New Phase 1a Clinical Data Presented on October 27 at the 16th Clinical Trials on Alzheimer’s Disease Meeting Support Continued Clinical Development

NEW HAVEN, CT, USA I October 30, 2023 I Allyx Therapeutics, a clinical-stage biotechnology company which is developing ALX-001, a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy for neurodegenerative diseases, presented Phase 1a single ascending dose study results at the 16th Clinical Trials on Alzheimer’s Disease (CTAD) meeting in Boston on October 27, 2023. The study showed that all doses of ALX-001 were well tolerated, up to and including levels high enough to achieve full brain target engagement, supporting the company’s plans to continue clinical development for the treatment of Alzheimer’s disease.

This phase 1a open-label study evaluated the safety, tolerability, pharmacokinetics, and brain receptor occupancy of increasing doses of orally-administered ALX-001. Single ascending doses were administered in 36 healthy adults between 50 and 80 years old with normal cognition. Safety findings were also encouraging. All doses of ALX-001 were well-tolerated and there were no serious adverse events. The data were presented by principal investigator Adam Mecca, M.D., Ph.D. of the Yale School of Medicine Alzheimer’s Disease Research Unit. The study was conducted at Yale and supported with grant funding from the National Institutes of Health and the Alzheimer’s Association.

“The unique mechanism of action of ALX-001 at mGluR5 holds great promise as a novel approach to preserve and protect synapses for people living with neurodegenerative diseases,” commented Tim Siegert, Ph.D., chief operating officer and co-founder of Allyx Therapeutics. “We are thrilled to observe the clinical community’s response to our initial clinical data as we work to deliver a first-in-class oral therapy for people living with Alzheimer’s disease.”

ALX-001 continues to demonstrate promise in ongoing studies that build on twelve years of research. Clinical research of ALX-001 in Alzheimer’s disease is advancing, with a Phase 1 multiple ascending dose study in healthy volunteers expected to finish in Q4 2023.

About ALX-001

ALX-001 (previously BMS-984923) is a silent allosteric modulator of mGluR5, and is a first-in-class compound that selectively blocks the pathogenic activation of the receptor while preserving the normal physiological glutamate signaling that is required for cognition. As such, ALX-001 has a wide therapeutic window that can saturate receptors while avoiding on-target toxicity observed with negative allosteric modulators. mGluR5 has been shown to be essential for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, and as such, presents a novel approach for treating Alzheimer’s and Parkinson’s disease. Importantly, ALX-001 is an orally bioavailable and brain penetrant small molecule with demonstrated mGluR5 selective engagement. The molecule was originally identified by Bristol Myers Squibb, but the mechanism of action for neurodegenerative diseases and the identification of ALX-001 as disease-modifying for Alzheimer’s disease was discovered by Allyx’s scientific founder Professor Strittmatter at Yale. Preclinical activities in Parkinson’s disease and other neurodegenerative diseases characterized by extracellular soluble protein aggregates are ongoing. Allyx Therapeutics obtained an exclusive worldwide license for use of ALX-001 from Bristol Myers Squibb and Yale University.

About Allyx Therapeutics

Allyx Therapeutics was founded in 2019 by a group of seasoned biopharma industry executives, venture capitalists, and scientific experts. The company 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 currently in clinical development in Alzheimer’s disease. Learn more at

SOURCE: Allyx Therapeutics