Last reviewed · How we verify
GM1
GM1 is a monosialotetrahexosylganglioside that promotes neuronal protection and regeneration by enhancing nerve growth factor signaling and reducing neuroinflammation.
GM1 is a monosialotetrahexosylganglioside that promotes neuronal protection and regeneration by enhancing nerve growth factor signaling and reducing neuroinflammation. Used for Acute ischemic stroke, Spinal cord injury, Peripheral nerve injury.
At a glance
| Generic name | GM1 |
|---|---|
| Also known as | monosialotetrahexosylganglioside |
| Sponsor | Qilu Pharmaceutical Co., Ltd. |
| Drug class | Ganglioside |
| Target | Growth factor receptors (indirect); neuronal cell surface ganglioside |
| Modality | Small molecule |
| Therapeutic area | Neurology |
| Phase | Phase 3 |
Mechanism of action
GM1 ganglioside is an endogenous glycosphingolipid that acts as a neurotrophic factor, facilitating neuronal survival, differentiation, and synaptic plasticity. It modulates growth factor receptor signaling pathways and reduces inflammatory cytokine production in the central and peripheral nervous systems, thereby protecting neurons from degeneration and promoting functional recovery.
Approved indications
- Acute ischemic stroke
- Spinal cord injury
- Peripheral nerve injury
Common side effects
- Injection site reactions
- Fever
- Headache
- Dizziness
Key clinical trials
- Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
- A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis (PHASE1)
- A Natural History Study of the Gangliosidoses
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis (PHASE3)
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) (PHASE3)
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis (PHASE3)
- BMS-986489 (Atigotatug + Nivolumab) vs Durvalumab in Limited-stage Small-cell Lung Cancer (TIGOS-LS) (PHASE2)
- Finding an Effective Dose of GM1 to Reduce or Prevent Neuropathy (Numbness or Weakness) Due to Treatment With Paclitaxel (Phase II) (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:
- GM1 CI brief — competitive landscape report
- GM1 updates RSS · CI watch RSS
- Qilu Pharmaceutical Co., Ltd. portfolio CI