Program Indication Lead Discovery Lead Optimization IND Enabling Studies Clinical StudiesCommercial Rights
Internal Programs
Antagonists of Steroidogenic Factor 1 (SF-1) Adrenocortical Cancer (ACC)
Orphagen: Worldwide
Undisclosed TargetInflammatory Bowel Disease
Orphagen: Worldwide
Undisclosed Target Precision Oncology
Orphagen: Worldwide
Partnered Programs
ROR-gamma Antagonists

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. Our corporate focus is to generate pre-clinical and clinical validation of these targets.

Orphagen has three major internal programs.  Our lead program is for antagonists to the orphan nuclear receptor steroidogenic factor-1 (SF-1). The lead antagonist, OR-449, is highly active in suppression of tumor growth. Our goal is to carry out initiate Phase I clinical trials in 2022 for the orphan indication adrenocortical cancer (ACC).

Follow-on programs are based on unexplored targets from the nuclear receptor family for which Orphagen has potent and proprietary ligands. The first of these two undisclosed target programs is focused on inflammatory bowel 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.

Early in the Company’s development, Orphagen signed a partnering agreement with the Pharmaceutical Division of Japan Tobacco (JT) to discover and develop oral drugs to treat psoriasis and other autoimmune diseases. The partnership was the first in the industry for the orphan nuclear receptor RORγ, and it was based on Orphagen’s suite of assays, animal models, and promising antagonist scaffolds. JTE-451, a clinical candidate that emerged from the JT partnership, is currently in a Phase 2 clinical trial for moderate to severe plaque psoriasis (IMPACT-PS).