Amylyx Pharmaceuticals

Also Clin2 PSP, Clin2 Wolfram syndrome, Clin2 Alzheimer's; AMX0114 Clin0 ALS, neurodegenerative diseases; undisclosed programs RD ALS, neurodegenerative diseases

Amylyx is developing therapeutics for neurodegenerative disorders. A common feature of neurodegenerative diseases is neuronal death, which can be triggered when multiple stress factors are activated. Amylyx is identifying ways to preserve neurons by understanding the stress pathways that underlie Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative disorders. AMX0035, Amylyx’s lead program, has been shown in preclinical studies to protect neurons against death in a damage model by mitigating cellular stress and mitochondrial dysfunction. Following positive Phase 2 clinical results for ALS, AMX0035 is now being investigated in Phase 3 trials. Amylyx is also testing AMX0035 in patients with Alzheimer’s disease and Wolfram Syndrome.