AS-202

AS-202 is an antisense oligonucleotide (ASO) drug candidate developed by AcuraStem for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). It works by suppressing the expression of PIKFYVE, a lipid kinase involved in regulating multivesicular body exocytosis, which is essential for clearing misfolded proteins such as TDP-43, C9ORF72 dipeptide repeat proteins, and tau from neurons. By inhibiting PIKFYVE activity, AS-202 promotes exosomal secretion to robustly clear these toxic protein aggregates that are implicated in neurodegeneration. The drug is administered intrathecally to target the central nervous system directly and avoid systemic toxicity. Preclinical studies have shown that AS-202 reduces neurodegeneration, restores motor function, improves survival in animal models of ALS and FTD, and effectively lowers PIKFYVE levels in patient-derived motor neurons[1][2][4][5][8].