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]^
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]^
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]^
AMX0035 is Amylyx's lead candidate, a combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodiol). The drug is designed to target mitochondrial dysfunction and endoplasmic reticulum stress, both implicated in the pathogenesis of ALS and other neurodegenerative diseases[@fda2022].
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
Mechanism of Action: The combination targets two key pathways:
Mitochondrial dysfunction: TURSO acts as a mitochondrial uncoupler, reducing [reactive oxygen species](/entities/reactive-oxygen-species)
ER stress: PB is a histone deacetylase inhibitor that reduces ER stress
Expansion to Other Indications
Amylyx is exploring AMX0035 in other neurodegenerative conditions: