Rare Disease Report

Data For Super-Refractory Status Epilepticus Encouraging

JANUARY 08, 2015
James Radke

Orphan drug SAGE-547 continues to show promise as a treatment for super-refractory status epilepticus. SAGE-547 is an intravenous agent that modulates synaptic and extra-synaptic GABAA receptors.
Super-refractory status epilepticus is frightening version of status epilepticus, which on its own can be a life-threatening condition (35,000 of a total of 150,000 die from it each year). When a patient presents with status epilepticus, they are usually treated with benzodiazepines, and if no response, they are treated with second-line, anti-seizure drugs. If the seizure persists after the second-line therapy, the patient is diagnosed as having refractory status epilepticus and placed into a medically induced coma. After 24 hours, an attempt is made to wean the patient from the anesthetic agents to evaluate whether the seizure condition has resolved.  If seizures persist following the weaning attempts, the patient must be maintained in the medically induced coma and is diagnosed as having super-refractory status epilepticus. There are currently no therapies approved for refractory, or super-refractory, status epilepticus.
Last November we reported on 8 of the 11 patients from a phase I/II study meeting the primary endpoint of successfully being weaned off their anesthetic agents while being given SAGE-547.  Today, Sage Therapeutics announced they have data on 17 patients and so for, 71% of the patients have met the key efficacy endpoint of being successfully weaned off their anesthetic agents while SAGE-547 was being administered. In addition, 71% were successfully weaned off SAGE-547 without recurrence of SRSE. As a group, patients who responded to SAGE-547 generally demonstrated rapid improvement over the first five days following treatment. Patients who responded also continued to improve over the 30-day follow-up period as assessed by several measures.
In addition to the Phase I/II study, SAGE-547 has also been tested by independent centers under emergency use Investigational New Drug (IND) applications. Seven of 9 evaluable patients treated with SAGE-547 achieved resolution of SRSE either during the course of or soon after SAGE-547 treatment, resulting in an overall response rate of 78 %.

Jeff Jonas, M.D., chief executive officer of Sage Pharmaceuticals said:

"SAGE-547 has continued to show the potential for significant benefit for patients facing SRSE, a devastating and life-threatening disorder, which supports our continued development of SAGE-547 for the treatment of this disorder."

"In the past year, we've achieved many important milestones in the development of SAGE-547. We believe these data, combined with the emergency use data, support the initiation of a pivotal study and look forward to discussing this with the FDA."

Sage therapeutics is hoping to start a pivotal phase III study  by mid-2015.

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