Inhibikase Therapeutics Receives Orphan Drug Designation for Treatment of Progressive Multifocal Leukoencephalopathy (PML)

Attacking a fatal brain infection with a novel therapy

Atlanta, GA – May 21, 2014 – Inhibikase Therapeutics, Inc., an emerging developer of products to treat infectious diseases with little or no resistance, announces today that it has received Orphan Drug Designation for imatinib to treat Progressive Multifocal Leukoencephalopathy (PML) from the U.S. Food and Drug Administration.

PML is a rare side effect of small molecule and antibody drugs given to patients with autoimmune diseases like arthritis and multiple sclerosis (MS); PML also occurs in 1-3% of clinical AIDS patients. Certain drugs used to treat autoimmune disease suppress the ability of a patient to fight infection, particularly for the virus known as JC (for John Cunningham virus).  JC lives inside most people, but when the immune system is suppressed, JC can occasionally migrate into the brain, ‘blowing up’ certain brain cells that results in a debilitating loss of cognitive and motor neuron function, often culminating in a patient’s death.

“Multiple Sclerosis (MS) can be a very disabling disease.  To date, Tysabri® is our most effective treatment,” noted neurologist Dr. Jeffrey B. English, Director of Clinical Research at the MS Center of Atlanta.  “Unfortunately, it carries a risk of a life threatening brain infection that can lead to PML.  There are ways to screen for PML early, but we have no effective treatments for this disease,” commented Dr. English.  “If there was a way to treat PML, this would open up a pathway for many more patients to receive Tysabri®, the most effective treatment for MS.”

Imatinib, the active ingredient in the Company’s lead product IkT-001Pro, is a host-directed protein kinase inhibitor that disrupts the ability of JC virus to reproduce in the patient.  IkT-001Pro delivers imatinib to its targets using a proprietary technology that should reduce the dose and side effects of imatinib therapy while simultaneously enhancing imatinib’s ability to suppress the causative virus of PML.  Imatinib is also the active ingredient in the anti-cancer drug Gleevec®, developed by Novartis AG and used to treat certain forms of blood and stomach cancer.  “The anti-JC virus activity of imatinib cannot be achieved by simply altering the frequency or amount of Gleevec® given to patients,” noted Milton H. Werner, PhD, President and CEO of Inhibikase.  “Early trial work has already shown this.  To succeed, we’re talking reengineering how imatinib is absorbed and distributed in the body.”

“The granting of Orphan Drug Designation is a pivotal milestone in the development of IkT-001Pro to treat this rare and debilitating illness,” said Dr. Werner.  “The Designation will enable resources for continued development, but more importantly the Designation provides an additional avenue for discussion with the FDA on the best path for bringing IkT-001Pro to market. PML is a rare side effect of at least 13 beneficial medications, and PML may also arise as a side effect of many more medications now in clinical development. IkT-001Pro, when administered as a companion therapeutic, could reduce the risks of these marketed and investigational treatments, thereby improving patient safety and focusing treatment decisions on the efficacy of the treatment, not just on the risk of an unintended and potentially fatal side effect.”

“PML can pose a grave risk to people with MS and other disorders who use powerful immune-modulating therapies, and this risk often forces people to avoid or limit the use of otherwise very effective treatments,” commented Dr. Timothy Coetzee, Chief Advocacy, Services and Research Officer at the National MS Society. “This Orphan Drug Designation by the FDA should provide incentive to develop a program focusing on the unmet need of a treatment for PML.”

About Inhibikase Therapeutics, Inc.

Incorporated in 2010, Inhibikase Therapeutics is a privately held specialty pharmaceutical company developing novel, small-molecule treatments for bacterial and viral infectious diseases. Headquartered in Atlanta, Georgia with operations in Atlanta, Georgia and Cambridge, Massachusetts, Inhibikase’s products block pathways in patients to disrupt the ability of bacteria or viruses to reproduce in the patient.  So-called ‘host-targeted’ anti-infective treatments have a lower likelihood of stimulating resistance and can result in vaccine-like outcomes. The Company’s pipeline is focused on antiviral treatments for rare infections of the brain, kidney and liver as well as on the development of Medical CounterMeasures for biodefense.

About Multiple Sclerosis

Multiple sclerosis, an unpredictable, often disabling disease of the central nervous system, interrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease.  According to the National MS Society, MS affects more than 2.3 million worldwide.

About the National Multiple Sclerosis Society

The Society mobilizes people and resources to drive research for a cure and to address the challenges of everyone affected by MS.  To fulfill this mission, the Society funds cutting-edge research, drives change through advocacy, facilitates professional education, collaborates with MS organizations around the world, and provides programs and services designed to help people with MS and their families move their lives forward.  To move us closer to creating a world free of MS, last year alone, the Society invested over $48 million to support more than 380 new and ongoing research projects around the world while providing program services to over one million people.

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