Amylyx Pharmaceuticals, Inc.

<div class="infobox infobox-company">
<div class="infobox-header">Amylyx Pharmaceuticals, Inc.</div>
<div class="infobox-row"><strong>Headquarters:</strong> Cambridge, Massachusetts, USA</div> [@centaur2020]
<div class="infobox-row"><strong>Founded:</strong> 2012</div>
<div class="infobox-row"><strong>Public:</strong> NASDAQ (AMLX)</div>
<div class="infobox-row"><strong>Market Cap:</strong> ~$1.2B USD</div>
</div>

Overview

Amylyx Pharmaceuticals is a pharmaceutical company focused on developing novel therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The company's lead product, Relyvrio (AMX0035), is approved in the US and Canada for the treatment of ALS, making it one of the few disease-modifying therapies available for this devastating condition.^[1]^

Pipeline / Products

| Program | Target/Mechanism | Indication | Phase | Status |
|---------|-----------------|------------|-------|--------|
| Relyvrio (AMX0035) | SOD1 reducer / mitochondrial dysfunction | ALS | Approved (2022) | Marketed |
| AMX0035 | SOD1 reducer / mitochondrial dysfunction | [Alzheimer's Disease](/diseases/alzheimers-disease) | Phase 2 (CENTAUR) | Completed |
| AMX0035 | SOD1 reducer | Progressive Supranuclear Palsy | Phase 2 | Planning |

Technology Platform

<div class="infobox infobox-company">
<div class="infobox-header">Amylyx Pharmaceuticals, Inc.</div>
<div class="infobox-row"><strong>Headquarters:</strong> Cambridge, Massachusetts, USA</div> [@centaur2020]
<div class="infobox-row"><strong>Founded:</strong> 2012</div>
<div class="infobox-row"><strong>Public:</strong> NASDAQ (AMLX)</div>
<div class="infobox-row"><strong>Market Cap:</strong> ~$1.2B USD</div>
</div>

Overview

Amylyx Pharmaceuticals is a pharmaceutical company focused on developing novel therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The company's lead product, Relyvrio (AMX0035), is approved in the US and Canada for the treatment of ALS, making it one of the few disease-modifying therapies available for this devastating condition.^[1]^

Pipeline / Products

| Program | Target/Mechanism | Indication | Phase | Status |
|---------|-----------------|------------|-------|--------|
| Relyvrio (AMX0035) | SOD1 reducer / mitochondrial dysfunction | ALS | Approved (2022) | Marketed |
| AMX0035 | SOD1 reducer / mitochondrial dysfunction | [Alzheimer's Disease](/diseases/alzheimers-disease) | Phase 2 (CENTAUR) | Completed |
| AMX0035 | SOD1 reducer | Progressive Supranuclear Palsy | Phase 2 | Planning |

Technology Platform

Amylyx's platform focuses on:

• SOD1 reduction: Targeting mutant superoxide dismutase 1 (SOD1) which accounts for ~20% of familial ALS
• Mitochondrial dysfunction: Addressing energy failure in neurodegenerative diseases
• Neuroprotection: Combined approach targeting multiple pathways
• Oral small molecules: Bioavailable compounds suitable for chronic dosing

Funding & Financials

• 2024: FDA approval and commercial launch of Relyvrio
• 2022: $100M+ from Viking Global Investors
• 2021: $70M Series C financing

Key People

• Joshua Cohen (Co-CEO & Co-Founder)
• Justin Klee (Co-CEO & Co-Founder)

Clinical Evidence

RELYVRIO Phase 3 PHOENIX Trial (2024): While the primary endpoint was not met in the larger Phase 3 trial, the drug showed sustained benefit in earlier Phase 2 CENTAUR trial.^[2]^

CENTAUR Phase 2 Trial (2020): AMX0035 showed significant benefit on the primary endpoint of Revised ALS Functional Rating Scale (ALSFRS-R) decline. Patients receiving AMX0035 had less functional decline compared to placebo over 24 weeks.^[3]^

Clinical Trial Pipeline

AMX0035 (Centrally Acting Mitochondrial Uncoupler)

Clinical Development:

• Phase II CENTAUR Trial: Demonstrated statistically significant slowing of functional decline in ALS patients (p=0.03)
• Phase III PHOENIX Trial: Currently enrolling to confirm efficacy and safety
• FDA Approval: AMX0035 received FDA approval in 2024 for treatment of ALS

Expansion to Other Indications

• [Alzheimer's Disease](/diseases/alzheimers-disease): Phase 2 trial planned
• [Parkinson's Disease](/diseases/parkinsons-disease): Preclinical data encouraging

Financial Overview

• Founded: 2013 (by Joshua Cohen and Justin Klee, Brown University alumni)
• Headquarters: Cambridge, Massachusetts
• IPO: 2022 (NASDAQ: AMLX)
• Market Cap: ~00M (as of 2026)

Partnerships and Funding

• ALS Association: Major funding partner for clinical trials
• National Institutes of Health (NIH): Grant support for preclinical research
• Venture Capital: Backed by Andreessen Horowitz, Morningside Ventures

See Also

• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Parkinson's Disease](/diseases/parkinsons-disease)

External Links

• [PubMed](https://pubmed.ncbi.nlm.nih.gov/)
• [KEGG Pathways](https://www.genome.jp/kegg/pathway.html)

References

Pathway Diagram

The following diagram shows the key molecular relationships involving Amylyx Pharmaceuticals, Inc. discovered through SciDEX knowledge graph analysis: