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Aug 1, 2013

NIH Awards SAGE Up to $10M for Fragile X Syndrome Treatment

  • The National Institutes of Health (NIH) has granted SAGE Therapeutics an award potentially worth up to $10 million to support the company’s development of a drug which could be used to treat anxiety and social deficits in patients with Fragile X Syndrome (FXS).

    The award was one of three made through the NIH Blueprint Neurotherapeutics Network and is designed to stimulate 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.

    “The vast majority of patients with Fragile X suffer from debilitating fear, anxiety and social dysfunction, and there are no approved drugs for this devastating condition,” said Kevin Starr, interim CEO 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 significant advances toward developing treatments for other forms of autism.”

    SAGE plans to develop a Positive Allosteric Modulator targeting GABAA that could 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. The company is planning to advance its FXS program toward a Phase I clinical trial in the next two years.


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