Clinical Trial: Neuroprotective Agent for Treating Severe Traumatic Brain Injury
At the Neurotrauma Society’s 2012 Annual Meeting, McGowan Institute for Regenerative Medicine affiliated faculty member David Okonkwo, MD, PhD, assistant professor with the Department of Neurological Surgery/University of Pittsburgh Medical Center, director of Neurotrauma and of the Spinal Deformity Program, clinical director of the Brain Trauma Research Center, and most recently, the associate director of the Center for Injury Research and Control, presented results from BHR Pharma’s SyNAPSe® clinical trial. Dr. Okonkwo, study principal investigator, announced the trial enrollment midpoint of 590 patients, which was the milestone for the global Phase 3, multi-center trial. This was achieved when a study subject was randomized by Songklanagarind Hospital in Thailand.
SyNAPSe is evaluating the effectiveness of BHR-100, a proprietary intravenous progesterone infusion formulation, as a neuroprotective agent for treating severe traumatic brain injury (TBI) patients. The U.S. Food and Drug Administration granted Orphan Drug designation to BHR-100 and placed the drug on a Fast Track status designed to accelerate its potential approval.
The trial currently has 149 participating sites (Level 1 and 2 trauma centers) worldwide. The total enrollment target is 1,180 severe TBI patients.
“This significant milestone is a testament to the work of more than 1,000 medical professionals participating in our study around the globe that are committed to advancing scientific understanding of progesterone as a neuroprotective treatment for traumatic brain injury,” said Thomas W. MacAllister, JD, PhD, and President & CEO of BHR Pharma. “Their dedicated efforts are redefining TBI standard of care and getting us closer to the world’s first-ever approvable treatment for this unmet medical need.”
TBI is a serious public health problem that affects more than 1.7 million Americans each year. Despite significant efforts in more than 75 clinical trials over the past 20 years, there is still no approved treatment for TBI.
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).
Building on the SyNAPSe study of BHR-100 and promising research conducted by Emory University, BHR is also developing BHR-310, an intranasal progesterone powder. This nasal spray is being evaluated as a potential treatment that can be administered to wounded warriors or athletes with TBI quickly after injury on the battlefield or playing field. Preclinical studies of BHR-310 in rats and monkeys support the feasibility of a high-dose, rapidly absorbed intranasal progesterone product able to deliver clinically meaningful doses of progesterone to the brain.