Axsome Therapeutics, Inc.

Overview

Axsome Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel therapies for central nervous system (CNS) disorders. Founded in 2012 and headquartered in New York, Axsome focuses on addressing unmet needs in neuropsychiatry, particularly in Alzheimer's disease, depression, and sleep disorders["@axsome"].

Funding

• IPO: 2018 (NASDAQ: AXSM)
• Market Cap: ~$6B (2026)

Pipeline

Alzheimer's Disease

Overview

Axsome Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel therapies for central nervous system (CNS) disorders. Founded in 2012 and headquartered in New York, Axsome focuses on addressing unmet needs in neuropsychiatry, particularly in Alzheimer's disease, depression, and sleep disorders["@axsome"].

Funding

• IPO: 2018 (NASDAQ: AXSM)
• Market Cap: ~$6B (2026)

Pipeline

Alzheimer's Disease

| Drug | Mechanism | Stage | Status |
|------|-----------|-------|--------|
| AXS-060 | Sigma-1 receptor agonist / M1 muscarinic modulator | Phase 2/3 | Recruiting |
| AXS-05 | NMDA receptor antagonist / sigma-1 agonist | Phase 2 | Completed |

AXS-060

AXS-060 (formerly known as ACD-680) is a novel, first-in-class, orally administered sigma-1 receptor agonist being developed for the treatment of agitation and other neuropsychiatric symptoms in Alzheimer's disease. Sigma-1 receptors are highly expressed in brain regions involved in cognition and emotion, including the hippocampus and prefrontal cortex[@sigma].

The sigma-1 receptor is a chaperone protein located at the endoplasmic reticulum-mitochondria interface that plays a critical role in:

• Calcium signaling
• [Mitochondrial](/mechanisms/mitochondrial-dysfunction) function
• Neuroprotection
• Response to oxidative stress

Other CNS Programs

| Drug | Indication | Mechanism | Stage |
|------|------------|-----------|-------|
| AXS-05 | Major depressive disorder | NMDA antagonist + sigma-1 agonist | Phase 3 |
| AXS-07 | Migraine | CGRP antagonist / 5-HT1F agonist | Phase 3 |
| AXS-12 | Narcolepsy | Norepinephrine reuptake inhibitor | Phase 2 |

Business Development

• Partnerships: Axsome has collaborated with various academic institutions and has in-licensed certain compounds
• Financing: Public company since 2015 (NASDAQ: AXSM), has raised over $500M in capital
• Regulatory: Received FDA Fast Track designation for AXS-060 in Alzheimer's disease agitation

Science

Sigma-1 Receptor Biology

The sigma-1 receptor is an unusual intracellular receptor that acts as a molecular chaperone. Unlike traditional neurotransmitter receptors, sigma-1 receptors modulate multiple signaling pathways:

In Alzheimer's disease, sigma-1 receptor function may be impaired, leading to:

• Increased calcium dysregulation
• Enhanced neuroinflammation
• Accelerated tau pathology
• Cognitive decline

Competitive Landscape

Axsome's approach with sigma-1 receptor agonism represents a novel mechanism in Alzheimer's disease, differentiating it from:

• Anti-amyloid antibodies (Leqembi, Donanemab)
• Anti-tau approaches
• BACE inhibitors (largely discontinued)
• Symptomatic treatments (acetylcholinesterase inhibitors, memantine)

Cross-References

• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Alzheimer's Disease](/diseases/alzheimers-disease) Treatment
• [Neuroinflammation](/mechanisms/neuroinflammation) in AD
• Sigma-1 Receptor
• Behavioral Symptoms of Dementia
• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Parkinson's Disease](/genes/ar)

External Links

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

References