Allyx Therapeutics Announces First Alzheimer’s Disease Patient Treated with Lead Compound ALX-001

With Growing Body of Clinical Evidence, ALX-001 is Ready to Commence Phase 2 Studies

NEW HAVEN, Conn., June 11, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics, a clinical-stage biotechnology company working to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases, announced that the first Alzheimer’s disease patient has been dosed with its lead compound, ALX-001, in a clinical study. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy that is ready for Phase 2 clinical development.

“Administering the first dose in patients is a watershed moment for a new therapeutic, and we are pleased to begin research in Alzheimer’s disease patients as we continue to advance clinical development of ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases,” commented Stephen Bloch, M.D., chief executive officer and co-founder of Allyx Therapeutics.

Individuals with Alzheimer’s disease are being enrolled in a 28-day study which will evaluate the safety of ALX-001 dosed twice daily at either 50 mg or 100 mg versus placebo in adults between 50 and 80 years of age. This is the second phase of an ongoing study which previously assessed multiple ascending doses in healthy volunteers (NCT05804383) and is being conducted at Yale School of Medicine with support from the National Institute on Aging.

“Every neuroscientist hopes for the moment that an asset they believe in and fight for advances to the critical stage of testing in patients,” stated Stephen Strittmatter M.D., Ph.D., scientific founder of Allyx Therapeutics and professor and chair of Neuroscience at the Yale School of Medicine. “The unique mechanism of action of ALX-001 and its promising early data have brought us to this point, and bolster our hope that it has the potential to be a difference-maker in Alzheimer’s disease.”

ALX-001 is ready to proceed to phase 2 clinical development, and is not restricted by completion of ongoing studies. Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in studies supported by highly reputable third parties. The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the U.S. Government’s highly competitive Small Business Innovation Research (SBIR) programs, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research, among others.

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 scientific founder Stephen Strittmatter at Yale School of Medicine. Allyx Therapeutics obtained an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale School of Medicine.

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 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at


Media: Eliza Schleifstein

Investors: Tim Siegert