AL001 – Amyotrophic lateral sclerosis with a C9orf72 mutation – Phase 2
We are conducting a randomized, controlled Phase 2 clinical study of AL001 in people with amyotrophic lateral sclerosis (ALS) who carry a C9orf72 mutation.
Excess aggregation of TDP-43 in brain cells is thought to lead to neuronal cell death and is associated with multiple neurodegenerative diseases. Increasing progranulin levels has been shown to reverse and be protective against TDP-43 pathology in multiple models of neurodegeneration, including ALS, where 95% of patients with ALS have TDP-43 pathology.
ALS is the first of several potential indications beyond frontotemporal dementia where we believe AL001 may have a positive impact on brain health by elevating progranulin levels.