Founded as a biomarker-diagnostics company in 2005, the company is developing new chemotherapy drugs by targeting molecular abnormalities of human cancers. Over the past year, based on the discovery of a new biomarker for brain cancers by collaborators at the National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), the company is evaluating new drugs for the treatment of glioblastoma multiforme (GBM), the most common and most aggressive type of primary brain cancer in adults and, to a much lesser extent, in children. NINDS (www.ninds.nih.gov) is a component of the National Institutes of Health (NIH), and is the nation’s primary supporter of biomedical research on the brain and nervous system.

Over the next year, as part of its collaboration with NINDS, the company plans to complete studies of the effectiveness of lead compounds from each of two different classes of drugs developed by Lixte that are active individually and, more so in combination, against GBM in the test tube. Initial studies showed that these compounds affect the intended biomarker target and have anti-tumor activity in a mouse model of cancer. The next steps are characterization of anti-tumor activity of these compounds in animal models of human GBM. Subsequently, the toxicity profiles, pharmacologic behavior, and metabolism of both classes of agents will be determined in pre-clinical toxicology studies.

Should the results of the foregoing tests and studies prove successful, Lixte hopes to satisfy FDA requirements for approval of, at least, one lead compound for evaluation in Phase I trials in early 2009. Lixte will also evaluate the anti-cancer activity of its new compounds in model systems of two life-threatening cancers of children, neuroblastoma and medulloblastoma, and against several common cancers of adults in addition to GBM.