SAGE Therapeutics Receives U.S. Orphan Drug Designation for Lead Compound SAGE-547 to Treat Status Epilepticus

In Press Release by Cameron

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May 1, 2014

SAGE Therapeutics (SAGE), a biopharmaceutical company developing novel medicines to treat life-threatening, rare central nervous system (CNS) disorders, today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its neuroactive steroid, SAGE-547, for the treatment of status epilepticus. SAGE Therapeutics is currently evaluating SAGE-547 in a Phase 1/2 clinical trial for the treatment of super-refractory status epilepticus (SRSE).

“Receiving orphan drug designation may help SAGE with the development of SAGE-547 and may ultimately allow us to deliver this promising medicine to patients who suffer from status epilepticus,” said Jeff Jonas, M.D., chief executive officer of SAGE Therapeutics. “Status epilepticus is a life-threatening disease with limited treatment options, and this advancement demonstrates our commitment to delivering SAGE-547 and other therapies to patients with life-threatening, rare CNS disorders.”

Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for tax credits for clinical development costs, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.

About SAGE-547

SAGE-547 is an allosteric modulator of both synaptic and extra-synaptic GABAA receptors. GABAA receptors are widely regarded as validated drug targets for a variety of CNS disorders, with decades of research and multiple approved drugs targeting these receptor systems. SAGE-547, developed by SAGE Therapeutics using its proprietary chemistry platform, is an intravenous agent in Phase 1/2 clinical development as an adjunctive therapy, a therapy combined with current therapeutic approaches, for the treatment of SRSE.

About Status Epilepticus

SE is a life-threatening seizure condition that occurs in approximately 150,000 people each year in the U.S.,1 of which 30,000 SE patients die. We estimate that there are 35,000 patients with SE in the U.S. that are hospitalized in the intensive care unit (ICU) each year. An SE patient is first treated with benzodiazepines, and if no response then treated with other, second-line, anti-seizure drugs. If the seizure persists after the second-line therapy the patient is diagnosed as having refractory SE (RSE), admitted to the ICU and placed into a medically induced coma. Currently, there are no therapies that have been specifically approved for RSE; however, physicians typically use anesthetic agents to induce the coma and stop the seizure immediately. After a period of 24 hours, an attempt is made to wean the patient from the anesthetic agents to evaluate whether or not the seizure condition has resolved. Unfortunately, not all patients respond to weaning attempts, in which case the patient must be maintained in the medically induced coma. At this point, the patient is diagnosed as having SRSE. Currently, there are no approved therapies for SRSE.

About SAGE Therapeutics

SAGE Therapeutics is a biopharmaceutical company committed to developing and commercializing novel medicines to treat life-threatening, rare CNS disorders. SAGE’s lead program, SAGE-547, is in clinical development for SRSE and is the first of several compounds the company is developing in its portfolio of potential seizure medicines. SAGE’s robust chemistry platform has generated multiple new compounds that target the GABAA and NMDA receptors, which are broadly accepted as impacting many psychiatric and neurological disorders. SAGE Therapeutics is a private company launched in 2010 by an experienced team of R&D leaders, CNS experts and investors. For more information, please visit

1 DeLorenzo, Robert J., Pellock, John M., Towne, Alan R., Boggs, Jane G. Epidemiology of Status Epilepticus. J Clin Neuro 1995; 12(4): 316-325.