Interim Report January – September 2019
Traumatic brain injury (TBI) is caused by external force to the head resulting in immediate damage to nerve cells. The damage continues to worsen for several days after the acute trauma. The most common causes for TBI are trips and falls, traffic accidents and assault and battery.1) With more than 50 million new cases occurring each year, TBI is estimated to cost the global economy nearly 400 billion dollars annually in direct and indirect healthcare costs.2) A large number of patients suffer moderate to severe functional disabilities requiring intensive care and various forms of lifelong support.
The aim for NeuroVive’s candidate drug for TBI treatment, NeuroSTAT, which targets the mitochondria, is to counteract the emergence of neurological and functional secondary brain damage after a traumatic injury, and thereby establish a therapy that will lead to increased survival, improved quality of life and preserved neurological function. NeuroSTAT has shown favorable properties in a Phase II clinical study that investigated safety, tolerability, pharmacokinetics, i.e. the chemical metabolism, and passage to the brain, of two different doses of the active ingredient ciclosporin in patients with severe traumatic brain injury. Further, analyses of brain cell damage biomarkers in samples from the patients, gave a first signal of clinical effect.
In addition, in advanced experimental TBI models at the University of Pennsylvania (Penn), a 35% decrease in volume of brain injury was observed after NeuroSTAT treatment, as well as positive changes in brain energy metabolite levels and mitochondrial respiratory function, together with decreased generation of reactive oxygen species.
NeuroSTAT has orphan drug designation both in Europe and the US, as well as an IND approval for clinical development in the US. In addition, In July 2019, NeuroSTAT received Fast Track designation from the FDA.
2) Maas A et al. Traumatic brain injury: integrated approaches to improve prevention, clinical care, and research. The Lancet Neurology. 2017 Nov; 16(12):987