An emerging microcap firm out of Irvine, California is developing novel drug therapies for the treatment of neurological and psychiatric disorders.

Cortex Pharmaceuticals, Inc.     0.09 +0.02 (+28.57%) Intraday 3 Month 6 Month 1 Year

The medical research community is engaged in a tireless quest to treat a wide variety of ailments that range in symptoms and complexity from Parkinson’s Disease and Fragile X syndrome to ADHD and obstructive sleep apnea.  Thus it might seem incredible to hear that a single pharmaceutical-based strategy could possibly be harnessed to address these maladies.   However, this is exactly what one pharmaceutical company, Cortex Pharmaceuticals (OTCBB: CORX), is now betting on.

The company has overseen the development of a class of molecules called AMPAKINE® compounds that may provide protection from these ailments as well as a range of others. We took time to reach out to Mark Varney, President and CEO of Cortex Pharmaceuticals, for some clarification on the company’s products and plans.

BioMedReports: Before we get into a discussion of their specific potential applications, can you say a little bit about what AMPAKINE compounds are and what effects they have on the human brain?

Mark Varney, CEO of Cortex Pharmaceuticals: The AMPAKINE compounds are a class of proprietary pharmaceuticals that act through a two-pronged approach. They increase the strength of signals at connections between brain cells, and stimulate the production and release of certain growth factors in the brain. AMPAKINE molecules interact in a highly specific manner with proteins in the brain called AMPA receptors. These receptors are activated by the neurotransmitter glutamate, the most prominent excitatory neurotransmitter in the brain. AMPAKINE compounds facilitate the response to glutamate, essentially amplifying the normal level of signaling between neurons. Research by Cortex and its collaborators, including Professors Gary Lynch and Christine Gall from the University of California, Irvine, have demonstrated that AMPAKINE molecules also stimulate the production and release of certain growth factors in the brain, including brain-derived neurotrophic factor (BDNF). BDNF is essential for maintaining cell health in the normal brain, and plays an important role in restoring brain function following damage to the brain. Through elevating BDNF in damaged brain regions, AMPAKINE compounds may restore function to previously damaged areas.

BioMedReports: Let’s begin with Parkinson’s disease. What is the status of Cortex’s efforts in this area?

Mark Varney, CEO of Cortex Pharmaceuticals: We have been awarded a grant by The Michael J. Fox Foundation for Parkinson’s Research to test selected compounds from our AMPAKINE platform for their ability to restore brain function in animal models for Parkinson’s disease (PD). We aim to test our selected AMPAKINE drug candidates in the mouse model of Parkinson’s, a well-validated model that exhibits many of the hallmarks of human PD and has been used extensively for drug development in PD. If successful, the work could lead to a neuroprotective treatment for the disease with the potential to slow or stop the course of the disease—something no currently available therapy has been proven to do. Current treatments for PD alleviate the symptoms but do not attack the underlying disease, or alter its course. Positive results will support moving selected compounds toward human clinical trials.

BioMedReports: Moving to Fragile X syndrome, how are you attempting to address this condition, which is the most common genetically proven cause of autism, with AMPAKINE compounds?

Mark Varney, CEO of Cortex Pharmaceuticals: We have been granted an exclusive license by the University of California and a patent application for the combination of two substances that have shown promise in alleviating Fragile X symptoms, which can range from fidgeting and impulsive actions to epilepsy, OCD, and autism or autistic-like behavior. The first of these two substances are AMPAKINE compounds, which, as I noted earlier, serve to increase the strength of signals at connections between brain cells and increases levels of BNDF. The second class of substance bears the name “metabotropic glutamate receptor type 5 antagonists,” better known as mGluR5 antagonists. These appear to amplify the positive effects of the AMPAKINE compounds in alleviating Fragile X symptoms. Early clinical studies with mGluR5 antagonists have shown promising results in Fragile X patients, and in animal studies the combination of these agents with our AMPAKINE compounds has been seen to provide additional benefit via a synergistic mechanism. If these effects hold up in clinical studies, the combination could be an important treatment option. 

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