4 August 2017 - - The National Institutes of Health, Small Business Innovation Research, and National Institute of Neurological Disorders and Stroke, has awarded Cambridge, Massachusetts-based neurotrauma and neurovascular disorders specialist BioAxone BioSciences, Inc. a USD 1.5m grant, the third year installment to continued research into BioAxone's first-in-class selective Rho Kinase 2 (ROCK2) inhibitor, BA-1049, a drug in development to treat cerebral cavernous malformations (CCM), the company said.
The award was based on research and development milestones achieved by BioAxone.
CCM is a serious genetic disease where patients have a lifetime risk of brain hemorrhage from vascular malformations in the brain in which endothelial cells form single or multiple cystic brain lesions that leak and may cause seizure, hemorrhagic stroke, and neurological deficits.
Inherited cases of CCM are caused by mutation in one of the 3 CCM genes (CCM1, CCM2 and CCM3) and CCM loss of function results in the overactivation of ROCK2, the target of BA-1049 development.
In preclinical experiments BA-1049, a first-in-class oral inhibitor of Rho kinase 2 (ROCK2), has shown promise to repair the leaky endothelial cell barrier and potentially to reverse progression of disease. BioAxone is progressing key non-clinical IND enabling studies with this small molecule.
BioAxone BioSciences is developing innovative drugs to restore neurological function for patients with spinal cord injuries, vascular malformations in the central nervous system, and glaucoma.