A Host-targeting Medication Targeting PML

Inhibikase Therapeutics’ third product candidate, IkT-01427, is designed to block the causative virus of progressive multifocal leukoencephalopathy (PML) from replicating in the body. PML is a rare disease that causes rapidly fatal brain infection in approximately 50% of patients. Onset of PML has been linked to treatment with  medications used to treat multiple sclerosis and other autoimmune diseases, as well as in patients with HIV and certain cancers of the blood and lymph. The sole cause of PML is the John Cunningham (JC) virus.

IkT-01427 is an oral, brain-penetrant medication for systemic administration that is designed to block the entry of the JC virus into the cell, thereby preventing the virus’ ability to replicate in the body. IkT-01427 is a new molecular entity that was developed with the company’s RAMP™ technology.

We believe that IkT-01427 has an approval pathway as an anti-PML companion therapeutic for currently marketed multiple sclerosis drugs as well as additional drugs that carry a PML boxed warning, including drugs for Crohn’s disease, ulcerative colitis, and organ transplantation.

About Progressive Multifocal Leukoencephalopathy

PML is a rare disease that causes rapidly fatal brain infection. It occurs as a side effect of some medications used to treat multiple sclerosis and other autoimmune diseases as well as in patients with HIV and certain cancers of the blood and lymph. PML is caused by a human polyomavirus, the John Cunningham virus (JC virus), which resides in an inactive form in the kidney and bone marrow of more than 60% of adults. PML develops when the inactive form of the JC virus becomes activated in patients, due to chronic or drug-induced immune suppression, whereupon the virus migrates into the brain and causes irreversible brain damage, a debilitating loss of cognitive and motor function, and likely death. There is currently no approved treatment for PML. 

RAMPTM Drug Innovation Engine

Our RAMP™ (Re-engineering Approach with Metabolism Preserved) drug innovation engine enables us to design and develop novel small-molecule product candidates with enhanced potency and preserved safety profile for the treatment of neurodegenerative diseases. 

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