Orphagen is structured around discovering drug candidates for potential drug targets for which small molecule ligandspotential drug-like molecules--have yet to be identified. The firm's goal is to identify, characterize, and position a new class of drug so that pre-clinical and clinical development can be initiated with partners and/or outside sources of funding. The targets come from the nuclear receptor family of drug targets. On a per target basis, nuclear receptors are one of the most successful target classes known to the pharmaceutical industry. Receptors that have yet to be exploited by the pharmaceutical industry are called orphan nuclear receptors, and form Orphagens central focus. Orphagen concentrates on the early stages of small molecule drug discovery: screening, hit-to-lead, lead optimization and preclinical development. The firm reduces the risks of downstream drug development by characterizing pharmacologically active molecules in cellular and animal models of disease. By pursuing this strategy, Orphagen signed the first strategic partnership for a novel target for treatment of autoimmune disease, ROR-gamma, with Japan Tobacco. Merck, BMS, Pfizer, Amgen and J&J later signed partnerships with other small biotech companies at the same target. Orphagen has identified novel approaches to several orphan indications, including retinitis pigmentosa, Cushings syndrome and adrenocortical cancer. It is also pursuing preclinical work on a chronobiotic for treatment of mood disorders and Alzheimers disease. Other projects at an earlier stage involve targets for treatment of sickle cell anemia and glioblastoma.
