KN38-7271, an investigational drug to treat severe brain injury, has been shown encouraging results and clear signs of therapeutic efficacy from early phase clinical studies, according to the developer of the drug KeyNeurotek Pharmaceuticals.
KN38-7271 is a dual cannabinoid receptor agonist with a unique mode of action against CB1 and CB2 receptors.It activates CB1- and CB2-receptors on nerve and immune cells. It thereby enhances natural protection mechanisms and at the same time prevents critical inflammatory responses.
Data from phase IIa study with KN38-7271 in patients with severe traumatic brain injury (TBI) has substantiated substantiate pharmacokinetics, safety and tolerability of the drug.
Currently, there is no pharmaceutical treatment available to patients suffering from traumatic brain injuries.
The pharmacokinetic analysis of KN38-7271 clinical data demonstrated a dose-dependent blood plasma concentration, the primary endpoint of the study.
KN38-7271 was also found to be safe and well tolerated.
KN38-7271′s is the first major clinical study investigating pharmaceutical interventions in severe traumatic brain injuries.
The therapy-relevant efficacy of KN38-7271 was suggested by:improved survival rate of patients receiving the study by 30 days after start of treatment; KN38-7271-treated patients, regardless of the dose applied, showed trends toward a better neurological outcome, e.g. improved orientation and amnesia 3 or 6 months after injury and the intracranial pressure (ICP) which is usually increased in TBI patients and threatens survival of the brain, was lowered in KN38-7271-treated patients.
Also, KN38-7271 improved cerebral perfusion pressure (CPP), which promotes oxygen and glucose supply of the brain and needs to be maintained within certain limits.
The double-blind, placebo-controlled proof-of-concept study included 97 adult comatose TBI patients and was performed in three European countries, including Germany, to investigate the pharmacokinetics, safety and efficacy of two dose levels of KeyNeurotek’s lead compound KN38-7271.
“We are extremely pleased with the quality and the outcome of this very important study which has exceeded our expectation and is also encouraging for other indications such as stroke,” stated Dr Frank Striggow, CEO of KeyNeurotek Pharmaceuticals, in a press announcement.
Based on the data and the Orphan Drug Designation of KN38-7271 for the treatment of moderate and severe closed TBI, KeyNeurotek will now discuss with to the European Medicines Agency about the next steps, he added.
In September 2008, KeyNeurotek’s compound was granted Orphan Drug Designation for the treatment of moderate to severe closed traumatic brain injury by the EU authorities.
Traumatic brain injuries are usually caused by accidents or assaults, and the death rate is dramatically increasing with the severity of the injury. Moreover, survivors carry a high risk of permanent neurological disabilities. With more than 700,000 hospitalized patients in the USA and Western Europe, TBI is one of the major reasons for death and severe disabilities among younger adults. It is also one of the major injuries in military conflicts.
At present, there is no pharmaceutical treatment option available.
The potential annual market potential of drugs improving the clinical outcome of traumatic brain injuries patients has been estimated to exceed US$1 billion.
Four earlier Phase-I-studies with healthy volunteers have demonstrated that KN38-7271 is safe and well tolerated.
KeyNeurotek Pharmaceuticals, a privately held biotechnology company, was founded in 2000 within the Magdeburg region in Germany.
KeyNeurotek pursues a number of drug candidates in various preclinical and clinical stages. The most advanced compound, KN38-7271, is in clinical development for TBI and stroke.
KeyNeurotek Pharmaceuticals has unique functional and tissue-based high throughput screening platforms for compatible ex vivo and in vivo studies.
KeyNeurotek has a network of local and international partners, including the Leibniz Association, the Max Planck Society and Fraunhofer Society, Evotec, Schwarz Pharma/UCB, Bayer Schering and Gruenenthal.
KeyNeurotek has been awarded the Hugo-Junckers Innovation Award of the State of Sachsen-Anhalt in 2002 and 2004 and the Innovation Award of the German Industry in 2006.