Prilenia Therapeutics has enrolled the first patients in a Phase III trial of pridopidine for the treatment of Huntington’s Disease (HD) in the US.
Conducted in collaboration with the Huntington Study Group (HSG ), the Phase III Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) trial plans to replicate previous findings of pridopidine showing maintenance of functional capacity in early HD patients.
Developed by Prilenia for treating neurodegenerative disorders, such as HD and Amyotrophic Lateral Sclerosis (ALS), pridopidine is a highly selective Sigma-1 receptor (S1R) agonist.
It attaches and activates the S1R, a protein expressed at high levels within the brain, and regulates key cellular pathways. S1R is commonly impaired in neurodegeneration.
The PROOF-HD randomised, double-blind, placebo-controlled trial will analyse the efficacy and safety of pridopidine 45mg bid in patients with early-stage HD.
It will have 480 participants aged 25 or above with adult-onset HD and will be conducted in about 60 centres in the US, Canada and Europe.
The trial will have a 78-week treatment period, with an optional open-label extension.
Prilenia Therapeutics CEO Michael Hayden said: “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 is a significant milestone that brings hope to others suffering from this devastating disease.”
Pridopidine 45mg bid, the trial treatment dose, has a favourable safety profile and was well tolerated in prior HD trials conducted on over 1,000 patients.
It is among the few oral drugs currently in the clinical development stage for the treatment of HD. Positive results from the trial could lead to the drug’s registration.
In June, Prilenia raised $62.5m through a Series A financing round to launch the HD and ALS late-stage clinical trials.