SAGE Therapeutics And Collaborators Announce Publication Highlighting Epidemiology And Burden Of Illness For Super-Refractory Status Epilepticus In Journal Of Medical Economics

Largest epidemiology-focused SRSE study to date and first study to examine economic impact of related hospitalization

Study suggests SRSE is associated with high mortality and morbidity, and significant healthcare cost

Projections based on study findings suggest estimated U.S. annual incidence of approximately 25k to 41k cases of SRSE

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sage Therapeutics (NASDAQ: SAGE), a clinical-stage biopharmaceutical company developing novel medicines to treat life-altering central nervous system (CNS) disorders, today announced the publication of a study conducted with collaborators from Massachusetts General Hospital (MGH) on the epidemiology of super-refractory status epilepticus (SRSE). The paper, titled “Burden of illness for super-refractory status epilepticus,” was recently published in the Journal of Medical Economics (doi: 10.1080/13696998.2016.1223680).

“By estimating the potential patient population and related medical expenses associated with SRSE, the data highlight the tremendous need for development of treatment options for this disorder and the importance of conducting additional studies in SRSE.”

“While SRSE has been known to be a devastating, life-threatening condition associated with high cost of care, few studies have examined the incidence rate or quantified the healthcare resource utilization of this condition,” said Lidia Moura, MD, MPH, a neurologist and health services researcher in the Department of Neurology at MGH and Harvard Medical School and co-author of the paper. “In addition, most of the existing studies focused on smaller patient populations outside of the U.S. The findings from this study help to provide a new understanding of the high mortality and morbidity, and significant use of healthcare resources associated with SRSE.”

“This research, undertaken by Sage and our collaborators, helps to provide a new data-driven understanding of the clinical and economic costs of SRSE,” said Tom Anderson, Chief Commercial Strategy Officer of Sage. “By estimating the potential patient population and related medical expenses associated with SRSE, the data highlight the tremendous need for development of treatment options for this disorder and the importance of conducting additional studies in SRSE.”

Study Summary and Key Findings:

• The Premiere Hospital Database was utilized to estimate the annual number of SRSE cases in the U.S. and to evaluate utilization of hospital resources by SRSE patients based on hospital discharges during 2012. The Premiere Hospital Database included 5.3 million hospital discharges in 2012, accounting for approximately 20% of U.S. annual hospital discharges.
• SRSE does not currently have a specific ICD-9 (International Classification of Diseases, 9th Revision) code and status epilepticus codes are used inconsistently. Discharges were classified as SRSE cases in the study based on an algorithm using seizure-related International Classification of Diseases-9 (ICD-9) codes, Intensive Care Unit (ICU) length of stay (LOS) and treatment protocols (e.g., benzodiazepines, antiepileptic drugs and ventilator use). Secondary analyses were conducted using more restrictive algorithms for SRSE.
• A total of 6,325 hospital discharges were classified as SRSE cases using the primary algorithm from the total 5.3 million hospital discharges in 2012 in the Premiere Hospital Database. This projects to an estimated 41,156 cases of SRSE in the U.S. during 2012, or an estimated annual incidence rate of approximately 13/100,000, when applying a weighted projection based on hospital characteristics and 2012 U.S. demographics. Secondary analyses using stricter algorithms to classify SRSE resulted in an estimated 35,150 annual SRSE cases (or an incidence rate of approximately 11/100,000) and 25,915 annual SRSE cases (or an incidence of 8/100,000), respectively. Given the limitations of the methodology, the authors noted that more in-depth studies are needed.
• The mean LOS for SRSE hospitalizations was 16.5 days and the mean LOS in the ICU was 9.3 days. The most common LOS was 10-19 days. Twenty-four percent of SRSE patients spent 20 or more days in the hospital. The mean cost of an SRSE hospitalization was $51,247, increasing with LOS to a mean cost of $156,500 for patients with LOS of 30 or more days (11% of SRSE cases identified).

About Super-Refractory Status Epilepticus
Status epilepticus (SE) is an acute medical emergency of persistent, unremitting seizure lasting greater than five minutes. An SE patient is first treated with benzodiazepines, and if no response, is then treated with other, second-line, anti-seizure drugs. If the SE persists after the second-line therapy, the patient is diagnosed as having refractory SE (RSE), admitted to the ICU and placed into a medically induced coma. Physicians typically use anesthetic agents to induce the coma, along with antiepileptic drugs in an attempt to stop the SE, in RSE patients. After a period of 24 hours, an attempt is made to wean the patient from the anesthetic agents to evaluate whether or not the SE has resolved. Unfortunately, not all patients respond to weaning attempts, in which case the patient must be maintained in the medically induced coma. At this point, the patient is diagnosed as having SRSE. Currently, there are no therapies specifically approved for SRSE.

About the STATUS Trial
Sage is currently enrolling the Phase 3 STATUS Trial to evaluate the efficacy and safety of SAGE-547 in approximately 126 evaluable patients with SRSE, ages two years or older, in the U.S., Canada and Europe. In the double-blind trial, patients are randomized 1:1 to receive either SAGE-547 or placebo in addition to standard-of-care third-line anti-seizure agents for six days. The primary endpoint is successful resolution of status epilepticus (SE) after weaning the patient off all third-line agents, and SAGE-547 or placebo, without resumption of SE within 24 hours after completion of blinded SAGE-547 or placebo administration. The STATUS Trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA).

About SAGE-547
SAGE-547 is an allosteric modulator of both synaptic and extrasynaptic GABA_A receptors. SAGE-547 is an intravenous agent in Phase 3 clinical development as an adjunctive therapy for the treatment of super-refractory status epilepticus (SRSE) and is being developed for severe postpartum depression. SAGE-547 has been granted both Fast Track and orphan drug designations by the U.S. Food and Drug Administration (FDA) for the treatment of SRSE. For more information about the STATUS Trial, please visit www.statustrial.com.

About Sage Therapeutics
Sage Therapeutics is a clinical-stage biopharmaceutical company committed to developing novel medicines to transform the lives of patients with life-altering central nervous system (CNS) disorders. Sage has a portfolio of novel product candidates targeting critical CNS receptor systems, GABA and NMDA. Sage's lead program, SAGE-547, is in Phase 3 clinical development for super-refractory status epilepticus, a rare and severe seizure disorder, and is being developed for severe postpartum depression. Sage is developing its next generation modulators, including SAGE-217, SAGE-689 and SAGE-718, with a focus on acute and chronic CNS disorders. For more information, please visit www.sagerx.com.

Forward-Looking Statement
Various statements in this release concern Sage's future expectations, including our estimates and projections as to the potential number of patients with SRSE and the burden and costs associated with SRSE. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. There are limitations in the methodology we and our collaborators used to estimate SRSE patient numbers and disease burden and costs. As a result, our estimates and projections may prove not to be accurate. The actual number of patients with SRSE and the burden and costs associated with SRSE may be significantly lower than our estimates. These risks and additional risks are more fully discussed in the section entitled "Risk Factors" in our most recent Quarterly Report on Form 10-Q, as well as in discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today, and should not be relied upon as representing our views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.