It is almost unheard of for a self-funded pharmaceutical company to discover a drug and prepare to enter it into Phase 3 clinical trials, but Winston Ko, founder of Genervon Biopharmaceuticals LLC, a biotech company in Pasadena, California, has succeeded in doing that.
As a result, Ko is in the middle of publicising his company’s line of drugs that target diseases hitting the central nervous system.
Ko speaks at prestigious drug industry conference
Ko’s campaign looks as if it could be quite successful given the news three months ago that Pfizer Inc. (NYSE:PFE) plans to end its research efforts to discover new drugs for Alzheimer’s and Parkinson’s diseases. Pfizer’s retreat from these areas throws up the opportunity for Genervon to introduce GM6, its lead drug which treats multiple symptoms of a range of deadly diseases -- from Alzheimer’s, Parkinson’s and Huntington’s to amyotrophic lateral sclerosis (ALS) and multiple sclerosis.
Indeed, Ko was invited to speak on not one subject, but two at this month’s 20th annual meeting of the American Society for Experimental Neurotherapeutics (ASENT), whose goal is to advance the development of improved therapies for diseases and disorders of the nervous system. While there, he presented Genervon’s therapy for ALS, also knows as Lou Gehrig's disease, and illustrated how GM6 affects multiple pathological conditions in ALS and other neurodegenerative diseases. Ko also presented Genervon’s full pipeline based on its GM6 endogenous regulator platform, which may regulate multiple symptoms of patients suffering from diseases of the central nervous system.
In science parlance, GM6 is an endogenous embryonic stage multi-target cationic regulator peptide consisting of six amino acids and it forms the basis for Genervon’s entire line of drugs.
It has been 20 years since the company’s scientists looked for and discovered what they believe is the regulator of the development of the human nervous system, called the MNTF (motoneuronotrophic factor) and its active fragment, GM6. MNTF is present in the body since conception and reaches its highest levels in week nine of a fetus, but remains present in adult tissue. This explains why humans do not reject the injection of MNTF or its fragments like GM6 for therapeutic purposes.
“The body recognizes this regulator. So, it can be reintroduced for healing and repair,” explains Ko. “Humans aren’t smart enough to design a multi-target drug”.
GM6: A multi-target drug
From the start, Genervon scientists have argued that a multi-target drug that addresses a range of symptoms of a disorder -- not the traditional single-target one, which zeroes in on one factor of the disease -- is the answer to curing central nervous system (CNS) and neurodegenerative (ND) diseases and disorders, which have multiple biological causes and symptoms.
The focus on the development of single-target drugs continues to result in clinical trial failures, says Ko.
Genervon has shown some promise in proving the effectiveness of its drugs. A proof-of-concept, randomized, placebo-controlled Phase 2A trial of GM604 -- the version created for Lou Gehrig’s disease -- was conducted at Massachusetts General Hospital and New York Presbyterian/Columbia University Medical Center, with 12 patients suffering from ALS reporting positive signals despite the small trial size.
“The trial … clearly demonstrates that the genes involved in ALS, and the biomarkers of the disease showed favourable expression shift upon treatment with GM604,” according to peer review comments published in F1000Research, an open research publishing platform for life scientists. “This also shows the evidence that the GM604 was able to alter the expression of certain ALS genes and its biomarkers in the positive direction, an evidence not shown before with any drug in the market for ALS treatment.”
Genervon’s scientists are pushing ahead with the Phase 3 trial of GM604 under guidelines established by the U.S. Food and Drug Administration.
Genervon -- which holds more than 80 global patents for MNTF as well as GM6 and its related drug varieties -- is working on conducting a Phase 3 trial for Lou Gehrig’s disease with GM604 to meet the FDA’s requirements and is Phase 2 ready for multiple sclerosis, Parkinson’s, Alzheimer’s, and Huntington’s diseases.
Opportunity in the drug industry
Given the shift away from drugs for Alzheimer’s and Parkinson’s disease by pharmaceutical companies, Ko thinks this may be the time for the industry’s acceptance of Genervon’s therapies and hopes other drug companies will work with Genervon to bring GM6 to market.
“What distinguishes GM6 as a curative and transformational therapeutic is that it is an endogenous, multi-target regulator peptide drug.” Ko concludes, “After 20 years of funding our own discovery, R&D, and drug development, Genervon is ready for partners in the pharmaceutical industry to help advance GM6 from a development-stage drug-candidate to marketable therapies for the many victims of neurodegenerative diseases and disorders who desperately need help.”