Orphagen Pharmaceuticals

Orphagen discovers and develops small molecules to drug targets that have significant unrealized potential in areas of high clinical need. We focus on the unexplored or “orphan” members of the nuclear receptor family. Nuclear receptors have been a rich source of successful drugs. Orphagen has identified drug-like small molecules to several orphan nuclear receptors and has described efficacy in animal models of disease. An early success led to a partnership with Japan Tobacco (JT) Pharma to discover and develop oral drugs to treat psoriasis and other autoimmune diseases. This partnership was the first in the industry for the nuclear receptor ROR-gamma. JTE-451, a product of the JT Pharma partnership, entered Phase 2 trials this year (in 2019). Orphagen has two major internal programs. One is to develop antagonists to the orphan nuclear receptor steroidogenic factor-1 (SF-1) for adrenocortical cancer (ACC) and other endocrine conditions. The second focuses on inflammatory disease.