Sage Wins $10M Grant to Push Fragile X Treatment Into Clinic

Xconomy Boston — 

Sage Therapeutics’ identity shift towards edgier central nervous system disorders is now paying dividends—as much as $10 million in grant money, to be exact.

The Cambridge, MA-based biotech landed a National Institutes of Health grant to help it push forward a treatment for some of the social problems—fear and anxiety, for instance—experienced by patients with a rare form of autism known as Fragile X Syndrome.

The award was given through the agency’s NIH Blueprint Neurotherapeutics Network, a collaborative effort between various NIH centers that support research on the central nervous system. Sage will work hand-in-hand with those centers as it passes its experimental drug through the discovery phase and into the early stages of clinical development.

The money will help cover all of those activities through the end of Phase 1 clinical trials, and would total $10 million if the project progresses. The NIH will review the funding semi-annually and hand it out in pieces should Sage hit certain milestones. Sage will own all the equity in the intellectual property that comes from the research.

Fragile X is a rare form of autism in which children are born with an essential protein for cognitive development—the FMR-1 gene—knocked out, or unexpressed. This causes significant intellectual disabilities and behavioral problems, such as social phobias, anxiety, and risks of aggression. There are no approved treatments for Fragile X syndrome—patients are instead typically given treatments for anxiety, ADHD, and epilepsy to help fight the symptoms of the disorder.

Recently, a drug being developed by Cambridge, MA-based Seaside Therapeutics failed a clinical trial to treat the disorder. Fragile X affects one in 3,600 males and one in 8,000 females. About 60,000 people in the U.S. currently have it, according to Sage.

Sage’s plan is to apply its “allosteric receptor modulators,” or drugs designed to create an equilibrium among the transmitters in the brain, to create a drug that can curb the symptoms of Fragile X. It hopes to advance a drug with these qualities into an early stage clinical trial within the next two years.

Sage was founded by Third Rock Ventures with the help of a $35 million Series A round in October 2011.