Our Science

Allyx Therapeutics aims to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases. Its lead compound, ALX-001, is a first-in-class oral therapy with a unique mechanism of action at mGluR5. Clinical programs are currently underway in Alzheimer’s disease (Phase 1b).

About ALX-001

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.

Our Studies

Evaluating the Safety, Tolerabillity, Pharmacokinetics and Receptor Occupancy of ALX-001.

Food Effect Study of ALX-001 in Healthy Older Adults.

A Multiple Ascending Dose Study in Healthy Volunteers and Patients with Alzheimer’s Disease.


Reversal of synapse loss in Alzheimer mouse models by targeting mGluR5 to prevent synaptic tagging by C1Q

June 2022

Metabotropic Glutamate Receptor 5 Is a Coreceptor for Alzheimer Aβ Oligomer Bound to Cellular Prion Protein

September 2013

Metabotropic Glutamate Receptor 5 Couples Cellular Prion Protein to Intracellular Signalling in Alzheimer’s Disease

February 2016

Silent Allosteric Modulation of mGluR5 Maintains Glutamate Signaling while Rescuing Alzheimer’s Mouse Phenotypes

July 2017