We have established a broad pipeline of drugs that target different points in Rho/ROCK signaling and related pathways. Rho kinase (ROCK) is hyperactive in many pathological processes where cell-cell interaction or changes in cell architecture are involved. Stroke and cerebral cavernous malformation are both neurovascular disorders where intercellular adhesive complexes between endothelial cells are aberrantly regulated by ROCK2, the form of ROCK highly expressed in the central nervous system. We focus on cerebral cavernous malformation, also called angioma, because of the unmet need, clear genetic proof of concept, wealth of literature that show ROCK hyperactivation is the underlying target pathology of the disease, and clear path to clinical proof of concept.

We also continue to develop drugs to treat spinal cord injury (SCI) because of unmet need, our deep knowledge of SCI,  and our success in drug development to treat neurotrauma. BA-434 is a drug that addresses the intrinsic barriers to axon regeneration. It is a biologic drug  that represses the expression of PTEN. Expression of PTEN is known to block axon regeneration in the adult central nervous system.

We validate regenerative mechanisms of new drugs in animal models of optic nerve injury because it is a simple white matter tract in the central nervous system. These studies are not only relevant to SCI, but also to glaucoma where high intraocular pressure leads to optic nerve injury. ROCK inhibitors are a new, effective target for glaucoma. BioAxone’s topical ROCK inhibitor is differentiated from other drugs in development because it does not cause the common side effects other ROCK inhibitors have shown.