ASD-005

ASD-005 is an investigational small molecule drug, developed by Asdera, designed to modulate endocytosis and lysosomal function. It is a derivative of hydroxypropyl-alpha-cyclodextrin (HP-α-cyclodextrin) and acts by down-regulating endocytosis to restore lysosomal capacity for autophagy, potentially through modulation of the PI-cycle and MTORC1 pathway. This mechanism aims to improve lysosomal function and autophagy in neurodegenerative diseases. ASD-005 has been studied for oral use as a disease-modifying treatment in multiple sclerosis (MS), with additional applications under investigation for Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and various lysosomal storage disorders. A liposomal parenteral formulation has also been explored for acute hypertension and congestive heart failure[1][2][3][4][5][6].