- Fragile X-associated Disorders
- Treatment & Intervention
- Support the NFXF
We reached out to Elizabeth Berry-Kravis, MD, PhD member of the Scientific and Clinical Advisory Committee regarding the NIH’s new Neurological Drug Development Project with SAGE Therapeutics. She says, “This funding is to develop a drug that works to increase activity at GABA-A receptors which there is some data to suggest have reduced activity in the brain of the fragile X mouse (GABA-A is different than the one arbaclofen works on which is GABA-B.) GABA-A is the receptor activated by the drug galaxonone that Dr. Randi Hagerman is conducting a trial of.”
Below is the official Press Release from SAGE Therapeutics:
Cambridge, Mass. – July 31, 2013 – SAGE Therapeutics, a neuroscience product-focused company creating novel medicines to treat important central nervous system (CNS) disorders, today announced that the National Institutes of Health (NIH) has granted the company 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).
“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 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 significant advances toward developing treatments for other forms of autism.”
The award was one of three made through the NIH Blueprint Neurotherapeutics Network (“the 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 1 activities, and could be worth up to $10 million if the project progresses as planned. 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.
“We are excited about the opportunity to apply cutting-edge science to the pursuit of novel treatments for Fragile X syndrome and other neurological disorders,” said Rebecca Farkas, Ph.D., a program director at the NIH’s National Institute of Neurological Disorders and Stroke, Office of Translational Research.
FXS is a monogenic form of autism and an orphan condition causing significant behavioral impairments, such as anxiety and social phobia, and cognitive deficits .1 The incidence of FXS is approximately 1 in 3,600 males and 1 in 8,000 females, making it the leading cause of intellectual disability and autism, with a prevalence of over 60,000 individuals in the United States.2,3 While there are no currently approved therapies for FXS, patients are often prescribed treatments for anxiety, Attention Deficit Disorder (ADHD) and epilepsy.4
“The hope is that this grant will enable us to accelerate our development of an innovative therapy designed to address the symptoms of Fragile X, and could potentially have a durable, disease-modifying effect, as well,” said Al Robichaud, Ph.D., chief scientific officer of SAGE Therapeutics and principal investigator on the grant. “We are honored to be partnering with the NIH to work toward a potential breakthrough treatment. We believe that public-private partnerships such as this will enable us to accelerate reaching our shared goal of profoundly improving the lives of these patients and their families.”
SAGE plans to develop a proprietary Positive Allosteric Modulator (PAM) targeting GABAA 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 1 clinical trial in the next two years.
SAGE Therapeutics is a neuroscience product focused company developing breakthrough medicines to treat central nervous system (CNS) disorders with tremendous unmet need including status epilepticus, anesthesia, FXS and traumatic brain injury. The company’s proprietary Positive and Negative Allosteric Modulator (PANAM) platform leverages extensive chemistry expertise to enable the safe and efficacious allosteric modulation of GABAA or NMDA receptors to restore the balance of activity that is disrupted in a variety of CNS disorders. As a result, SAGE has built a robust product pipeline initially focused on acute and orphan CNS indications with clinically validated targets, accelerated development timelines and a wide range of additional applications. SAGE Therapeutics is a private company launched in 2011 by a proven team of R&D leaders, renowned CNS experts and Third Rock Ventures. For more information, please visit www.sagerx.com.