PROOF-HD is a Phase 3 clinical study that evaluates the efficacy and safety of pridopidine in patients with early stage of Huntington Disease. Pridopidine is a small molecule developed by Prilenia for the treatment of neurodegenerative disorders such as HD.
According to Prilenia, the Pridopidine is an oral drug administered in a small easy-to-swallow capsule twice a day, which enters the brain and spinal cord, where it activates a protein called the sigma-1 receptor (S1R). Activation of the S1R by pridopidine enhances the clearance of toxic proteins, enhances energy production, and reduces cellular stress and inflammation. These mechanisms are crucial for a neuron’s function and survival.