Allyx Therapeutics to Present Phase 1 Data for Lead Compound ALX-001 at the 16th Clinical Trials on Alzheimer's Disease Meeting

Allyx’s lead compound, ALX-001, is a small molecule with unique mechanism of action at mGluR5, to protect and preserve synapses in neurodegenerative diseases

New Clinical Data to be Presented on October 27

NEW HAVEN, Conn., Oct. 19, 2023 (GLOBE NEWSWIRE) -- 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, today disclosed its plans to present the Phase 1 single ascending dose study results at the 16th Clinical Trials on Alzheimer's Disease (CTAD) meeting in Boston on October 27, 2023.

ALX-001 continues to demonstrate promise in ongoing studies that build on twelve years of research. The results are from a single ascending dose study in healthy older adults which was conducted at Yale University and supported with grant funding from the National Institutes of Health and the Alzheimer’s Association. The data will be presented by principal investigator Adam Mecca, M.D., Ph.D. This study evaluated the safety, tolerability, pharmacokinetics, and brain receptor occupancy of increasing doses of orally-administered ALX-001.

“We are excited to share these data, which build on strong preclinical evidence for this promising therapy,” commented principal investigator Adam Mecca, M.D., Ph.D. of the Yale School of Medicine Alzheimer’s Disease Research Unit. “This study leveraged Yale’s expertise in mGluR5 PET imaging, which was used to evaluate receptor occupancy and illustrate the compound’s ability to cross the blood-brain barrier and engage the therapeutic target.”

Allyx Therapeutics continues to advance clinical research of ALX-001 in Alzheimer’s disease, with its Phase 1 multiple ascending dose study in healthy volunteers expected to finish in Q4 2023.

“Our quest for meaningul, disease-modifying therapies for people living with Alzheimer’s disease continues as we pursue this novel approach which targets neurons and acts independently of amyloid beta removal,” commented Stephen Strittmatter, M.D., Ph.D., scientific founder of Allyx Therapeutics and professor and chair of Neuroscience at Yale School of Medicine. “The potential for ALX-001 as a first-in-class oral therapy for neurodegenerative disease continues to be reinforced by a growing body of clinical evidence.”

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 Meyers 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


Media: Eliza Schleifstein

Investors: Tim Siegert