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Charting new territories

Charting new territories

We are committed to advancing science and innovation for the broader industry to open new pathways and approaches to developing treatments. We strategically diversify our pipeline as we find the right opportunity to make an impact. We’ve strategically evaluated hundreds of assets over the past few years and only pursue a potential therapy if it meets our scientific criteria, aligns with our expertise, and could meet an unmet need.

Venn diagram of Amylyx' R&D strategy. "Mechanism of Action" and "Mechanism of Disease" overlap as "Area for Amylyx Impact."

Overview of Amylyx’ pipeline

We are advancing a pipeline in which we’ve matched investigational therapies with diseases in which they can make the greatest impact, based on well-defined mechanistic rationale, clear clinical outcomes and biomarkers, and rigorous preclinical data.

We are currently developing three investigational therapies for potential impact across several diseases: avexitide in post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI), AMX0035 in Wolfram syndrome and progressive supranuclear palsy (PSP), and AMX0114 as part of our continued commitment to amyotrophic lateral sclerosis (ALS).

Staying modality-agnostic allows us to apply the right modality to the diseases we’re focused on:

  • Avexitide is a first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist.
  • AMX0035 is an oral, fixed-dose combination of two small molecules, sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.).
  • AMX0114 is our antisense oligonucleotide (ASO) targeting calpain-2.

Curiosity fuels our internal discovery engine as well as our clinical progress. We are tackling several of the industry’s unanswered questions, one data point at a time, to develop a deeper understanding of some of the scientific pathways driving our diseases of focus, as well as studying additional modalities to make targeting those pathways possible. Learn more about our growing pipeline and see the statuses of our clinical trials.

How to get in touch

If you are a researcher or biotech company looking for a partner in your mission, we would love to hear from you about your work and to consider the various ways in which we could partner together.