Amyotrophic Lateral Sclerosis (ALS) PH. 2/3
Alzheimer’s Disease PH. 2
Wolfram Syndrome
Preclinical ValidationPreclinical Validation
IND Enabling StudiesIND Enabling Studies
Phase 1Phase 1
Phase 2Phase 2
Phase 3Phase 3

About AMX0035

AMX0035 is our first-in-class investigational therapy — a fixed-dose co-formulation of two active compounds, sodium phenylbutyrate (PB) and Taurursodiol — optimized to treat both the energy crisis in the mitochondria and the toxic, unfolded proteins in the endoplasmic reticulum, ultimately disrupting the neurological chain of events that leads to patient suffering. We are currently exploring the potential of AMX0035 as a treatment option for ALS and Alzheimer’s disease patients. Our aim is to expand upon this innovation to treat other neurodegenerative diseases.

Clinical Trials


The CENTAUR trial of AMX0035 was a 24-week, randomized, double-blind, placebo-controlled Phase II/III clinical trial that assessed the drug’s safety and tolerability, impact on disease progression, and effect on other measures that are critical to people with ALS, including muscle strength, slow vital capacity, and biomarkers of neuronal degeneration.

The CENTAUR trial enrolled patients between 18 and 80 years old with definite ALS — and within 18 months of symptom onset — and allowed edaravone or riluzole use.

Conducted by investigators at 25 top ALS medical centers throughout the Northeast ALS (NEALS) consortium, the CENTAUR trial completed in late 2019. AMX0035 demonstrated statistically significant treatment benefits for people with ALS, based on the ALS Functional Rating Scale (ALSFRS-R), with 92% of participants who completed CENTAUR electing to enroll in an extension study.

The importance of moving forward as efficiently as possible is not something we take lightly, as patients living with ALS don’t have time to wait. Meanwhile, we’re working with the U.S. Food and Drug Administration and ALS community to ensure people will be able to access AMX0035 as soon as possible.


Recognizing that the drug has potential with other neurodegenerative diseases, the PEGASUS trial is underway to evaluate the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer’s disease. Thanks to additional funding from the Cure Alzheimer’s Fund, the PEGASUS trial has doubled its planned enrollment to 100 participants during a 24-week trial. Trial collaborators include the Alzheimer’s Association, the Alzheimer’s Drug Discovery Foundation (ADDF) and Cure Alzheimer’s Fund. Amylyx completed enrollment for the trial in June of 2020, dosing 96 of the previously planned 100 participants. Topline results of the PEGASUS trial will be shared in the first quarter of 2021.

The trial endpoints include cognitive function, brain imaging and spinal fluid biomarkers and will assist in demonstrating the effects of AMX0035 and its potential in treating Alzheimer’s disease.

Latest News

Amylyx Pharmaceuticals Announces FDA Granted Orphan Drug Designation for AMX0035 for the Treatment of Wolfram SyndromeNovember 16, 2020

Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035 for the treatment of Wolfram syndrome. “There is a critical unmet need for people living with Wolfram syndrome, a rare genetic disease in which many patients die prematurely with severe neurological disabilities,” said Wolfram syndrome expert Fumihiko Urano, M.D., Ph.D., a Professor of Medicine and of Pathology and Immunology at Washington University School of Medicine in St. Louis. “Endoplasmic reticulum (ER) dysfunction is a critical component of Wolfram syndrome. AMX0035 is designed to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome.” The FDA may grant

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Pegasus Alzheimer’s Trial

PEGASUS is a 3:2 randomized, double-blind, multi-center, placebo-controlled trial evaluating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer’s disease over 24 weeks.

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