Date: 2014-11-03
Type of information: Publication of results in a medical journal
phase: 1-2
Announcement: publication of results in the Annals of Neurology
Company: Sage Therapeutics (USA - MA)
Product: SAGE-547
Action
mechanism: allosteric modulator. 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.
Disease: super-refractory status epilepticus (SRSE)
Therapeutic area: CNS diseases - Neurological diseases
Country:
Trial details:
Latest
news: * On November 3, 2014, Sage Therapeutics announced that SAGE-547, an allosteric modulator of GABAA receptors, was used to treat super-refractory status epilepticus (SRSE) in two pediatric patients. The research, published online in the Annals of Neurology, is the first report of SAGE-547 treatment in children. Following treatment with SAGE-547, resolution of status epilepticus (SE) was demonstrated in both patients after weaning from general anesthetic infusions with no drug-related serious adverse effects. Both patients were treated with SAGE-547 under emergency-use Investigational New Drug Applications. The first patient, who was treated at Ann & Robert H. Lurie Children\'s Hospital of Chicago, was an otherwise healthy 11-year-old girl who presented with SE caused by an autoimmune disorder with antithyroid and anti-glutamic acid decarboxylase antibodies. She received an infusion of SAGE-547 over five days, after which pentobarbital sedation was weaned and discontinued. Over the remainder of the hospitalization she had intermittent, controllable seizures. She was transferred for inpatient rehabilitation, regained her ability to walk, and is now back at home, continuing to show cognitive improvement, reading, doing arithmetic and playing the piano. The second patient, a two-year-old girl, presented with SE associated with a febrile illness. SAGE-547 was infused over four days and tapered off between 96 and 120 hours. SE resolved after SAGE-547 treatment and 12 days following the completion of treatment with SAGE-547 all anti-seizure therapies were discontinued. She was transferred to inpatient rehabilitation and is now able to walk and speak. In both patients, there were no drug-related serious adverse effects detected by any of the laboratory tests used.
