ALX-001 is a first-in-class, orally bioavailable small molecule being developed as a disease-modifying treatment for neurodegenerative diseases. It functions as a silent allosteric modulator of the metabotropic glutamate receptor 5 (mGluR5). Its multi-targeted mechanism works by inhibiting amyloid beta oligomer toxicity, reducing tau hyperphosphorylation, and preventing microglial-mediated synaptic pruning, addressing key drivers of cognitive decline. The molecule was originally identified by Bristol Myers Squibb, and its potential for neurodegenerative conditions was discovered at Yale School of Medicine.