ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the physiological glutamate signaling that is required for normal cognition. ALX-001 has a wide therapeutic window that can saturate receptor binding while avoiding on-target toxicity observed with negative allosteric modulators. Preclinically, mGluR5 has been shown to be essential for mediating synaptic function and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating neurodegenerative diseases. ALX-001 is an orally bioavailable and brain penetrant small molecule hitting a druggable target.

The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases was discovered by Allyx scientific founder and Yale University professor Stephen Strittmatter, M.D., Ph.D. Allyx Therapeutics obtained an exclusive worldwide license for use of ALX-001 from Bristol Meyers Squibb and Yale University.