CCM
Our small molecule drug candidates, Rho kinase (ROCK) inhibitors, inhibit a downstream target of Rho. They have the potential to repair a broken blood brain barrier. They have been out-licensed to Neurelis for continued development.
ROCK is hyper-activated in endothelial cells in neurovascular disorders, including the disease of cerebral cavernous malformation (CCM), also called cavernous angioma, a serious genetic disease where endothelial lesions in the brain become leaky and bleed. There is no treatment for CCM except invasive surgery. ROCK is a target for treatment because the genetic mutation leading to angioma causes ROCK hyper-activation in brain capillary endothelial cells. Treatments effective for CCM also have utility in treating the blood brain defects in other neurovascular disorders, including stroke.
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