AS2015

AS2015 is an early-stage, preclinical small-molecule drug candidate being developed by AcuraStem as part of a PIKFYVE inhibitor research program for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) caused by C9orf72 repeat expansions.[3][5][9][11] The program is based on patient-derived cellular models to identify compounds that modulate PIKFYVE, a lipid kinase involved in endolysosomal trafficking, with the goal of correcting disease-relevant cellular phenotypes in genetic ALS/FTD.[5][9][11] Detailed mechanistic, pharmacologic, and clinical data for AS2015 itself have not yet been publicly disclosed, and the asset remains in discovery/early development without registered clinical trials.