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 New England Journal of Medicine Publication of Pivotal AMX0035 Data Demonstrating Statistically Significant Benefit in People with ALSSeptember 2, 2020

- Patients Retained Function Longer on AMX0035 Versus Placebo; Study Achieved Its Primary Outcome of a Difference on the Revised ALS Functional Rating Scale - AMX0035 is the First Investigational Therapy to Demonstrate Statistically Significant Benefit on this Prespecified Primary Outcome in People with ALS Since Approved Therapy Edaravone - AMX0035 Showed Numerical Benefits on Secondary Outcomes Including Measures of Muscle Strength, Breathing, and Hospitalizations - AMX0035 Was Generally Well Tolerated with Similar Rates of Adverse Events Recorded in the AMX0035 and Placebo Groups 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 the publication of results from the pivotal CENTAUR trial evaluating AMX0035 – an investigational neuroprotective therapy designed to reduce the death and dysfunction of motor neurons – in people with

Read More
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.

More Information

Amylyx was built on the foundation of audacious curiosity, relentless drive, and meaningful relationships. If that sounds like you, we’d love to talk.

Open Positions