ASHA-624

ASHA-624 is a first-in-class, novel intra-molecular glue small molecule designed to inhibit SARM1 (sterile alpha and TIR motif containing 1), a key protein that drives axonal degeneration and neurodegeneration. Developed by Asha Therapeutics using their proprietary PRISM™ drug design technology, ASHA-624 works by selectively “gluing” two regions of the SARM1 protein together to lock it in an inactive conformation. This prevents SARM1 activation and thereby blocks axon loss and neuronal degeneration—a mechanism distinct from other less selective SARM1 inhibitors. Preclinical studies have shown robust neuroprotection with reversal of motor impairment in models of ALS (amyotrophic lateral sclerosis) as well as promising results for other neurodegenerative diseases such as Charcot-Marie-Tooth disease (CMT2A), multiple sclerosis, chemotherapy-induced peripheral neuropathy (CIPN), glaucoma, spinal cord injury, traumatic brain injury (TBI), and autosomal dominant optic atrophy (ADOA). The compound is advancing toward first-in-human clinical trials for ALS and ADOA[1][2][3][4][5][6][7][8].