Orphagen’s first-in-class pipeline has been successful in finding the first drug-like small molecules to several orphan nuclear receptors and in evaluating efficacy in animal models of disease. An early success led to Orphagen’s partnering agreement 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 RORγ. JTE-451, a product of the JT Pharma partnership, entered Phase 1 trials in 2016.
Orphagen has two major internal programs. One is to develop OR-682 for immuno-oncology, with a focus on ovarian, pancreatic and liver cancer. The second is to develop antagonists to the orphan nuclear receptor steroidogenic factor-1 (SF-1) for the orphan indications adrenocortical cancer (ACC) and Cushing’s syndrome.
Orphagen also provides assay and screening services for many members of the nuclear receptor family. For more information, see Assays.