Pan-PPAR Agonists for the Treatment of Tauopathies and Huntington's Disease

Background & Unmet Need

• Huntington’s disease (HD) is a dominantly inherited progressive neurodegenerative disease characterized by progressive motor impairment, personality changes, and gradual intellectual decline
• While therapies exist to help treat symptoms, there are currently no approved therapies that stop or reverse decline
• Previous studies have identified a potential link between impairment of the peroxisome proliferator-activated receptor (PPAR)-γ-coactivator 1α (PGC-1 α) levels and activity are HD pathogenesis
• Unmet Need: Disease-modifying therapeutics for the treatment of HD and other neurodegenerative diseases

Technology Overview

• The Technology: Administration of bezafibrate or combinations of PPAR agonists for the treatment of HD and tauopathies
• The Discovery: Bezafibrate improved behavioral impairments, neuronal loss, and prolonged survival in mouse models of HD
• Administration of bezafibrate increased numbers of mitochondria in both brain and muscle tissue
• Bezafibrate treatment also improved the behavioral impairments and tau aggregation in mouse models of tauopathy

Technology Applications

• Repurposing of bezafibrate, or novel combinations of selective PPAR agonists
• Treatment of neurological conditions

Technology Advantages

• Bezafibrate is already approved drug for other indications, which may streamline development
• Potentially applicable to multiple indications