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NIH Grant Worth Up to $10 Million Will Advance Potential Fragile X Treatment

Award to SAGE Therapeutics will advance testing of medicine for autism symptoms in fragile X; promise for other forms of autism
August 02, 2013


The National Institutes of Health (NIH) has granted SAGE Therapeutics an award worth up to $10 million to help the company explore a new class of medicines with the potential to address the anxiety and social disabilities associated with fragile X syndrome.

Fragile X is a disorder of brain development associated with autistic behaviors and intellectual disability. In recent years, a small but growing number of pharmaceutical companies have been developing medicines that might relieve its symptoms. The larger hope is that these medicines will likewise prove effective in treating the social disabilities associated with other forms of autism.

“It’s exciting to see another commercial player take on the challenges of bringing new medicines to our families in the autism community,” says Autism Speaks Chief Science Officer Rob Ring. “As the recent trials with arbaclofen have taught us, there are no sure things for a company like SAGE as they enter clinical development,” he adds. “But I can assure you there will be no new treatments for autism unless someone takes these shots.”

Just as encouraging, Dr. Ring says, is to see the NIH make the kind of substantial investment needed to move scientific discoveries beyond academic research and into clinical trials.

“The scale of an award like this is what’s required to move these stories forward,” he says. “Like Autism Speaks, the NIH is exploring innovative funding models to bring scientific discoveries across the proverbial ‘valley of death’ between basic academic research and the costly commercial development of new medicines.”

The NIH award provides SAGE Therapeutics with resources from a number of NIH agencies and centers to support its early clinical testing of fragile X medicines.

“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,” says Kevin Starr, of 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 will be exploring a class of medicines classified as “PAM-GABA-a,” for positive allosteric modulators targeting the GABA-a brain cell receptor. As with the experimental fragile X medicine arbaclofen, the hope is to restore the right balance of excitatory and inhibitory signals in the brain. (Arbaclofen targets the related brain-cell receptor GABA-b.)

SAGE Therapeutics announced that it hopes to begin early clinical trials within the next two years.