YA-101

YA-101 is a novel small molecule drug developed by Yoda Pharmaceuticals (also referred to as Yoda Therapeutics) for the treatment of neurodegenerative diseases, primarily multiple system atrophy (MSA). It acts as an inhibitor of both GluN1 (glutamate ionotropic receptor NMDA type subunit 1) and NLRP3 (NACHT, LRR and PYD domains-containing protein 3), targeting neuroinflammation and modulating neuronal plasticity. The drug has orphan designation in the United States for MSA. Preclinical studies have shown that it improves motor-related behavioral impairments in animal models. As of early 2025, it is undergoing Phase II clinical trials in the US and Taiwan for MSA. Additional preclinical development has been reported for Alzheimer’s disease and schizophrenia[1][4][6].