Patient samples from the previously completed CHIC study have been analyzed in a research collaboration with Kevin K.W. Wang, Ph.D., at the University of Florida. The aim of the collaboration is to develop innovative endpoints for NeuroVive’s clinical traumatic brain injury (TBI) program.
The GFAP, UCH-L1, NF-L and Tau biomarkers, which reflect different aspects of the ongoing brain cell damage following TBI, were measured in repeated samples of cerebrospinal fluid from the severe trauma patients in NeuroVive’s CHIC study. The results show a time-based change in biomarker levels correlating with NeuroSTAT drug administration for all four biomarkers. These biomarkers are being developed with the aim of improving diagnosis, enhancing short and long-term patient care and assisting clinical trials developing new treatments for TBI.
“This early efficacy signal indicates that NeuroSTAT suppresses the secondary brain injury cascade and clearly supports our continued development of NeuroSTAT for TBI. The results from this fruitful collaboration are very promising in relation to our efforts to optimize efficacy measurement in TBI drug development and bring the much-needed treatment options to these patients,” commented Magnus Hansson, Chief Medical Officer and VP of Preclinical and Clinical Development at NeuroVive.
The company plans to use these innovative biomarkers as outcome measures together with other new endpoints to establish proof of concept for NeuroSTAT in its next larger randomized, placebo-controlled phase II efficacy study, planned to start in 2019.
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About biomarkers in TBI drug development
The use of biofluid-based biomarkers in TBI drug development is increasingly recognized as being of utmost importance for diagnosis, prognosis and therapy evaluation. GFAP (Glial fibrillary acidic protein), UCH-L1 (Ubiquitin carboxy-terminal hydrolase L1), NF-L (Neurofilament Light) and Tau are proteins found in different cells and cellular compartments of the brain. They are released following injuries to astrocytes, neurons and axons, respectively, and their levels in blood and cerebrospinal fluid have been correlated to the severity of several different brain diseases. The FDA has recently approved a GFAP and UCH-L1-based blood test for diagnosis of mild TBI. 1)
About traumatic brain injury (TBI)
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. 2) 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. 3) A large number of patients suffer moderate to severe functional disabilities requiring intensive care and various forms of support.
3) Maas A et al. Traumatic brain injury: integrated approaches to improve prevention, clinical care, and research. The Lancet Neurology. 2017 Nov; 16(12):987.
About the Phase IIa clinical study CHIC and NeuroSTAT
The phase II CHIC (Copenhagen Head Injury Ciclosporin) study was an open label study. The primary objective with the study was to establish safety and to characterize the pharmacokinetic profile of two dosing regimens of NeuroSTAT in severe Traumatic Brain Injury (TBI) patients. In addition, exploratory measurements to evaluate the efficacy of NeuroSTAT at mitochondrial level, and studies on how NeuroSTAT affects various biochemical processes after a brain injury, were conducted. Principal Investigator for the study is Jesper Kelsen, MD, PhD, Specialist in Neurosurgery, Department of Neurosurgery, Rigshospitalet, Copenhagen University Hospital.
NeuroSTAT has been shown to reduce the volume of brain injury by 35% in a pre-clinical trial using an advanced experimental model of TBI. 4) NeuroSTAT has orphan drug designation in both Europe and the US. NeuroVive has also partnered with TRACK-TBI, a network of leading TBI researchers in the US with the mission to improve clinical trials in TBI.
4) Karlsson et al. J Neurotrauma. 2018: https://www.liebertpub.com/doi/10.1089/neu.2018.5706
NeuroVive Pharmaceutical AB is a leader in mitochondrial medicine, with one project in clinical phase II development for the prevention of moderate to severe traumatic brain injury (NeuroSTAT®) and one project in clinical phase I (KL1333) for genetic mitochondrial diseases. The R&D portfolio also consists of projects for genetic mitochondrial disorders, cancer and NASH. The company advances drugs for rare diseases through clinical development into the market. For projects for common indications the goal is out-licensing in the preclinical phase. A subset of compounds under NeuroVive’s NVP015 program has been licenced to Fortify Therapeutics, a BridgeBio company, for local treatment development of Leber’s Hereditary Optic Neuropathy (LHON). NeuroVive is listed on Nasdaq Stockholm, Sweden (ticker: NVP). The share is also traded on the OTCQX Best Market in the US (OTC: NEVPF).