- PROOF-HD is designed to replicate previous findings of pridopidine demonstrating maintenance of functional capacity in early Huntington’s Disease patients
- Conducted in collaboration with the Huntington Study Group, a world leader in clinical research for HD
- If successful, PROOF-HD could lead to the registration of pridopidine, one of the few oral drugs currently in clinical development for the treatment of HD.
Naarden: 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 patients in the U.S. into PROOF-HD, a global phase 3 clinical trial for Huntington’s Disease (HD). The study is being conducted in collaboration with the Huntington Study Group (HSG).
Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is a randomized, double-blind, placebo-controlled, phase 3 study evaluating the efficacy and safety of pridopidine 45mg bid in patients with early stage HD. The study will enroll 480 participants aged 25 or older with a clinical diagnosis of adult-onset HD at approximately 60 study centers in the U.S., Canada and Europe. The treatment period will last up to 78 weeks and there will be an optional open-label extension.
The study is designed to replicate previous findings that demonstrated pridopidine’s effect in maintaining functional capacity in patients with early HD. The trial treatment dose (45mg bid) has a favorable safety profile based on over 1,000 patient years in previous HD trials.
Andrew Feigin, MD, North American Principal Investigator for the PROOF-HD Phase 3 Trial, said: “Huntington’s Disease is a serious condition with no known treatments that slow functional decline. A successful result would be a major advance in our ability to treat HD. I look forward to working with the Prilenia team as we push forward with patient recruitment.”
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”. He added: "Enrolling our first patients is a significant milestone that brings hope to others suffering from this devastating disease.”
Pridopidine is a first in class small molecule developed by Prilenia for the treatment of neurodegenerative disorders such as HD and Amyotrophic Lateral Sclerosis (ALS). Pridopidine is a highly selective Sigma-1 receptor (S1R) agonist. It 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.
Further details of the trial can be found here: https://huntingtonstudygroup.org/proof-hd/
For further information, please contact:
Contacts:
Prilenia Therapeutics
Michael Hayden, CEO
Limor Ben Har, COO
E: info@prilenia.com
Instinctif Partners
Melanie Toyne-Sewell / Agnes Stephens / Siobhan Sanford
T: +44 (0) 207 457 2020
E: prilenia@instinctif.com