FIRST-IN-CLASS PIPELINE

Orphagen has discovered the first drug-like small molecules to several orphan nuclear receptors. In addition, Orphagen has identified preclinical animal and cellular models appropriate for target validation for these novel classes of orally bioavailable small molecules.

Our lead program is for antagonists to the orphan nuclear receptor steroidogenic factor-1 (SF-1). The clinical candidate, OR-449, is highly active in suppression of certain tumor models, confirming its therapeutic potential. Our goal is to initiate Phase I clinical trials in late 2023 for the orphan indication adrenocortical cancer (ACC).

Our second pipeline program is focused on inflammatory bowel disease (IBD) and other forms of gut inflammation, including graft versus host disease (GVHD).

Orphagen has insight into and technology for several other nuclear receptors at the ligand discovery and pre-clinical validation stage. The Company’s goal is to create one or more additional development programs from these targets.

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 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, co-crystal structures, and promising antagonist scaffolds. JTE-451, a clinical candidate that emerged from the JT partnership reached a Phase 2 clinical trial for oral therapy of moderate to severe plaque psoriasis (IMPACT-PS). Currently, JTE-451 is under development as a topical therapy for inflammatory disease.