OSAN, South Korea--(BUSINESS WIRE)--GNT Pharma (“GNTP”) announced that the Korea Ministry of Food and Drug Safety has approved the company's Investigational New Drug (IND) application to initiate a Phase II clinical study of Neu2000 in ischemic stroke patients receiving endovascular therapy, a new interventional treatment to recanalize arteries and salvage the ischemic brain, within 8 hours of onset.
Neu2000 is a first in class multi-target drug inhibiting both N-methyl-D-aspartate (NMDA) receptor-mediated excitotoxicity and oxidative stress, two major routes of brain cell death occurring in stroke. The drug shows better efficacy and therapeutic time windows than NMDA antagonists or antioxidants alone in animal models of brain ischemia. Human safety of single and multiple ascending doses of Neu2000 has been demonstrated beyond therapeutic target doses in two phase I studies including 165 healthy young and elderly volunteers in the US and China. GNTP expects to initiate the Phase II clinical trial during Q2, 2016.
The Phase II study is a double-blinded, randomized, placebo-controlled multi-center trial designed to evaluate Efficacy and safety of Neu2000 for Ischemic Stroke patients (ENIS I). For the proof of concept study, 204 ischemic stroke patients showing Alberta Stroke Program Early CT Score (ASPECTS) of 6 or higher will be randomly assigned to receive twice-daily doses of Neu2000 or placebo for 5 days. The efficacy of Neu2000 will be evaluated by accessing neurological functions and occurrence of intracranial hemorrhage over 12 weeks. GNTP expects to complete ENIS I in 2017.
“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.” Said Byoung Joo Gwag, Ph.D., Chief Executive Officer of GNTP. “ENIS I is the first clinical trial to investigate therapeutic effects of the multi-target neuroprotectant Neu2000 for ischemia-reperfusion injury evolving after endovascular therapy. Moreover, Neu2000 is administered to stroke patients within the therapeutic time window determined in preclinical studies. In light of the well-established roles of NMDA receptor-mediated excitotoxicity and oxidative stress in ischemic brain injury, we believe that safety and efficacy of Neu2000 will be verified in such an optimized clinical study for neuroprotection therapy.”
Neu2000 was designed as the first dual neuroprotectant targeting both the NMDA receptor, a Ca2+-permeable glutamate receptor, and free radicals, two major routes of neuronal death in stroke and trauma. Neu2000 is a moderate NR2B-selective NMDA receptor antagonist and spin trapping molecule (=free radical scavenger or antioxidant). Therapeutic potential of Neu2000 has been demonstrated in four animal models of stroke with better efficacy and therapeutic time windows than either NMDA receptor antagonist or anti-oxidants advanced to clinical trials. Neu2000 also showed prominent efficacy in animal models of spinal cord injury, acute myocardial infarction, and burn injury. Nonclinical and human phase I studies in the United States and China demonstrate that Neu2000 is remarkably safe and thus expected to show promising outcome in proof of concept (POC) studies for acute ischemic stroke patients.
About GNT Pharma
The founding scientists of GNT Pharma have delineated mechanisms underlying nerve cell death occurring in stroke, trauma, and neurodegenerative diseases and discovered therapeutic targets for the interventional therapy that is for medical unmet needs. The drug candidates of GNT Pharma were derived from novel findings that aspirin and sulfasalazine, salicylate derivatives widely used for the treatment of inflammatory diseases, protected neurons as a low affinity NMDA receptor antagonist and antioxidant. Through the rational drug design, GNT Pharma discovered chemical structures essential for neuroprotective effects and has established chemical libraries with promising efficacy and safety that are enough to advance to clinical stage.
Forward Looking Statements
This press release contains forward-looking statements regarding GNTP, including statements about GNTP's expectations regarding the company's proposed Phase 2 clinical trial and investigational new drug (IND) application for GNTP, as well as GNTP's potential to produce therapeutic benefits. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of GNTP's product candidate development activities and clinical trials; and risks regarding regulatory requirements, among others. These forward-looking statements speak only as of the date hereof. GNTP disclaims any obligation to update these forward-looking statements.