Allyx Therapeutics Announces Positive Phase 1b Multiple Ascending Dose Data and Advances ALX-001 to Patients

2024-03-11

临床1期引进/卖出临床结果

Safety of ALX-001 Reinforced by New Data Presented at AD/PD™ 2024 Conference in Lisbon

Two Oral Doses Selected for Further Study in Alzheimer’s and Parkinson’s Disease Patients

NEW HAVEN, Conn., March 11, 2024 (GLOBE NEWSWIRE) -- Allyx Therapeutics, a clinical-stage biotechnology company, announced that its lead compound, ALX-001, is ready to proceed to Phase 2 clinical development in Alzheimer’s and Parkinson’s disease. This announcement was made in conjunction with the presentation of positive findings from the company’s Phase 1b multiple ascending dose study at the AD/PD™ 2024 Conference in Lisbon. ALX-001 is a highly selective, first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases.

The multiple ascending dose study (NCT05804383) examined the safety, tolerability and pharmacokinetics of twice-daily oral doses of ALX-001 in 32 heathy adult participants aged 50-80. Study results show that ALX-001 was safe in cognitively normal older adults at all tested doses. ALX-001 achieved high exposure and successfully characterized a wide safety window at all doses, which ranged from 50mg to 150mg. Two doses were identified for future studies: 50mg and 100mg. All doses were well-tolerated and there were no serious adverse events.

“The data show that ALX-001 achieved high target engagement without any adverse events related to mGluR5, which supports our vision to mediate synaptic dysfunction and loss while avoiding the on-target toxicity observed with other treatment modalities,” commented Stephen Strittmatter, M.D., Ph.D., scientific founder of Allyx Therapeutics and professor and chair of Neuroscience at the Yale University School of Medicine.

Building on twelve years of clinical research, ALX-001 continues to demonstrate promise in ongoing studies. Allyx Therapeutics has initiated a 28-day safety study of ALX-001 in Alzheimer’s disease patients (NCT05804383) and is initiating a study in Parkinson’s disease patients.

“We are excited to begin the next phase of development with the initiation of two 28-day patient studies, the first studies of ALX-001 in people living with Alzheimer’s and Parkinson’s disease,” commented Tim Siegert, Ph.D., chief operating officer and co-founder of Allyx Therapeutics. “We believe that the results from the Phase 1b MAD study directly support larger scale Phase 2 clinical development to more fully understand the potential for ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases.”

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. In addition to Allyx’s continued clinical development of ALX-001 in Alzheimer’s disease, the company has announced an expansion into Parkinson’s disease clinical research with the opening of an Investigational New Drug Application with the U.S. Food and Drug Administration (FDA).

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. Allyx Therapeutics obtained an exclusive worldwide license for 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 in clinical development for Alzheimer’s disease and Parkinson’s disease. Learn more at allyxthera.com.

Contact

Media: Eliza Schleifstein

917.763.8106

Eliza@schleifsteinpr.com

Investors: Tim Siegert

203-691-6543

tsiegert@allyxthera.com