors (government, industry, and private) that enrolled over 38,000 research participants in US, Canada, Europe, New Zealand, and Australia.
Visit the CTCC website