Sage Therapeutics announced
very good response to their orphan designated drug SAGE-547 for the treatment of super-refractory status epilepticus in a phase 1/2 study.
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.
In the Phase 1/2 open-label trial, 12 adult patients with super-refractory status epilepticus who had not responded to conventional therapy and remained in a state of persistent seizure following one or more weaning attempts from general anesthesia were administered SAGE-547 intravenously for 5 days.
In the study, the primary endpoint (safety and tolerability) was met in all 12 patients. Further, of the 11 patients evaluable for efficacy, 8 patients met the key efficacy endpoint of being successfully weaned off their anesthetic agents while SAGE-547 was being administered, and 8 patients were successfully weaned off SAGE-547 without recurrence of super-refractory status epilepticus.
Jeff Jonas, M.D., chief executive officer of SAGE said:
"We believe SAGE-547 has the potential to dramatically improve the therapeutic approach for patients with SRSE, and the efficacy and safety results from this trial support our continued development of SAGE-547 as a treatment for this disorder."
"We look forward to working with the U.S. Food and Drug Administration (FDA) on the appropriate design of a pivotal trial, which we anticipate initiating in the first half of 2015 pending our discussions with the FDA. We believe SAGE-547 has the potential to be the first therapy intended specifically for the treatment of SRSE, and that is very exciting for patients and clinicians managing this life-threatening disease."
Trail Will Continue and Expanded to Include Children
The phase 1/2 study will continue and the company has reported that they have been given approval to include children as young as 2 years of age.
Steve Kanes, M.D., Ph.D., chief medical officer of SAGE said:
"We are also pleased that the approved protocol amendment to our Phase 1/2 trial will enable us to explore the potential of SAGE-547 in a broader population, particularly in very young children affected with this disorder that have no other treatment options."