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Our path to progress

CENTAUR (NCT03127514) was a Phase 2 multicenter trial in adults with amyotrophic lateral sclerosis (ALS), encompassing both a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. This trial included 137 participants in the U.S. and evaluated safety and efficacy of AMX0035 — a fixed-dose coformulation of 2 active compounds, sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine). The trial completed in 2019 and showed positive results.1

The primary objectives of the randomized phase of CENTAUR 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, lung vital capacity and biomarkers of neuronal degeneration, were also assessed in CENTAUR.1

The CENTAUR trial enrolled participants between 18 and 80 years old with definite ALS (as described by revised El Escorial criteria) — and within 18 months of symptom onset — and allowed edaravone and/or riluzole use.1

Open Label Extension (OLE) Phase

Participants who completed the CENTAUR randomized phase were eligible to enroll in an OLE phase to assess the longer-term safety and therapeutic potential of AMX0035. Of the participants who completed the randomized phase, 92% opted to enter the OLE phase.2

  1. Paganoni S, et al. N Engl J Med. 2020;383(10):919-930.
  2. Paganoni S, et al. Muscle Nerve. 2021;63(1):31-39.

Randomized phase

Graph displaying the ALS Functional Rating Scale (ALSFRS-R) total at week 24 in the CENTAUR trial

Randomized phase

Conducted by investigators at 25 top ALS medical centers throughout the Northeast ALS (NEALS) consortium, the CENTAUR trial completed in late 2019. The trial met its primary efficacy endpoint—demonstrating statistically significant functional benefits for people living with ALS, based on the ALS Functional Rating Scale (ALSFRS-R).1

In the CENTAUR trial, participants experienced similar rates of adverse events in the AMX0035 and placebo groups; however, GI events occurred with greater frequency (≥2%) in the AMX0035 group.1

Overall survival results

Centaur Video Thumbnail

Watch the video to learn more about the results of the CENTAUR trial from Machelle Manuel, PhD, Global Head Medical Affairs at Amylyx.

Overall survival results

In an interim survival analysis conducted in all randomized participants from CENTAUR who were followed for up to three years, which included participants who continued to receive AMX0035 in an open-label extension phase during the follow-up period, participants who started on AMX0035 during the placebo-controlled phase of CENTAUR showed a 44% lower risk of death compared to those who started on placebo during the placebo-controlled phase (HR 0.56; 95% CI, 0.34-0.92). Median survival duration through the open-label long-term follow-up phase was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference.2

  1. Paganoni S, et al. N Engl J Med. 2020;383(10):919-930
  2. Paganoni S, et al. Muscle Nerve. 2021;63(1):31-39

An ALS Community Collaboration

Map of the United States displaying locations of the CENTAUR trial

An ALS Community Collaboration

The CENTAUR trial was an ALS community collaboration, with trial sites, organizations, and participants partnering to execute this trial, such as the Northeast ALS (NEALS) consortium, Barrow Neurological Institute, ALS Association, and ALS Finding A Cure. The trial was conducted at 25 NEALS centers in the United States.

AMX0035

Zoomed in microscopic view of motor neuron

AMX0035

We are currently exploring the potential of our investigational therapy, AMX0035 (sodium phenylbutyrate and taurursodiol), for the treatment of ALS, Alzheimer’s disease, and Wolfram syndrome. Our aim is to expand upon this innovation to address other neurodegenerative diseases.

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Our commitment

At Amylyx, we are relentlessly driven by a commitment to those living with neurodegenerative diseases. We dedicate every passing minute to developing potential therapies that will give them more quality time with friends, family, and loved ones.

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Clinical trials

Our innovative approach to developing transformative therapeutics has far-reaching potential to improve the management of multiple neurodegenerative diseases. Learn more about our clinical trials.

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