BHR Pharma Announces SyNAPSe® Trial Completes Patient Enrollment

HERNDON, Va., Sept. 3, 2013 /PRNewswire/ --BHR Pharma, LLC (BHR) announced today that it has successfully randomized the 1,180th and final patient into its global, Phase III SyNAPSe Trial. The study is evaluating the safety and efficacy of BHR's proprietary intravenous progesterone formulation, BHR-100, for treating severe traumatic brain injury (TBI).

If the final data support BHR-100 efficacy, the drug could be the first-ever approved treatment for TBI.

The study's last patient was enrolled in the United States at the University of Iowa Hospitals and Clinics. In total, the SyNAPSe trial has 154 participating sites in the United States, Israel, Argentina, and 18 other countries in Europe and Asia.

"Achieving our enrollment goal is a significant milestone worth celebrating, especially in light of the many failed TBI trials that have come before and the significant difficulty of enrolling patients within eight hours after the initial injury," said Roland Gerritsen van der Hoop, M.D., Ph.D., BHR Pharma Chief Medical Officer and Head of Global Research and Development. "The study now moves into its important final phase patient follow up and data collection. We will continue to work closely with our sites to ensure that these activities are completed in full."

SyNAPSe patients are evaluated at six months post-injury using the Glasgow Outcome Scale (GOS), the study's primary endpoint. Secondary endpoints include GOS evaluation at three months post-injury, GOS-Extended and quality of life (SF-36) evaluations at three and six months post-injury, and mortality assessment at one month and six months post-injury. Results are expected in May 2014.

In addition to its well-known effects on the female reproductive system, progesterone is a potent neurosteroid and progesterone receptors are abundant and widely distributed in the central nervous system (CNS) in both males and females. Previous research has shown progesterone exerts its neuroprotective effects by protecting or rebuilding the blood-brain barrier, decreasing development of cerebral edema (brain swelling), down-regulating the inflammatory cascade and limiting cellular necrosis and apoptosis (programmed cell death) all potentially fatal injuries set in motion by TBI.

The European Medicines Agency (EMA), responsible for evaluating medicines developed for use in the European Union, granted an orphan medicinal product designation for treating moderate and severe TBI to BHR-100 in February of this year. In 2009, the U.S. Food and Drug Administration (FDA) granted BHR-100 an Orphan Drug designation also for treating moderate and severe TBI. FDA had previously placed the drug on Fast Track status designed to accelerate its potential approval.

BHR announced in January the trial's independent Data and Safety Monitoring Board (DSMB) formal interim analysis of the primary six-month efficacy data from 400 SyNAPSe patients concluded that there was no reason to stop the study for futility. The DSMB has conducted five safety reviews during the course of the study and has concluded each time that SyNAPSe should continue to its intended completion.

About Traumatic Brain Injury (TBI)The condition is a serious public health problem that, according to the Centers for Disease Control and Prevention, affects more than 3.5 million Americans each yearmore than all U.S. cases of breast cancer, HIV and Multiple Sclerosis combined (1.6 million). Despite significant efforts and more than 75 clinical trials over the past 20 years, there is still no approved treatment for TBI. Once a TBI occurs, medical treatment focuses on preventing further injury, symptomatic relief and promoting rehabilitation. TBI can have a debilitating impact on a person's life, and may require daily living assistance.

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BHR Pharma Announces SyNAPSe® Trial Completes Patient Enrollment