ArQule, Inc. announced preclinical data demonstrating the potential for miransertib (ARQ 092), it’s pan-AKT inhibitor, to treat PIK3CA driven vascular malformations (VMs) in a poster at the 2019 American Society of Human Genetics Annual Meeting. Studies conducted in an experimental mouse model show that miransertib prevents the formation of PIK3CA-driven VMs and shows high efficacy in regressing vascular growth in already developed VMs. The poster presented October 21, 2019 at ASHG details findings from studies of miransertib in an experimental mouse model that develops PIK3CA-driven VMs resembling those found in humans. Data show that miransertib prevents the formation of PIK3CA-driven VMs by suppressing vascular growth and endothelial cell proliferation. Treatment with miransertib was also found to lead to a regression in growth of already developed VMs, as measured by a significant reduction in retinal vascularity and endothelial cell number and proliferation. VMs are painful, disfiguring lesions that can be present in any tissue and can lead to bleeding and obstruction of organs. The condition has an overall incidence of 1 in 5,000, and no approved pharmacological options currently exist. Most VMs are caused by mutations in the PIK3CA or TEK gene, both of which lead to increased PIK3CA signaling through the PI3K/AKT pathway. Miransertib works by inhibiting AKT, thereby reducing the abnormal cell growth which results from increased PIK3CA signaling.