BHR Pharma, LLC announced today that the SyNAPSe® Trial's independent Data and Safety Monitoring Board (DSMB) evaluated safety data from 803 randomized patients and determined that no issues exist; therefore, the trial may continue towards its intended completion.
The SyNAPSe® Trial is evaluating BHR-100, a proprietary intravenous progesterone lipid emulsion product as a neuroprotective agent to treat severe traumatic brain injury (TBI). If proven effective, BHR-100 could be the first-ever approved treatment for TBI.
In addition, based on the formal interim analysis of primary six-month efficacy data from 400 SyNAPSe patients, the DSMB concluded that:
- There was no reason to stop the study for futility
- The interim analysis results were not statistically significant at the threshold (p-value = 0.0003) for stopping the trial early
The company said that it anticipated the DSMB interim analysis primary endpoint findings, as it purposefully set the threshold for stopping the trial based on efficacy extremely high.
"We are feeling very positive about the DSMB's analysis and are eager to complete enrollment as soon as possible, so we can we can truly determine if BHR-100 can be the world's first-ever approvable treatment for TBI," said Thomas W. MacAllister, BHR Pharma President and CEO. “This significant trial milestone is a testament to the work of more than 1,000 medical professionals participating in our study worldwide that are committed to advancing scientific understanding of progesterone as a neuroprotective treatment for traumatic brain injury."
The U.S. Food and Drug Administration granted BHR-100 an Orphan Drug designation and placed the drug on Fast Track status designed to accelerate its potential approval.
The SyNAPSe® Trial currently has 154 participating sites, including U.S. Level 1 and 2 trauma centers, in 21 countries worldwide. The total enrollment target is 1,180 severe TBI patients.
TBI is a serious public health problem that affects more than 1.7 million Americans each year – more than all U.S. cases of multiple sclerosis, AIDS, breast cancer and spinal cord injuries put together. Despite significant efforts and 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).
About BHR Pharma
Founded in January 2008, BHR Pharma, LLC (www.bhr-pharma.com) is a pharmaceutical research and development company located near Washington, DC. BHR is committed to bringing to market specialty treatments that employ non-oral delivery systems, with an emphasis on unmet and underserved medical needs. The company is a wholly owned subsidiary of Besins Healthcare SA (www.besins-healthcare.com), which markets healthcare products in 93 countries.