BTK Inhibitor Shows Potential in Early Phase Trial for MS

This article originally appeared on The American Journal of Managed Care.

A phase 1 trial evaluating the safety and tolerability of PRN2246, a Bruton's tyrosine kinase (BTK) inhibitor, found the drug having no serious medication-related adverse events in healthy volunteers while also being able to reach the brain.

Multiple sclerosis (MS) is a neurodegenerative disease in which a person’s immune system functions abnormally, causing inflammation and damage to myelin, which in turn prevents normal signaling by nerve cells and can result in motor disorders and disability.

With a lack of treatments that reverse or prevent disease progression/relapses, MS continues to be a debilitating disease in many patients around the world. Several drug companies are invested in finding alternative therapies that patients with MS and their physicians can use. One major issue with these drugs is that they must be formulated to be able to cross the blood-brain barrier (BBB) to be effective, which makes drug development a challenge.

The purpose of this phase 1 trial was to determine if the BTK inhibitor PRN2246, developed by Principia Biopharma in collaboration with Sanofi, is safe profile in healthy volunteers and can penetrate the BBB. BTK inhibitors work by inhibiting the B-cell signaling pathway and limiting B-cell maturation. If PRN2246 can successfully penetrate through the BBB, the hope is that the drug will be able to modulate the activity of immune B-cells in the central nervous system (CNS) and delay further progression or relapses of the disease.

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