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Early Clinical Data Reported for New Brain Injury Drug
Vasopharm GmbH, based in Wuerzburg, Germany, announced on September 17 that the explorative phase IIa NOSTRA (NO Synthase inhibition in TRAumatic brain injury) trial in patients with traumatic brain injury (TBI) met all clinical endpoints for safety and showed evidence of clinical benefit.
TBI is the leading cause of death and disability among young adults in the developed world. The condition accounts for more potential years of life lost than cancer and cardiovascular disease combined, and no drug therapies are available.
The NOSTRA trial was a European, multicenter, placebo-controlled, double-blind study with safety and tolerability as the primary endpoints. The study used in vivo microdialysis to monitor pharmacodynamic and pharmacokinetic properties of the experimental compound VAS203. VAS203 is a novel allosteric nitric oxide (NO) synthase antagonist that interrupts the inducible NO process involved in brain swelling.
Thirty-two patients with moderate to severe traumatic brain injury were enrolled at six study centers in Europe. All of the centers used continuous microdialysis to monitor cerebral energy, NO metabolism, and drug concentrations in brain tissues. VAS203 was administered in addition to standard-of-care treatment.
Final analysis of the data showed that all clinical endpoints were achieved: the drug demonstrated good safety and tolerability and reached the brain tissues in pharmacologically relevant amounts. Furthermore, the mortality rate was 12.5% in the placebo group versus 0% in the active treatment group.
The extended Glasgow Outcome Scale (eGOS) was used off-protocol to assess the level of neurological recovery achieved 6 months after injury. The eGOS was significantly increased in patients treated with VAS203 compared with those given placebo (P = 0.006), demonstrating substantially improved recovery in the VAS203 group.
Approximately 1.7 million Americans experience some degree of TBI each year, resulting in 52,000 deaths, 275,000 hospitalizations, and 80,000 cases of long-term disability. TBI poses a significant cost burden to society, with direct and indirect costs estimated to exceed $6 billion per year in the U.S.
For more information, visit the Vasopharm Web site.