Single pharmaceutical-based strategy aims to solve neurological and psychiatric disorders Print E-mail
By M.E.Garza   
Wednesday, 19 October 2011 05:26

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

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. 


BioMedReports: And AMPAKINE compounds may also prove useful for those with the symptoms of ADHD?

Mark Varney, CEO of Cortex Pharmaceuticals: Correct. Again, the key idea here is the ability of AMPAKINE compounds to increase levels of neurotransmitters in parts of the brain that help people focus and control impulses, and to activate brain regions that are sluggish so that they regulate cognitive activity at a more normal level. Now, there are a host of drugs already on the market—including Ritalin, Adderall, Concerta and Vyvanse—that operate on these principles. However, these medications belong to the class of drugs known as stimulants, and have an increased risk for addiction, and in some patients, lead to unacceptable increases in heart rate and blood pressure. In contrast, AMPAKINE compounds have the potential to be unique agents in treating ADHD, because they lack the side-effect liabilities of existing treatments.

BioMedReports: Additionally, Cortex believes AMPAKINE compounds will find use in treating the symptoms of central sleep apnea. Could you brief us on this endeavor?

A: The majority of patients with congestive heart failure experience sleep apnea—multiple interruptions of breathing that last 10 seconds or more while they sleep. The repeated periods of lack of breathing, often many hundreds of times throughout the night, add additional strain to an already compromised heart, resulting in a poor outcome for the patients. It is now clear from following these patients over time that those with severe sleep apnea have a worse outcome, often requiring a heart transplant. There is currently no treatment for these central sleep apnea events. In a pilot study in healthy individuals who suffered from sleep apnea, one of Cortex’s AMPAKINE molecules reduced certain measures of sleep apnea.  The AMPAKINE molecule does this by stimulating a unique part of the brain that controls breathing. Put in a simpler way, AMPAKINE compounds work by telling the brain to keep on breathing, and also amplifying the signals from the brain to the upper airway muscles to maintain muscle tone.  

BioMedReports:  Can you tell us a bit more about how the AMPAKINE compounds can treat respiratory depression?

Mark Varney, CEO of Cortex Pharmaceuticals: In those who take opioid painkillers legally or otherwise, breathing can slow and in some cases stop entirely.  In 2009 we detailed how our AMPAKINE CX717 demonstrated the rescue of respiratory depression in rats and subsequently we have shown that the compound can prevent the onset of opioid-induced respiratory depression in humans without interfering with the beneficial analgesic effects of opioids.  In this study, CX717 demonstrated equal efficacy with the opioid-antagonist Naloxone, a drug used to counter the effects of opioids on suppression of breathing.  CX717 did not, however, interfere with the action of pain-killing opiates.  This offers a distinct advantage compared with Naloxone and could provide a novel therapeutic means of treating those patients who are particularly prone to breathing depression with opiates while achieving maximum pain relief. We hope AMPAKINES may one day serve as rescue therapies for patients exhibiting respiratory depression, or perhaps an adjuvant to painkillers.

BioMedReports:  The trial of Michael Jackson’s doctor, now underway in Los Angeles, has turned the public attention to the issue of respiratory depression.  Could AMPAKINE compounds have played a role in saving a person like him?  

Mark Varney, CEO of Cortex Pharmaceuticals:  Possibly.  Michael Jackson was injected with propofol (Diprivan), a very dangerous drug that could have led to respiratory depression and subsequently cardiac arrest.  If respiratory depression occurs while under the supervision of an experienced anesthesiologist, the outcome can usually be managed to ensure there will be minimal long-term effects to the patient.  Unfortunately, there are many situations during both in-patient and out-patient procedures where strong analgesic therapy is required but there is no attending anesthesiologist.     

BioMedReports: Are manufacturers of CPAP devices aware of your efforts?

Mark Varney, CEO of Cortex Pharmaceuticals:
Yes, in fact we have had discussions with some CPAP manufacturers to explain to them what our AMPAKINE compounds are designed to do. CPAP devices represent a huge business, with sales of at least one billion dollars annually. We certainly hope that as our molecules undergo further clinical testing on the road to market, that they will one day gain the prominence among healthcare providers and obstructive sleep apnea patients as CPAPs currently enjoy.       

BioMedReports: Who do you see as your chief competitors, and what is the current status of the clinical testing involving Cortex’s AMPAKINE compounds?

Mark Varney, CEO of Cortex Pharmaceuticals: At this time, we at Cortex do not see any genuine competition in our quest to develop molecules that can be used as widely and as effectively as our AMPAKINE compounds. As for clinical testing, we currently have two compounds in clinical development, CX717 and CX1739, the former for drug-induced respiratory depression and the latter for sleep apnea and ADHD. We also have a robust portfolio of earlier-stage compounds that are awaiting clinical development.     

BioMedReports: In conclusion, what would you say to those who either experience the symptoms of Parkinson’s, Fragile X, ADHD or obstructive sleep apnea, or know someone who does? And what message do you have for potential investors?

Mark Varney, CEO of Cortex Pharmaceuticals: Those who currently have one of these conditions or who have friends or family with them should be aware of cutting-edge research developments such as the ones we have been discussing. Although at present there is no firm timeframe for bringing Cortex’s AMPAKINE compounds to market, we are all looking forward to a day in the not-too-distant future when these compounds may offer a very substantial alternative or supplement to existing strategies. An important message should also be sent to healthcare providers, who stand to reap potentially big savings if AMPAKINE technology becomes standard. We feel our AMPAKINE compounds will tempt physicians to take a fresh look at the treatment options available to them. Keep in mind that the total cost of these ailments involves more than just the immediate price of existing treatments themselves. Dealing with the complications that sometimes result from these conditions can be more expensive than anyone expects. When hospitals see the potential savings involved in switching to our AMPAKINE compounds as a standard treatment, we think they will be enthusiastic. 

As for potential investors, I would stress the fact that Cortex Pharmaceuticals’ goals are broader than merely improving the lives of those with any one specific condition. We are a neuroscience company focused on the discovery, development and commercialization of novel drug therapies for the treatment of a variety of neurological and psychiatric disorders. While we have been discussing specific areas of AMPAKINE therapy here, the fact is that they can potentially do much more. It is a very exciting time to be working in this field, and we at Cortex are looking forward to developing our technology and giving a wide range of patients new hope for the future

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