There are significant unmet medical needs in many neurological diseases, including pain, multiple sclerosis and Alzheimer's Disease. Plexxikon is pursuing a number of different target classes with its unique platform approach to develop novel agents to treat these debilitating diseases.

Pain

A major market opportunity exists to treat patients with moderately severe and severe pain with an oral, non-opioid agent with a rapid onset of action. This market is expected to increase significantly as the overall population ages.

PLX5568, a novel, oral non-opioid kinase inhibitor, has demonstrated efficacy in a number of preclinical models of pain. These studies have shown opiate-like efficacy in treating acute nociceptive, acute inflammatory and chronic pain. Plexxikon's drug candidate also features attractive pharmaceutical properties, with favorable exposure and half-life. Currently, PLX5568 is being tested in a Phase 1 trial. Further clinical development will initially focus on the use of PLX5568 for the treatment of polycystic kidney disease (PKD).

Multiple Sclerosis

Plexxikon has two programs in development for multiple sclerosis (MS).

PLX3397 is a novel, oral and highly selective kinase inhibitor that down-modulates macrophages, osteoclasts and mast cells. These are all key drivers of a number of inflammatory diseases, including multiple sclerosis and rheumatoid arthritis, as well as certain cancers. Potentially key for MS and other neuro-degenerative diseases, PLX3397 has been shown to eliminate microglia in the CNS in preclinical models. The compound is initially being tested in a Phase 1 clinical trial for safety, tolerability and pharmacokinetics in cancer patients.

Plexxikon’s second MS program, an oral, novel PPAR (peroxisome proliferator-activated receptor) pan-agonist compound, has been shown in preclinical studies to have an anti-inflammatory effect, as well as the ability to promote myelin synthesis. This candidate represents a first-in-class DMARD for the treatment of this neurodegenerative disease.