ALBRIOZA Commercial Launch
Progressive Supranuclear Palsy (PSP) Selected as Next Indication for AMX0035
Clear unmet need
Typically fatal within just 5 to 8 years; no disease-modifying treatments for PSP
Strong scientific rationale
PSP leads to rapid & significant neurodegeneration and is characterized by tau protein deposition in affected regions of
brain; AMX0035 shown preclinically to protect neurons against degeneration and clinically to lower tau
Biomarker evidence
AMX0035 demonstrated a statistically significant lowering of phosphotau181 & total tau in the CSF of people with
Alzheimer's disease
Existing and robust understanding of the natural history of the disease
PSP progression is predictable and well understood
Potential to move directly into a Phase 3 pivotal trial
Preparing for Phase 3 study with ~600 adults in a randomized, placebo-controlled study
Adjacencies and synergies with ALS
Shares mechanistic characteristics with ALS - unfolded protein response, mitochondrial dysfunction and related cell death
Interest and support from KOLS and advocacy groups
Collaborated with key global academic leaders, people living with PSP, and advocacy groups
AMYLYX 29View entire presentation