ANAVEX®2-73 (blarcamesine) is an investigational oral therapy being studied for its ability to support cellular health by activating SIGMAR1, a receptor that helps restore balance within cells. Researchers are exploring its potential to slow neurodegeneration by boosting autophagy, a natural process that clears protein build-up and misfolded proteins in the brain.
Alzheimer’s Disease (AD) is a progressive and irreversible brain disorder that slowly erodes memory, cognitive abilities, and essential functions for independent living. It is the most common form of dementia and poses significant emotional, physical, and financial challenges for individuals and their families.
Rett syndrome (RS) is a rare neurological disorder that primarily affects girls, causing severe cognitive, motor, and communication impairments. It is typically diagnosed in early childhood and is often marked by a loss of previously acquired skills.
Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and developmental delays. It is a genetic condition that affects learning, behavior, and social interactions, often presenting alongside physical and emotional challenges.
Autophagy is the body’s natural process of clearing damaged proteins and cellular debris. In Alzheimer’s disease, this process can become impaired, leading to toxic protein buildup. Investigational therapies like blarcamesine may potentially restore autophagy, targeting a key mechanism that occurs upstream of amyloid beta and tau.
Therapeutic Candidates
We are committed to advancing research into the underlying mechanisms of neurodegenerative and neurodevelopmental diseases. Our investigational therapies are designed to address these challenges through innovative approaches currently being evaluated in clinical trials.
An investigational therapy being studied for its potential role in Alzheimer’s, Rett syndrome. and Fragile X syndrome.
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