AT-001 (Arctic Therapeutics)

AT-001 is an orally administered small molecule developed by Arctic Therapeutics for the treatment of neurodegenerative protein aggregation disorders, including hereditary cystatin C amyloid angiopathy (HCCAA) and Alzheimer's disease. The drug's mechanism of action involves destabilizing the inter- and intra-molecular interactions that lead to the formation and maintenance of toxic amyloid plaques and tau tangles. By preventing and dissolving these aggregates, AT-001 aims to modify the course of diseases where toxic oligomers drive neurodegeneration. It has received Orphan Drug Designation from the EMA for HCCAA and is currently being evaluated in Phase 2 clinical trials for both its primary indication of HCCAA and for Alzheimer's disease.