US-based biopharmaceutical firm SAGE Therapeutics has started dosing patients in a Phase I/II clinical trial of SAGE-547, a neuroactive steroid, for treatment of super-refractory status epilepticus (SRSE).
The trial is designed to assess the safety, tolerability and efficacy of SAGE-547 as an adjunctive therapy for the treatment of SRSE, a life-threatening condition that involves the brain being in a state of persistent seizure.
SAGE Therapeutics chief medical officer Stephen Kanes said 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," Kanes said.
"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."
The Phase I/II clinical trial will enrol about between ten and 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 offer clear data around safety, exposure and the ability of SAGE-547 to effectively halt SRSE.
SAGE Therapeutics said that electrical brain activity will be evaluated via continuous electroencephalogram (EEG) monitoring before, during and after treatment with SAGE-547.
In the trial, patients will be given SAGE-547 intravenously for five days while weaning from anesthesia is attempted and will be monitored for four weeks following treatment.
SAGE Therapeutics chief executive officer Jeffrey Jonas said: "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."
SAGE-547 is a potent positive allosteric modulator of both synaptic and extra-synaptic GABAA receptors, which are primary inhibitory neurotransmitters and widely regarded as validated drug targets for a variety of CNS disorders.