SAGE Therapeutics has announced that SAGE-547 demonstrated robust activity, with a 77% response rate in evaluable patients with super-refractory status epilepticus (SRSE), in a successfully completed Phase 1/2 clinical trial.
SAGE-547 also demonstrated favourable tolerability and a benefit-risk profile supporting development in this acutely ill patient population. SRSE is a critical condition in which the brain is in a state of persistent seizure, where patients are placed in a medically induced coma in an attempt to stabilise them and where conventional and approved therapies fail to awaken the patients. Currently, there are no therapies specifically approved for SRSE.
SAGE-547 is an allosteric modulator of both synaptic and extra-synaptic GABAA receptors. GABAA receptors are widely regarded as validated drug targets for a variety of disorders, with decades of research and multiple approved drugs targeting these receptor systems.
Phase 3 trial of SAGE-547 to be initiated by mid-2015
“We believe these results are unprecedented in the treatment of SRSE and have the potential to be profoundly meaningful for patients affected by this devastating disorder. We believe these data help validate our platform, our unique chemistry capabilities and our approach to drug development based on rare disease indications with high unmet needs and rapid development pathways,” said Jeff Jonas, M.D., chief executive officer of SAGE.
Stephen Kanes, M.D., Ph.D., chief medical officer of SAGE, added, “We are extremely pleased by these data, which demonstrate the potential for SAGE-547 as an interventional treatment for patients where all other treatments have failed. Importantly, SAGE-547 also showed clear pharmacodynamic activity as measured by continuous EEG in patients that had previously failed third-line treatment while burst-suppressed under general anesthesia. We would like to thank the patients, their families and the physicians that participated in this trial.”
SAGE plans to initiate the STATUS Trial, a Phase 3 randomised, double-blind, placebo-controlled clinical trial of SAGE-547 for the treatment of patients with SRSE, by mid-2015. The results from the STATUS Trial, along with other results from the SAGE-547 development programme, may form the basis of a New Drug Application (NDA) submission for SAGE-547.
