Amyotrophic Lateral Sclerosis (ALS) PH. 2
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 product — an oral fixed-dose co-formulation of two active compounds, sodium phenylbutyrate (PB) and taurursodiol (TURSO) — optimized to address both the toxic, unfolded proteins in the endoplasmic reticulum and the energy crisis in the mitochondria that occur in neurodegenerative diseases. We are currently exploring the potential of AMX0035 as a treatment option for ALS, Alzheimer’s disease and Wolfram syndrome. Our aim is to expand upon this innovation to address other neurodegenerative diseases.

Clinical Trials


CENTAUR was a 24-week, randomized, double-blind, placebo-controlled Phase II trial that evaluated AMX0035 in people with ALS. The primary objectives of the study were to assess the safety and tolerability of AMX0035 as well as the impact of AMX0035 on disease progression, as measured by the Revised ALS Functional Rating Scale (ALSFRS-R). Additional measures that are critical to people with ALS, including muscle strength and slow vital capacity, were also assessed in CENTAUR.

The CENTAUR study enrolled participants 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 functional benefits for people with ALS, based on the ALS Functional Rating Scale (ALSFRS-R). Of the participants who completed CENTAUR, 92% elected to enroll in an open-label extension study. In the CENTAUR trial, participants experienced similar rates of adverse events in the AMX0035 and placebo groups.

The importance of moving forward as efficiently as possible is not something we take lightly, as people living with ALS don’t have time to wait. We are in discussions with regulatory authorities in the United States and other territories to move AMX0035 through the clinical development process as quickly as possible.


Recognizing that AMX0035 has potential with other neurodegenerative diseases, Amylyx initiated the PEGASUS trial in participants with late mild cognitive impairment (MCI) or dementia due to Alzheimer’s disease in 2018. The Phase II PEGASUS trial is evaluating the safety, tolerability and biological activity of AMX0035 in 96 participants over 24 weeks of treatment. PEGASUS was supported by the Alzheimer’s Combination Therapy Opportunities (ACTO) program, a joint research funding initiative supported by the Alzheimer’s Association and the Alzheimer’s Drug Discovery Foundation, and Cure Alzheimer’s Fund. Top-line results of the PEGASUS trial are expected to be available in 2021.

The primary purpose of the trial is to investigate the safety and tolerability of AMX0035 in participants with Alzheimer’s disease, however, the inclusion of trial endpoints such as cognitive function, brain imaging and spinal fluid biomarkers will provide an informative dataset that will allow for evaluation and correlation of imaging-based markers, neurobiological changes, functional measures, and cognitive outcomes in Alzheimer’s disease.

Latest News

Amylyx Supports Rare Disease Day® and #ShowYourStripes Campaign to Raise Awareness for Rare Disease CommunitiesFebruary 22, 2021

#ShowYourStripes In Community Solidarity by Using New Facebook Filter or Downloadable Video Conferencing Background Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, has joined forces with the National Organization for Rare Disorders (NORD®) to help raise awareness for this year’s Rare Disease Day® on February 28 th . Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases and emphasizing the special challenges the community faces. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210222005597/en/ #ShowYourStripes Video Conferencing Background Available for Download (Graphic: Business Wire) Amylyx is honored to be a Rare Disease Day “Campaign Supporter” and participate in NORD’s #ShowYourStripes campaign, which aims to empower advocates,

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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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