BHR Pharma said Tuesday that it’s completed enrollment in a Phase III safety and efficacy study of its progesterone formulation, BHR-100, as a neuroprotective agent for people who experience moderate to severe traumatic brain injury.
Started back in June of 2010, the SyNAPSe trial has extended the experimental therapy to nearly 1,200 TBI patients at 150 participating sites in the U.S., Israel, Argentina and 18 other countries in Europe and Asia. Patients in the trial received a five-day continuous IV infusion of progesterone beginning within eight hours of injury, the company says.
The primary goal is to see improved outcomes six months after injury using the Glasgow Outcome Scale. Results for the trial, which has been done in conjunction with the American Brain Injury Consortium and the European Brain Injury Consortium, are expected in May 2014. A company representative could not be reached for additional comments on Tuesday.
Although it’s most commonly known for its role in regulating certain functions of ovulation and menstruation in women, progesterone therapy has also been found to affect the central nervous system of both men and women. In research studies, progesterone has demonstrated neuroprotective effects resulting in less brain swelling, less cell death in the brain and increased survival compared to a placebo.
If the drug meets its endpoints in this trial, it could be on its way to becoming the first drug treatment for TBI, which results in an estimated 1.5 million visits to the emergency room and 52,000 deaths in the U.S. each year. Current treatments for TBI involve emergency surgery or focus on preventing further injury, relieving symptoms or promoting rehabilitation.
Despite scientists’ great strides in understanding the course of TBI over the last few decades, a slew of promising pre-clinical therapies have failed to live up to expectations in human trials. Despite working against a backdrop of failed clinical trials, BHR has a few things going for it. Don Stein, the Emory University professor behind the work in TBI and progesterone, told the Washington Business Journal in 2009 that the treatment works at a number of different levels rather than targeting a specific mechanism, as most other trials have.
Regulators in the U.S. and EU have both given orphan drug status to BHR Pharma’s candidate, which was licensed from Emory University in 2009. The company formed near Washington D.C. in 2008 as an R&D subsidiary of Besins Healthcare, which markets progesterone, estrogen and testosterone therapy products in more than 90 countries.
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