Orphan Drug Designation Granted to Treatment for Rare Multiple Sclerosis Drug Complication

The Food and Drug Administration granted Orphan Drug Designation to Inhibikase Therapeutics’, Inc. lead product, IkT-001Pro (imatinib), to treat progressive multifocal leukoencephalopathy (PML). According to the National Institute of Neurological Disorders and Stroke, PML affects the white matter of the brain usually through a virus known as Polyomavirus JC (JC virus). If left untreated, JC virus can destroy motor neurons and cognitive ability, eventually leading to death. Multiple sclerosis patients are particularly prone to PML due to their lowered immunity from their disease and use of certain drugs.

“Multiple sclerosis can be a very disabling disease.  To date, Tysabri® is our most effective treatment,” explained Dr. Jeffrey B. English, Director of Clinical Research at the MS Center of Atlanta, in a news release. “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.”

To address this need, Inhibikase has been researching and developing imatinib for delivery to patients with PML. Imatinib is a host-directed protein kinase inhibitor that interferes with JC virus replication. It has also been implicated in Novartis AG’s anti-cancer drug Gleevec® to treat blood and stomach cancers. To safely deliver imatinib to PML patients by reducing the required dose and side effects, Inhibikase formulated imatinib using its proprietary technology to make IkT-001Pro. “The anti-JC virus activity of imatinib cannot be achieved by simply altering the frequency or amount of Gleevec given to patients,” said 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.”

Since Inhibikase is essentially developing a new drug, it must go through clinical trials, which can be a burden for diseases with small populations. “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.”

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According to Dr. English, a treatment like IkT-001Pro could open Tysabri prescription to many more multiple sclerosis patients, allowing more people to benefit from the most effective treatment for multiple sclerosis. “PML can pose a grave risk to people with multiple sclerosis 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,” explained Dr. Timothy Coetzee, Chief Advocacy, Services and Research Officer at the National MS Society.

“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,” concluded Dr. Werner.