Sage Therapeutics The National Institutes of Health (NIH) has launched three projects that will focus on development of therapeutics, including one project for Fragile X syndrome.

“We are excited about the opportunity to apply cutting-edge science to the pursuit of novel treatments for these debilitating disorders” said Rebecca Farkas, Ph.D., program director at NIH’s National Institute of Neurological Disorders and Stroke (NINDS), Office of Translational Research.

The investigator and organization for the Fragile X syndrome project is:

Sage Therapeutics, Cambridge, Mass.
Principal Investigator: Al Robichaud, Ph.D.
Disorder: Fragile X syndrome
Project Summary: Fragile X syndrome is a genetic disorder linked to a range of neurodevelopmental disorders including learning disabilities and cognitive impairment. Many patients experience general and social anxiety yet benzodiazepines, which are drugs typically used to treat anxiety disorders, provide little relief. Their anxiety has been linked to reduced activity in the brain by a protein called, the GABA A receptor. Sage Therapeutics is developing positive allosteric modulators, designed to enhance the receptor’s activity and possibly relieve the anxiety.

Source: NIH

SAGE Therapeutics says that the grant will support a drug candidate that could be used to treat anxiety and social deficits in patients with Fragile X syndrome, or FXS.

According to the company, the award could be worth up to $10 million.

Kevin Starr, interim chief executive of SAGE Therapeutics, said in a statement, “There is hope that in developing treatments for a monogenic form of autism like Fragile X, we may be able to make significant advances toward developing treatments for other forms of autism.”


The NFXF will follow-up with more information as soon as it is available to us.