The NIH has granted SAGE Therapeutics, a neuroscience product-focused company creating novel medicines to treat central nervous system (CNS) disorders, an award potentially worth up to $10 million to support the company's development of a novel drug which could be used to treat anxiety and social deficits in patients with Fragile X Syndrome (FXS).

FXS is a monogenic form of autism and an orphan condition causing significant behavioral impairments, such as anxiety and social phobia, and cognitive deficits. The incidence of FXS is approximately one in 3,600 males and one in 8,000 females, making it the leading cause of intellectual disability and autism, affecting over 60,000 individuals in the U.S. While there are no currently approved therapies for FXS, patients often are prescribed treatments for anxiety, Attention Deficit Hyperactive Disorder (ADHD) and epilepsy.

"The vast majority of patients with Fragile X suffer from debilitating fear, anxiety and social dysfunction, and there are no approved drugs for this condition," said Kevin Starr, interim chief executive officer at SAGE Therapeutics. "There is hope that in developing treatments for a monogenic form of autism like Fragile X, we may be able to make advances toward developing treatments for other forms of autism."

The award was one of three made through the NIH Blueprint Neurotherapeutics Network and is designed to spur potential, day-to-day collaboration between SAGE and the Network's 15 agency institutes and centers as the company advances its FXS program through discovery and early clinical development. The award includes resources in kind to support discovery, early development and phase I activities. Funding will be reviewed semi-annually and awarded based on successful completion of preset milestones. SAGE will retain all intellectual property rights to all equity derived from the grant research.

SAGE plans to develop a proprietary Positive Allosteric Modulator (PAM) targeting GABA_A that may provide symptomatic and potentially disease-modifying therapeutic benefits to patients with FXS, with a focus on ameliorating anxiety and social deficits. This approach may offer an effective therapy with a favorable safety profile and minimal side effects for patients with few treatment options. SAGE is planning to advance its FXS program toward a phase I clinical trial in the next two years.