IkT-01427 for PML

Companion Therapeutic to Remove the Risk of PML

Our second product candidate, IkT-01427, blocks the causative virus of PML (Progressive Multifocal Leukoencephalopathy) from replicating in the body. PML is a rare disease that causes rapidly fatal brain infection in 30%-60% of patients. PML occurs as an unintended side effect of more than 15 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 (see sidebar). The sole cause of PML is John Cunningham (JC) virus infection.

IkT-01427 is an oral, brain-penetrant, high-potency 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 chemical entity that was developed with the company’s RAMP technology and is 40 times more potent than imatinib against Abl kinases.

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

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