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AMX0035
AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol that reduces neuroinflammation and protects motor neurons by stabilizing mitochondrial function and reducing ER stress.
AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol that reduces neuroinflammation and protects motor neurons by stabilizing mitochondrial function and reducing ER stress. Used for Amyotrophic lateral sclerosis (ALS).
At a glance
| Generic name | AMX0035 |
|---|---|
| Also known as | Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO; also known as Tauroursodeoxycholic Acid [TUDCA]), Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine or Tauroursodeoxycholic Acid [TUDCA]), Proprietary formulation of taurursodiol and sodium phenylbutyrate, RELYVRIO |
| Sponsor | Amylyx Pharmaceuticals Inc. |
| Drug class | Neuroprotective agent; combination therapy |
| Target | ER stress pathway, mitochondrial stability, neuroinflammation |
| Modality | Small molecule |
| Therapeutic area | Neurology |
| Phase | Phase 3 |
Mechanism of action
The combination works through dual mechanisms: sodium phenylbutyrate acts as a nitrogen scavenger and chemical chaperone to reduce protein misfolding and ER stress, while taurursodiol is a bile acid derivative that stabilizes mitochondrial membranes and reduces apoptosis. Together, these agents target the neurodegeneration cascade in motor neuron diseases by reducing oxidative stress, neuroinflammation, and cell death pathways.
Approved indications
- Amyotrophic lateral sclerosis (ALS)
Common side effects
- Nausea
- Vomiting
- Diarrhea
- Abdominal pain
- Headache
Key clinical trials
- Open Label Extension Study of AMX0035 in Patients With ALS (PHASE2)
- AMX0035 in Adult Patients With Wolfram Syndrome (PHASE2)
- Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease (PHASE2)
- Extension Study Evaluating The Safety And Tolerability of AMX0035 (PHASE3)
- AMX0035 and Progressive Supranuclear Palsy (PHASE2, PHASE3)
- Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment (PHASE3)
- AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (PHASE2)
- Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AMX0035 CI brief — competitive landscape report
- AMX0035 updates RSS · CI watch RSS
- Amylyx Pharmaceuticals Inc. portfolio CI