We are developing new medicines to make life better for patients suffering from rare, life-threatening CNS disorders.
Our different approach to drug discovery and development has allowed us to create and advance multiple candidates with the potential to treat a range of central nervous system (CNS) disorders.
Our proprietary approach to drug discovery and development has generated selective compounds with precise pharmacologic properties that may be applied to the treatment of multiple CNS disorders. These compounds, which we call second-generation neurosteroids, have improved pharmacokinetic properties that may allow for both oral availability and improved therapeutic indexes. As a result, they may be better suited for long-term, or chronic, use. SAGE-689 and SAGE-217 are second-generation neurosteroids we have selected as clinical candidates.
Our lead product candidate is SAGE-547, an allosteric modulator of both synaptic and extra-synaptic GABAA receptors. SAGE-547 is an intravenous, or IV, treatment in clinical development as an adjunctive therapy, a therapy combined with current therapeutic approaches, for the treatment of super-refractory status epilepticus, or SRSE.
SAGE-547 was granted fast track designation by the U.S. Food and Drug Administration (FDA) in July 2014 for SRSE. Fast track designation is granted by the FDA to facilitate the development and expedite the review of drug candidates that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. SAGE-547 also received orphan drug designation, which is intended to facilitate drug development for rare diseases, from the FDA in April 2014.
Learn more about the results of SAGE's clinical trials.
SAGE-217 is a novel neuroactive steroid that acts as a positive allosteric modulator of synaptic and extra-synaptic GABAA receptor subtypes. Unlike many of the naturally occurring neuroactive steroids, SAGE-217 has a pharmacokinetic profile to potentially support once-daily oral dosing and a selectivity profile that minimizes potential off-target side effects.
SAGE-217 is currently in IND-enabling toxicology and safety pharmacology testing to support potential clinical development for a range of seizure conditions, including orphan epilepsy disorders, such as Rett Syndrome and Dravet Syndrome.
We are exploring additional potential products in a variety of CNS disorders based on modulation of both the GABAA and NMDA receptors. In addition to our products focused on status epilepticus (SE), other central nervous system (CNS) disorders we believe our approach may have a significant impact on include Rett syndrome, Dravet syndrome, fragile X syndrome, anxiety, depression, sleep disorders, mania, tremor, tinnitus and post-traumatic stress disorder.