Pharmaceutical Business review

Inhibikase secures FDA orphan drug status for imatinib to treat PML

PML is a rare side effect of small molecule and antibody drugs given to patients with autoimmune diseases such as arthritis and multiple sclerosis (MS).

It also occurs in 1-3% of clinical acquired immune deficiency syndrome (AIDS) patients.

The company said that certain drugs used to treat autoimmune disease suppress the ability of a patient to fight infection, particularly for John Cunningham (JC) virus.

JC exist inside most people, but when the immune system is suppressed, it 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.

MS Center of Atlanta director of Clinical Research neurologist Jeffrey English said multiple Sclerosis (MS) can be a very disabling disease and to date, Tysabri is its most effective treatment.

"Unfortunately, it carries a risk of a life threatening brain infection that can lead to PML," English said.

"There are ways to screen for PML early, but we have no effective treatments for this disease.

"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."

The company said that imatinib is a host-directed protein kinase inhibitor that disrupts the ability of JC virus to reproduce in the patient.

Imatinib is the active ingredient in the company’s lead product IkT-001Pro as well as in the anti-cancer drug Gleevec, developed by Novartis.


Image: Imatinib is also the active ingredient in the anti-cancer drug Novartis’ Gleevec. Photo: courtesy of BrandonSigma/ freedigitalphotos.net