Trial supports neurodegenerative disease-modifying drug


Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases.

The findings were presented at the AD/PD 2024 Conference in Lisbon.

The Phase Ib study 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 characterised 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, scientific founder of Allyx Therapeutics and Professor and Chair of Neuroscience at the Yale University School of Medicine.

Moving to Phase II studies

Allyx Therapeutics has initiated a 28-day safety study of ALX-001 in Alzheimer’s disease patients 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, Chief Operating Officer and Co-Founder of Allyx Therapeutics. “We believe that the results from the Phase Ib MAD study directly support larger scale Phase II 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 programme 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.

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 US Food and Drug Administration (FDA).

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