SAGE Therapeutics, a biopharmaceutical company developing novel medicines to treat life-threatening and rare central nervous system (CNS) disorders, today announced dose initiation of the first patient in a Phase 1/2 clinical trial of SAGE-547, a neuroactive steroid, in super-refractory status epilepticus (SRSE), a critical condition in which the brain is in a state of persistent seizure. The trial is designed to evaluate the safety, tolerability and efficacy of SAGE-547 as an adjunctive therapy for the treatment of SRSE.
"Status epilepticus is a serious condition for which there are limited treatment options. Of the many patients whose condition escalates to refractory or super-refractory status epilepticus, most will not survive or will be discharged from the hospital with significant morbidities," said Stephen Kanes, M.D., Ph.D., chief medical officer of SAGE Therapeutics. "SAGE-547 is supported by strong preclinical data and scientific rationale, and we believe this compound has the potential to reduce or eliminate seizures in these patients who have exhausted all other therapeutic options."
This Phase 1/2 trial is an open label study of SAGE-547 as adjunctive therapy in people with SRSE. The trial is expected to enroll 10 to 15 adult patients with SRSE who have not responded to conventional therapy with continuous intravenous antiepileptic agents and who remain in a state of persistent seizure following one or more weaning attempts from anesthesia. The trial is designed to provide clear data around safety, exposure and the ability of SAGE-547 to effectively halt SRSE. Electrical brain activity will be evaluated via continuous electroencephalogram (EEG) monitoring before, during and after treatment with SAGE-547. Patients will be administered SAGE-547 intravenously for five days while weaning from anesthesia is attempted and will be monitored for four weeks following treatment.
"This clinical trial exemplifies SAGE's approach to designing studies with well-defined endpoints and development pathways that we believe allow us to make highly informed decisions regarding whether to continue development of our product candidates in an accelerated timeframe," said Jeffrey Jonas, M.D., chief executive officer of SAGE Therapeutics. "Our science and chemistry capabilities are targeting the creation of medicines to treat SE at various stages, and we believe SAGE-547 will be the first of several product candidates for the treatment of seizure disorders. We look forward to further evaluating SAGE-547 for the treatment of this critical and difficult-to-treat CNS condition."