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A new approach to serious neurodegenerative diseases

AMX0035 is an investigational therapy—an oral coformulation of 2 active compounds, sodium phenylbutyrate and taurursodiol. Amylyx is currently exploring the potential of AMX0035 as a treatment option for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Wolfram syndrome.

* Also known as Ursodoxicoltaurine.

Multipathway coformulation

Graphic displaying the combination of PB and TURSO and its impact on Cortical Neuronal Viability

Multipathway coformulation

The multipathway and complex nature of neurodegenerative diseases may ultimately be successfully treated by a combination of therapies simultaneously targeting multiple destructive pathways.1,2

  • AMX0035 is proposed to reduce neuronal death by simultaneously mitigating endoplasmic reticulum stress and mitochondrial dysfunction.

In a preclinical primary cortical neuron damage model, AMX0035 has shown the ability to synergistically protect neurons against death.3

  • Neuron viability was synergistically increased with specific concentrations of PB and TURSO, achieving >90% peak neuronal viability.3

AMX0035 is formulated to ensure quality and optimal dosing

Key steps in manufacturing ensure that these 2 compounds are combined properly to maintain powder form, ensure proper absorption, and meet rigorous quality control standards; outside the AMX0035 coformulation, there is no information to support combining these 2 molecules in such a way that patient safety and product quality are maintained.

  1. Brown RH, Al-Chalabi A. N Engl J Med. 2017;377(2):162-172.
  2. Gan L, Cookson MR, Petrucelli L, La Spada AR. Converging pathways in neurodegeneration, from genetics to mechanisms. Nature Neuroscience. 2018;21(10):1300–9.
  3. Cohen J, et al. Poster presented at: 28th International Symposium for ALS/MND; December 4–10, 2017; Boston, MA.

Our Pipeline

Learn more about our pipeline and explore information about our clinical trials and progress to date

ALS clinical trials

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CENTAUR Phase 2 Study

CENTAUR (NCT03127514) was a Phase 2, randomized, placebo-controlled study in 137 participants in the U.S. that evaluated safety and efficacy of AMX0035 in ALS. The study completed in 2019 and showed positive results.

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PHOENIX Phase 3 Study

PHOENIX (NCT05021536) is a Phase 3, randomized, placebo-controlled study in approximately 65 sites across the U.S. and Europe evaluating the safety and efficacy for AMX0035 in ALS. Recruiting for this trial is ongoing.

Alzheimer’s disease clinical trial

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PEGASUS Phase 2 Study

PEGASUS (NCT03533257) is a Phase 2a, randomized, placebo-controlled study in 95 participants in the U.S. evaluating safety, tolerability, neurobiological activity, and preliminary clinical efficacy of AMX0035 in a broad range of participants with dementia or mild cognitive impairment due to Alzheimer’s disease.