AMX0035, from Amylyx, is a combination of two compounds, sodium phenylbutyrate and tauroursodeoxycholic acid, that work together to maximize cellular mechanisms linked to cell death in amyotrophic lateral sclerosis, or ALS.

The first patient already has been dosed in a Phase 2 clinical trial assessing AMX0035’s safety, tolerability, and effectiveness in treating ALS. Researchers expect this study to provide new insights into ALS.

How AMX0035 works

AMX0035 is designed to reduce nerve cell death by blocking key cellular death pathways that originate in the mitochondria and endoplasmic reticulum.

Mithocondrial and endoplasmic reticulum stress are key mediators of both nerve cell death and neuroinflammatory processes. Mitochondria are the energy production centers of the cell, and the endoplasmic reticulum can be thought of as the “quality control center.” They are in constant communication and are vital for cell survival. When one of the two processes breaks down, the resulting stress can kill the cell or create inflammation. Brain cells are particularly sensitive to this stress, leading to neurodegenerative diseases such as ALS.

AMX0035 targets both the mitochondria and endoplasmic reticulum simultaneously by blocking stress and creating a balance between them.

AMX0035 research

Both elements of AMX0035, sodium phenylbutyrate, and tauroursodeoxycholic acid, have been used clinically and were shown to be safe and to demonstrate preliminary signs of effectiveness in laboratory and animal studies.

A preclinical study has shown showed that AMX0035 limited cell death and neurotoxic inflammation in different ALS models.

The Phase 2 CENTAUR study (NCT03127514) is currently recruiting participants to assess the safety and effectiveness of AMX0035 for the treatment of ALS patients. Primary objectives include the measure of decline in function according to the ALS functional rating scale, the incidence of adverse events, and the number of participants in each group who are able to remain on treatment until planned discontinuation. Secondary objectives include changes in muscle strength, blood and imaging-based biomarkers, slow vital capacity, survival, need for tracheostomy, and hospitalizations during the study.

The trial is estimated to end by May 2019 and expects to include 132 participants with ALS.


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