Program Indication Lead Discovery Lead Optimization IND Enabling Studies Clinical StudiesCommercial Rights
Internal Programs
Antagonists of Steroidogenic Factor 1 (SF-1)
Adrenocortical Cancer (ACC) & Cushing's Syndrome
Orphagen: Worldwide
Undisclosed Target
Inflammatory Diseases
Orphagen: Worldwide
Undisclosed Target
Precision Oncology
Orphagen: Worldwide
Partnered Programs
ROR-gamma Antagonist
Psoriasis and other Autoimmune Diseases
Pharmaceutical Division of Japan Tobacco: Worldwide

Orphagen’s proprietary screening technology has led to successful discovery of the first drug-like small molecules to several orphan nuclear receptors. In addition, Orphagen performs critical target validation in animal and cellular models of disease based on its novel, potent and specific small molecule ligands.

An early success led to Orphagen’s partnering agreement with the Pharmaceutical Division of Japan Tobacco (JT) 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 and was based on Orphagen’s pioneering identification and pharmacological evaluation of RORγ antagonists. JTE-451, a clinical candidate that emerged from the JT partnership, entered Phase 1 clinical trials in 2016.

Orphagen has three major internal programs.  Our lead program 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.  Two follow-on programs are based on novel, unexplored targets from the nuclear receptor family for which Orphagen has potent and proprietary ligands. The first of these two undisclosed target programs is primarily focused on inflammatory disease. The second is based on an orphan receptor that is ectopically expressed in several cancers and has the potential to drive precision cancer therapy.

Orphagen also collaborates with select pharmaceutical and biotechnology companies to offer proprietary nuclear receptor screening services for the discovery of new medicines. For more information, see Assays.