Allyx Therapeutics Announces First Parkinson’s Disease Patient Treated

Allyx Therapeutics recently announced the first patient has been dosed with its lead compound, ALX-001, in a new clinical study assessing safety, pharmacokinetics and potential therapeutic response in patients with Parkinson’s disease. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy. Allyx Therapeutics is a clinical-stage biotechnology company working to deliver a novel approach to preserve and protect synapses for people living with neurodegenerative diseases.

“Allyx Therapeutics is moving forward with strong momentum to develop ALX-001 as the first-ever disease-modifying small molecule for neurodegenerative diseases, with two concurrent safety studies in patients with Parkinson’s disease and with Alzheimer’s disease, adding important information to the body of knowledge for this novel therapeutic approach,” said Tim Siegert, PhD, Chief Operating Officer and co-founder of Allyx Therapeutics.

The 28-day Parkinson’s disease study (NCT06309147) is assessing the safety of ALX-001 dosed twice daily at either 50 mg or 100 mg versus placebo in adults between 21 and 80 years of age, and will also investigate dopamine transporter levels in the brain measured with single photon emission computed tomography as an early marker of therapeutic response to a treatment that targets synapse restoration. The study is being conducted by the Duke Clinical Research Institute (DCRI) and is supported with grant funding awarded to Allyx Therapeutics from The Michael J. Fox Foundation for Parkinson’s Research.

“I’m excited to collaborate with the Allyx team as we advance understanding of how Parkinson’s disease affects the brain and what more we might be able to do to provide a real impact for patients. Investigating the safety of this disease-modifying small molecule is a crucial first step, and one I’m proud to be a part of,” said Laurie H. Sanders, PhD, Associate Professor of Neurology at Duke University School of Medicine and DCRI.

Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in ongoing studies. The ALX-001 program has received more than $20 million in grant funding from the National Institutes of Health, the US 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.

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.

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 allyxthera.com.

The DCRI, part of the Duke University School of Medicine, is the largest academic clinical research organization in the world. Our mission is to develop, share, and implement knowledge that improves global health through innovative clinical research. The institute conducts multinational clinical trials, manages major national patient registries, and performs landmark outcomes research. The DCRI is a pioneer in cardiovascular and pediatric clinical research and conducts groundbreaking clinical research across multiple other therapeutic areas, including infectious disease, neuroscience, respiratory medicine, and nephrology.