Date: 2015-04-20
Type of information: Treatment of the first patient
phase: 3
Announcement: treatment of the first patient
Company: Sage Therapeutics (USA - MA)
Product: SAGE-547
Action
mechanism: Allosteric modulator. 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. SAGE-547 is an intravenous agent in Phase 1/2 clinical development as an adjunctive therapy, a therapy combined with current therapeutic approaches, for the treatment of super-refractory status epilepticus (SRSE), as well as in exploratory Phase 2 clinical trials for the treatment of essential tremor and as an adjunctive therapy for the treatment of severe postpartum depression (PPD). In 2014, the FDA granted both Fast Track and orphan drug designation to SAGE-547 for the treatment of SRSE.
Disease: super-refractory status epilepticus (SRSE)
Therapeutic area: CNS diseases - Neurological diseases
Country: USA
Trial
details: Study 302, the Phase 3 open-label expanded access protocol, will make SAGE-547 available to patients in the United States, aged two years or older, who are affected with SRSE. Patients will receive SAGE-547 as an adjunctive therapy for a six-day treatment regimen and may be eligible for up to two treatments. Dose regimen, trial procedures and assessment of patient outcomes in the expanded access protocol will be consistent with the planned Phase 3 randomized, double-blind, placebo-controlled clinical trial of SAGE-547 for the treatment of patients with SRSE. Patients treated in the expanded access protocol will be monitored for two months following initiation of the last treatment (NCT02433314).
Latest
news: * On April 20, 2015, Sage Therapeutics, a clinical-stage biopharmaceutical company developing novel medicines to treat life-threatening, rare central nervous system (CNS) disorders, announced completion of treatment for the first patient enrolled in its now initiated open-label expanded access protocol, designated Study 302. Study 302 is designed to offer SAGE-547 to patients affected by super-refractory status epilepticus (SRSE) and to evaluate the safety of SAGE-547 in patients with SRSE. The results from this clinical trial, along with results from SAGE\'s planned Phase 3 placebo-controlled clinical trial - called the STATUS Trial - and other clinical data obtained from the SAGE-547 development program are intended to form the basis of a New Drug Application (NDA) submission for SAGE-547. Under expanded access, the FDA works with companies to allow access to investigational therapies to patients with serious or life-threatening illnesses who do not otherwise qualify for participation in a clinical trial and for whom there are no comparable or satisfactory alternate therapies. \"We are pleased to commence our Phase 3 development program with the initiation of Study 302, our open-label expanded access protocol for SAGE-547 in SRSE, a life-threatening seizure disorder for which there are no approved therapies,\" said Jeff Jonas, M.D., chief executive officer of SAGE. \"Study 302 will allow for the expedited treatment of patients who are located at hospitals that are not participating in our Phase 3 placebo-controlled clinical trial and who cannot be transferred to trial sites for medical or other reasons. In addition to Study 302, our team continues to rapidly execute on our development timelines, and we look forward to beginning enrollment of the Phase 3 STATUS Trial by mid-year.\" \"Study 302 will be an important feature of our SAGE-547 development program, as these data will contribute significantly to the SAGE-547 safety database for our planned NDA submission,\" said Steve Kanes, M.D., Ph.D., chief medical officer of SAGE. \"We also plan to convert Study 302 sites into future Phase 3 STATUS Trial sites, potentially accelerating our Phase 3 trial enrollment.\" SAGE plans to begin enrollment of the planned STATUS Trial by mid-year, following submission and review by the FDA of the final clinical trial protocol and updated chemistry, manufacturing and controls information. In addition, the Phase 1/2 clinical trial of SAGE-547 in patients with SRSE has completed enrollment, and SAGE plans to report final clinical data at the Antiepileptic Drug and Device Trials XIII Conference, taking place May 13-15.
