A Grand Rapids-based clinical stage biotechnology company is beginning phase two of a potential treatment for the most common genetic form of autism.
Tetra Discovery Partners announced this week the testing of BPN14770, a drug that may improve the quality of connections between neurons and improve multiple behavioral outcomes caused by Fragile X Syndrome.
The treatment has received an Orphan Drug Designation from the U.S. Food and Drug Agency for the treatment of Fragile X Syndrome. The designation provides incentives to sponsors to develop products “intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders,” according to the agency website.
The study will test the drug on 30 adult male participants ages 18 to 45.
The study includes preliminary cognitive and behavioral assessments to determine efficacy, safety and tolerability. It also will gather information on how the treatment acts inside the body and how the body reacts to the treatment.
The study is being conducted at Rush University Medical Center in Chicago by principal investigator Dr. Elizabeth Berry-Kravis, with financial support from the FRAXA Research Foundation.
“We are very pleased to support this clinical investigation of BPN14770 in patients with Fragile X Syndrome by Dr. Berry-Kravis, whose early research was instrumental to our understanding of biochemical changes underlying the condition,” said Dr. Michael Tranfaglia, medical director and chief scientific officer of the FRAXA Research Foundation.
Tetra Discovery Partners
Tetra Discovery Partners is a Grand Rapids-based clinical-stage biotechnology company working to develop a portfolio of therapeutic products for those suffering from neuro-developmental conditions such as Fragile X Syndrome, Alzheimer's disease, traumatic brain injury and other brain disorders.
Tetra was a recipient of an NIH Blueprint Neurotherapeutics Program cooperative research agreement and receives major funding from the National Institute on Aging, the Alzheimer’s Drug Discovery Foundation, the National Institute of Neurological Disorders and Stroke, and the National Institute of Mental Health’s Small Business Innovation Research program.