EPH-101 for Treatment of Amyotrophic Lateral Sclerosis

EPH-101 is a proprietary Efralex small molecule targeting the EphA4 receptor, a common link in multiple types of neurodegenerative disease, being developed as a treatment for Amyotrophic Lateral Sclerosis (ALS)

More than 6 million patients are affected in the United States (worldwide more than 50 million) by neurodegenerative disorders including Alzheimer’s Disease, Amyotrophic Lateral Sclerosis (ALS), and Frontotemporal Dementia (FTD). These conditions are most frequently diagnosed in people over the age of 60. However, many cases are diagnosed earlier, between the ages of 30 to 50. While clinical presentation varies in each disease type and individual patient, these symptoms often span a continued decline in memory, cognitive, and motor abilities, with disease progression determined by common cellular pathways.

EPH-101 targets several of these neurodegenerative disease pathways commonly linked to dysregulation of the EphA4 receptor. When dysregulated and overexpressed, the EphA4 receptor has been shown to reduce synaptic plasticity, promote inflammation, and cause neuronal cell death. Moreover, the EphA4 receptor is suggested to directly bind toxic ligands, such as amyloid beta and tau, implicated in the pathologies of Alzheimer’s disease. The EPH-101 small molecule agent uniquely binds the EphA4 receptor, resulting in a conformational change that triggers receptor internalization and inhibition of aberrant signaling for cell death.