GNT Pharma (GNTP) has received approval from the Korean Ministry of Food and Drug Safety to initiate a phase 2 clinical trial of Neu2000 in ischemic stroke patients receiving endovascular therapy, a new interventional treatment to recanalise arteries and salvage the ischemic brain, within 8h of onset.
Neu2000 is a multi-target drug inhibiting both N-methyl-D-aspartate (NMDA) receptor-mediated excitotoxicity and oxidative stress, which are two major routes of brain cell death occurring in stroke.
The drug is claimed to show improved efficacy and therapeutic time windows than NMDA antagonists or antioxidants alone in animal models of brain ischemia.
GNTP stated that human safety of single and multiple ascending doses of Neu2000 has been demonstrated beyond therapeutic target doses in two phase 1 trials, including 165 healthy young and elderly volunteers in the US and China.
The company expects to initiate the phase 2 clinical trial during the second quarter of this year.
The double-blinded, randomised, placebo-controlled multi-centre trial designed to evaluate efficacy and safety of Neu2000 for ischemic stroke patients.
For the proof of concept study, 204 ischemic stroke patients showing Alberta Stroke Programme Early CT Score (ASPECTS) of six or higher will be randomly selected to receive twice-daily doses of Neu2000 or placebo for five days.
The efficacy of Neu2000 will be evaluated by accessing neurological functions and occurrence of intracranial hemorrhage over a period of 12 weeks.
GNTP expects to close the study by next year.
GNTP chief executive officer Byoung Joo Gwag said: "Although almost 200 clinical trials for stroke patients with neuroprotectants have been conducted over the last two decades, none of them showed beneficial effects.
"Such failure is largely attributable to unwanted side effects of the neuroprotectants in human and also heterogeneity of patients including both transient (ischemia-reperfusion) and permanent ischemic strokes in clinical trials conducted with the neuroprotectants while the beneficial effects of the neuroprotectants were well documented in animal models of ischemia-reperfusion brain injury."