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SAGE Therapeutics, a biopharmaceutical company developing novel medicines to treat central nervous system (CNS) diseases, has announced new data presented at the American Epilepsy Society (AES) Annual Meeting that reveal a novel mechanism and approach for the treatment of status epilepticus (SE) and identify new, orally bioavailable compounds that may lead to improved therapies for chronic epilepsies.
"Epilepsy is among many disorders of the CNS that have a critical unmet need," said Al Robichaud, Ph.D., chief scientific officer of SAGE. "These data highlight SAGE's robust chemistry platform as well as our ability to design and develop molecules that directly impact specific mechanisms involved in epilepsy and other CNS diseases. Our focus on GABAA receptor modulation is unlike previous efforts, opening up significant potential to treat diseases with no current therapies."
GABAA receptors are an attractive target for the treatment of a multitude of CNS disorders, including acute and chronic seizures. Traditional approaches of inhibiting or activating the GABA pathway have been associated with significant toxicities. SAGE's unique and proprietary approach of both positive and negative allosteric modulation of the GABAA receptor "fine-tunes" brain activity and has the potential to more effectively treat these diseases, while limiting the harmful side effects seen with many CNS therapies.
The SAGE team has developed SAGE-547, a proprietary investigational treatment for SE that has potent activity at both synaptic and extra-synaptic GABAA receptors and has been shown to effectively treat seizures and SE in preclinical studies. In the findings presented at the AES meeting, SAGE researchers describe new compounds that are orally bioavailable, selective for the GABAA receptor and show robust anti-seizure activity in preclinical models. These findings may lead to new therapies for the treatment of chronic epilepsies and orphan genetic epilepsies, such as Fragile X syndrome, Dravet Syndrome and Rett Syndrome.
"We are rapidly entering the clinic with our lead program in status epilepticus and these additional novel oral compounds have the potential to quickly follow," said Jeff Jonas, chief executive officer of SAGE. "We are committed to delivering better therapies to patients in dire need of more effective treatments for debilitating CNS disorders."
The research was presented in a poster titled "Generation of synthetic neuroactive steroids with potent positive modulatory activity at GABAA synaptic and extra-synaptic receptors for the treatment of epilepsy," at the AES annual meeting in Washington, D.C.
Article adapted by Medical News Today from original press release. Click 'references' tab above for source.
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Therapeutics, SAGE. "SAGE Therapeutics presents new data identifying highly selective, oral compounds and novel approach for the treatment of chronic epilepsies." Medical News Today. MediLexicon, Intl., 12 Dec. 2013. Web.
19 Apr. 2014. <http://www.medicalnewstoday.com/releases/269998>
Therapeutics, S. (2013, December 12). "SAGE Therapeutics presents new data identifying highly selective, oral compounds and novel approach for the treatment of chronic epilepsies." Medical News Today. Retrieved from
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