SAGE Therapeutics has announced details of its planned SAGE-547 global Phase 3 development programme for the treatment of patients with super-refractory status epilepticus (SRSE). SRSE is a rare and life-threatening seizure disorder for which there are no approved therapies.
srse
At a recent End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), there was general agreement on the design and key elements of a Phase 3 clinical programme for SAGE-547. SAGE believes the results from the planned Phase 3 clinical trial, together with other clinical data obtained from the SAGE-547 development programme, could form the basis of a New Drug Application (NDA) submission for SAGE-547.
There is a pressing need for new drugs to treat patients with SRSE
“We are very pleased with the outcome of the End-of-Phase 2 meeting, and we look forward to initiating our Phase 3 clinical trial for SAGE-547 as potentially the first approved treatment for patients with SRSE,” said Jeff Jonas, M.D., chief executive officer of SAGE. “Our Phase 3 programme will be an important milestone for SAGE and, we hope, for patients suffering from SRSE. Our team has made rapid progress in advancing SAGE-547 in this indication and in furthering our GABAA modulation platform. This progress brings us one step closer to our goal of developing a family of molecules offering potential new treatment options for other forms of epilepsy and CNS disorders.”
“There is a pressing need for new drugs to treat patients with SRSE, a devastating seizure disorder for which current treatment options are severely limited,” said Andrew J. Cole, M.D., F.R.C.P.(C.), director of the Massachusetts General Hospital Epilepsy Service and professor of neurology at Harvard Medical School. “The combined safety and clinical activity data from SAGE’s Phase 1/2 clinical trial and emergency-use cases suggest that SAGE-547 has the potential to improve the therapeutic approach for patients with SRSE.”
The Phase 3 clinical trial is planned as a randomized, double-blind, placebo-controlled Phase 3 trial designed to assess the efficacy and safety of SAGE-547 in approximately 126 patients with SRSE, aged two years or older, at up to 150 sites in the U.S. and Europe.
The planned primary endpoint of the Phase 3 clinical trial will be successful resolution of status epilepticus (SE) after weaning the patient off all third-line anti-seizure agents, and SAGE-547 or placebo, without resumption of SE within 24 hours after completion of blinded SAGE-547 or placebo administration. Secondary endpoints are expected to explore the rate of recovery, regaining of consciousness, mental status and functional outcome. Patients who fail to respond to initial blinded treatment (SAGE-547 or placebo) may be eligible to be treated with an open-label, higher dose regimen of SAGE-547.
Phase 3 trial of SAGE-547 is a first-if-its-kind trial for SRSE
“Our planned Phase 3 randomized clinical trial of SAGE-547 is a pioneering and first-of-its-kind trial for SRSE. It marks an important step in developing new treatment options for patients affected with SRSE,” said Steve Kanes, M.D., Ph.D., chief medical officer of SAGE. “Our clinical and operations teams have executed aggressively to advance SAGE-547 through to late-stage development. Building off SAGE-547’s safety and clinical activity observed to date, we believe we have designed a highly efficient Phase 3 development program that, if successful, positions us to bring a first-in-class targeted treatment for patients with SRSE.”
In conjunction with its planned Phase 3 clinical trial, SAGE also plans to initiate an open-label, expanded access protocol designed to offer SAGE-547 to patients affected with SRSE with limited treatment options. Dose regimen, trial procedures and assessment of patient outcomes in the expanded access protocol will be consistent with the planned Phase 3 clinical trial.
