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CCM (also known as cavernous angioma) is a genetic disorder caused by loss of function in one of three CCM genes, resulting in over-activation of Rho kinase in cerebral endothelial cells. The CCM mutation leads to benign endothelial cell tumors that cause headache, seizures and neurological deficits such as paralysis. The tumors may bleed, requiring emergency medical intervention. Cavernous angiomas are estimated to occur in approximately 0.2% of the general population. This includes the incidence of sporadic forms, which may be up to 70% of diagnosed cases. Early-stage
poradic lesions can be asymptomatic and remain undetected until they grow or bleed, and multiple lesions occur in people with the familial disease. Individuals often experience their first symptoms in their 20s or 30s. There is no current treatment to stop progression except surgical removal.Dr. Lisa McKerracher, CEO of BioAxone, stated, "We are delighted that this grant has allowed us to form a strong consortium of industry and academic partners to develop the first effective drug to treat this disorder, which is devastating for patients and their families. The Angioma Alliance was instrumental in helping us form the consortium."The project funds a consortium of collaborators including Dr. Issam Awad (University of Chicago), Dr. Douglas Marchuk (Duke University), and Dr. Rebecca Stockton (LA BioMed and the David Geffen School of Medicine at UCLA). They are all experts in diverse aspects of CCM, ranging from the genetic basis of disease to neurosurgical treatment. The academic and medical collaborators will complete proof of concept studies to determine efficacy of BA-1049 with human vascular tissue and transgenic mice expressing defective copies of the CCM gene. BioAxone will coordinate the collaboration, complete dose-ranging studies in mice, and carry out good-laboratory practice (GLP) safety studies to support an IND application to the FDA.

Dr. Amy Akers, Chief Scientific Officer of Angioma Alliance, remarked, "Angioma Alliance and the CCM patient community is committed to facilitating novel scientific collaborations and driving research for a cure. We are thrilled by