NST0019

NST0019 is an experimental small molecule therapeutic being developed by Nervosave Therapeutics for the treatment of neurodegenerative diseases, primarily Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). The compound specifically targets TDP-43 proteinopathy, a pathological hallmark found in approximately 97% of ALS cases and 50% of FTD cases. NST0019 is designed to promote the clearance of toxic TAR DNA-binding protein 43 (TDP-43) aggregates and prevent their accumulation in neurons, thereby mitigating neurotoxicity and preserving motor function. As of 2024, the program is in the preclinical stage of development, having originated from research at the National University of Singapore.