For example, Cortendo is a Swedish biotech with a pioneering approach in the field of orphan drugs and metabolic diseases, and specifically, cortisol inhibition. Their lead drug candidate for endogenous hypercortisolism (Cushing’s Syndrome), NormoCort (COR-003), the 2S, 4R-enantiomer of ketoconazole, is now in Phase III development. Cushing’s Syndrome is an orphan disease that affects women three times as often as men and is most prevalent in those aged 20–50. Endogenous Cushing´s Syndrome (CS) is a rare but serious and potentially lethal endocrine disease caused by excessive cortisol exposure to human organs, and is generally caused by small tumors in the brain (pituitary gland) or adrenal gland. Treatment options for CS are limited to surgery, radiation therapy and drug treatment. Drug treatment is used to suppress excessive cortisol production and/or ameliorate its clinical manifestations prior to surgery or in patients awaiting the effects of radiation therapy as well as in patients where surgery either has failed, is contra-indicated or when a tumor cannot be found. The company expects Normocort to be a more potent inhibitor of key enzymes in the cortisol synthesis pathway, with improved potency and safety benefits over racemic ketoconazole.
Another interesting company is Amarantus Bioscience Holdings, Inc. based in San Francisco, which is developing treatments and diagnostics for diseases associated with neurodegeneration and apoptosis (programmed cell death), and specifically, Alzheimer’s Disease. The Lymphocyte Proliferation, or LymPro Test®, is the company’s flagship blood test for Alzheimer’s disease. LymPro was designed with the purpose of diagnosing Alzheimer’s in its mild to moderate stage. This patient-specific identification has the potential to become an invaluable tool for Alzheimer’s disease clinical trials, where there has been a well-documented history of patient recruitment errors related to inaccurate diagnosis of Alzheimer’s. You can read the white paper here.
There are also some exciting companies is the area of combination cancer immunotherapy. These companies are developing therapies that use the body’s own immune system to combat cancer. One example is NuvOx Pharma, a University of Arizona spinoff biotechnology company based in Tucson, Arizona that is developing an innovative platform of dodecafluoropentane (DDFP)-based oxygen therapeutics to treat a host of human conditions. DDFP has already demonstrated therapeutic feasibility in a radiation-resistant cancer called glioblastoma multiforme, a rare but particularly deadly form of brain cancer.
Or, consider San Diego based Polynoma, an immuno-oncology company developing a novel polyvalent antigen therapy for the treatment of melanoma. Polynoma has two lead candidates in Phase II, and commenced its Phase III MAVIS trial (Melanoma Antigen Vaccine Immunotherapy Study) with seviprotimut-L (formerly POL-103A) in 2012. MAVIS is an adaptive Phase III trial being conducted under a Special Protocol Assessment (SPA). The therapy is designed for melanoma patients for whom there are currently no other alternatives.
This is just a small taste of companies that will be presenting. Registration information for Biotech Showcase is available online.