Woolsey -Model ROCKIT-1 -Progressive Supranuclear Palsy (PSP) and Corticobasal Syndrome (CBS) Technology
Progressive supranuclear palsy (PSP) and corticobasal syndrome (CBS) are rare neurodegenerative disorders associated with the accumulation of insoluble deposits of predominantly 4 microtubule binding domain repeat (4R) tau protein in specific brain regions, mainly in the absence of other pathogenic proteins including β-amyloid, which is found in Alzheimer’s disease.
(ROCK Inhibitor in Tauopathies – 1)
ROCKIT-1 is a phase 2a open-label study to assess preliminary safety, tolerability, and effect on neurodegeneration biomarkers of BRAVYL in patients with the 4-Repeat (4R) Tauopathies of Progressive Supranuclear Palsy-Richardson Syndrome (PSP-RS) or Corticobasal Syndrome (CBS). In addition, ROCKIT-1 will assess whether BRAVYL improves clinical features of the disease.
The study will enroll approximately 15 patients (10 PSP-RS, 5 CBS), all of whom will be treated with BRAVYL for 48 weeks.