Enterin Inc., a biopharmaceutical company based in Philadelphia, is aiming to become the first to develop a treatment that targets the gut-brain axis in patients with neurodegenerative disease. The gut-brain axis allows bidirectional communication between the enteric and central nervous systems. Research has demonstrated that alpha-synuclein accumulates in the enteric nervous system of patients with Parkinson’s disease, impairing neuronal function and leading to cell death.
Enterin’s lead investigational compound, ENT-01 (squalamine phosphate), is an orally administered small molecule that clears accumulated alpha-synuclein from cell membranes and prevents new aggregates from forming. ENT-01 completed a Phase 2a study in patients with Parkinson’s disease, where it was found to improve constipation, hallucinations, depression, and motor symptoms. In a Phase 2b study, ENT-01 significantly improved change in spontaneous bowel movement vs placebo in patients with Parkinson’s and constipation.
Last week, the FDA approved Enterin’s Investigational New Drug application to treat a patient with prodromal multiple system atrophy with ENT-01. The patient is a 42-year-old male with an ATP13A2 gene mutation, confirmed presence of alpha-synuclein in enteric neurons, and a 2-year history of symptoms including constipation, erectile dysfunction, and sleep disturbances suggestive of REM sleep behavior disorder. He will be treated with ENT-01 indefinitely or until the patient develops significant adverse events.
Dr. Robert Hauser, the patient’s treating physician and Director of the Parkinson’s Disease and Movement Disorders Center of the University of South Florida said, “We hope that ENT-01 will be helpful in preventing progression of MSA in this individual and that this work will help promote personalized medicine for neurodegenerative diseases.”
Enterin also plans to study ENT-01 in patients with Parkinson’s and psychosis, Parkinson’s and dementia, and in patients with autism.
