Allyx Therapeutics Advances ALX-001 to Patient Trials After Positive Phase 1b Results

Allyx Therapeutics, a clinical-stage biotechnology firm, has declared that its lead drug candidate, ALX-001, is moving forward to Phase 2 clinical trials for Alzheimer's and Parkinson's diseases. This decision follows the positive results from a Phase 1b study showcased at the AD/PD™ 2024 Conference in Lisbon. ALX-001 is a novel, oral, synapse-targeted treatment developed for neurodegenerative disorders.

The Phase 1b study (NCT05804383) evaluated the safety, tolerability, and pharmacokinetics of ALX-001 administered twice daily to 32 healthy adults aged 50-80. The findings indicated that the drug was safe across all dosages tested, ranging from 50mg to 150mg, with two dosages—50mg and 100mg—being selected for further research. ALX-001 demonstrated high exposure levels and a broad safety margin without any serious adverse events, highlighting its potential as a disease-modifying therapy.

Stephen Strittmatter, M.D., Ph.D., Allyx's scientific founder and a professor at Yale University School of Medicine, stated that the drug's high target engagement without mGluR5-related adverse events aligns with their strategy to address synaptic dysfunction and loss without the toxicity associated with other treatments.

With a foundation of twelve years of clinical research, ALX-001 continues to show promise. Allyx has commenced a 28-day safety study in Alzheimer's patients (NCT05804383) and is preparing to start a similar study for Parkinson's patients. Tim Siegert, Ph.D., COO and co-founder of Allyx, expressed enthusiasm for the initiation of these patient studies, which will be the first to involve ALX-001 in Alzheimer's and Parkinson's patients.

The ALX-001 program has garnered over $20 million in grants from prestigious institutions, including the National Institutes of Health, the Small Business Innovation Research (SBIR) programs, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research. Allyx has also expanded its focus to include Parkinson's disease research, with an Investigational New Drug Application filed with the FDA.

ALX-001, previously known as BMS-984923, is a first-in-class compound that selectively modulates mGluR5, a receptor crucial for synaptic function. It has a broad therapeutic window and is orally bioavailable, with the ability to penetrate the brain and selectively engage mGluR5. The molecule was originally identified by Bristol Myers Squibb and later acquired by Allyx through an exclusive worldwide license from both Bristol Myers Squibb and Yale University.

Founded in 2019, Allyx Therapeutics is dedicated to developing innovative treatments for neurodegenerative diseases, with ALX-001 as its lead compound. The company's mission is to preserve and protect synapses for those affected by such conditions.