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
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Overview
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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"].
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
Preclinical studies have demonstrated that sigma-1 receptor activation reduces neuroinflammation, protects against excitotoxicity, and improves cognitive function in animal models of Alzheimer's disease[@axs].
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:
Calcium homeostasis: Regulates calcium release from endoplasmic reticulum stores
Mitochondrial function: Controls mitochondrial dynamics and energy production
Neuroprotection: Activates protective pathways against oxidative stress and excitotoxicity
Neuroinflammation: Modulates microglial activation and inflammatory responses
In Alzheimer's disease, sigma-1 receptor function may be impaired, leading to:
Increased calcium dysregulation
Enhanced neuroinflammation
Accelerated tau pathology
Cognitive decline
AXS-060 aims to restore sigma-1 receptor function, potentially addressing multiple aspects of Alzheimer's disease pathology[@sigmaa].
Competitive Landscape
Axsome's approach with sigma-1 receptor agonism represents a novel mechanism in Alzheimer's disease, differentiating it from: