ALZHEIMER’S DISEASE

Alzheimer’s disease is the most common form of dementia, accounting for 60%-80% of all cases. Synaptogenix has conducted the first placebo-controlled, Phase 2 trial of a protein kinase C epsilon (PKCɛ) activator for the treatment of Alzheimer’s, which in preclinical studies induced the growth of new synapses and prevented neuronal death. Recent clinical trial data showed sustained increase in Severe Impairment Battery measures and improvement in cognition, suggesting the drug may treat the progression of AD, rather than just symptoms. A confirmatory trial will commence in 2018.

FRAGILE X SYNDROME

Fragile X syndrome is a genetic disorder that causes developmental delays, intellectual and learning disabilities, anxiety, and autism spectrum disorders. FXS affects 135,000 in the U.S. alone. Bryostatin-1 for Fragile X has been designated as an orphan drug, and in animal models, was shown to restore synaptic networks, rectify memory deficits, and reverse biochemical abnormalities. Synaptogenix preclinical data, in a collaboration with the FRAXA Research Foundation, showed that Bryostatin-1 significantly improves autism spectrum abnormalities. Synaptogenix plans to initiate clinical testing of FXS patients in 2018.

NIEMANN PICK TYPE C

Niemann Pick Type C is both a debilitating and lethal disease, affecting about 35,000 children per year. Synaptogenix is working with world renowned experts at the Icahn School of Medicine, Mt. Sinai to develop its lead compound, Bryostatin-1, as a potential treatment for the excessive accumulation of cholesterol and other lipids in the viscera and brain resulting in cell death and to neuologic symptoms, including difficulty in swallowing and speaking, loss of coordination, seizures, and progressive dementia. There is no FDA approved treatment for Niemann-Pick Type C.