SAGE has built a robust product pipeline initially focused on acute and orphan CNS indications with clinically validated targets and accelerated development timelines – enabling us to bring important medicines to patients rapidly.
Status Epilepticus (SE) is an acute, life-threatening form of epilepsy or seizures that occurs in approximately 150,000 U.S. patients each year, with a mortality rate of nearly 20%. Refractory SE, in which currently available treatment options are not effective, occurs in the approximately one-third of SE patients. These patients are moved to an ICU setting with little or no treatment options.
SAGE has developed SAGE-547, a potent positive allosteric modulator (PAM) of both synaptic and extra-synaptic GABAA receptors that rapidly advanced into Phase 1/2 clinical development for the orphan indication of refractory SE in early 2014. SAGE-547 demonstrated robust findings in preclinical models of SE, including significantly increased efficacy over current standard-of-care benzodiazepine treatment, and compelling early human experience data.
SAGE is also currently developing a seizure franchise of advanced next generation compounds of novel GABAA PAMs for the treatment of SE and other forms of seizure and epilepsy.
SAGE is continuing to build its robust pipeline of proprietary positive and negative allosteric modulators targeting NMDA receptors at a unique, previously unknown binding site (non-glycine site). By targeting NMDA receptors at a novel allosteric binding site, SAGE is able to mitigate the issues typically seen with alternate approaches including limited efficacy, tolerance, and safety. SAGE is evaluating future programs in cognition (schizophrenia, Alzheimer’s disease, Parkinson’s disease, and other conditions), autism, depression, epilepsy, and pain.