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]
| Program | Target/Mechanism | Indication | Phase | Status |
|---|---|---|---|---|
| Relyvrio (AMX0035) | SOD1 reducer / mitochondrial dysfunction | ALS | Approved (2022) | Marketed |
| AMX0035 | SOD1 reducer / mitochondrial dysfunction | Alzheimer's Disease | Phase 2 (CENTAUR) | Completed |
| AMX0035 | SOD1 reducer | Progressive Supranuclear Palsy | Phase 2 | Planning |
Amylyx's platform focuses on:
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[1].
Clinical Development:
Mechanism of Action:
The combination targets two key pathways:
Amylyx is exploring AMX0035 in other neurodegenerative conditions: