Inhibikase Wins Orphan Drug Status For Brain Infection Treatment
Inhibikase Therapeutics announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for imatinib as treatment for Progressive Multifocal Leukoencephalopathy (PML). Inhibikase is an emerging firm focused on developing drugs to treat infectious diseases with little or no resistance.
Progressive multifocal leukoencephalopathy is a rare side effect of small molecule and antibody drugs administered to patients with multiple sclerosis and other autoimmune diseases. PML also occurs in 1 to 3 percent of patients with clinical AIDS. Some drugs used against autoimmune diseases also weaken the patient’s ability to fight infection, in particular a virus called John Cunningham (JV) virus. The virus survives inside most people but sometimes migrates to the brain when the immune system is suppressed. There, JC ‘blows up’ certain brain cells which can cause loss of cognitive and motor neuron function. The medical outcome in these cases is often fatal.
According to neurologist Dr. Jeffrey B. English, Director of Clinical Research at the MS Center of Atlanta, finding a way to treat PML would make it possible for more MS patients to take Tysabri, currently the best drug available, but which carries the risk of PML.
Imatinib is a host-directed protein kinase inhibitor that disrupts the JC virus’ reproductive ability. It is the active ingredient in Inhibikase’s lead product IkT-001Pro as well as in Novartis AG’s Gleevec for blood and stomach cancer. IkT-001Pro is designed to deliver imatinib to its targets while reducing the dose and improving the substance’s efficacy against the cause of PML. “The anti-JC virus activity of imatinib cannot be achieved by simply altering the frequency or amount of Gleevec given to patients. Early trial work has already shown this. To succeed, we're talking reengineering how imatinib is absorbed and distributed in the body,” said Milton H. Werner, President and CEO of Inhibikase.
Dr. Werner said that the FDA granting of Orphan Drug Designation to IkT-001Pro is a milestone in the drug’s development. “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,” he said.