Orphagen is pioneering small molecule therapies to tackle cancer and other serious diseases. Our drug candidates block novel members of the nuclear receptor superfamily, a target class that has produced leading treatments for inflammation, oncology, and metabolic disease. Our first-in-class nuclear receptor programs, internally conceived and developed by us, are now on the path to preclinical and clinical development.

Our most advanced programs are directed in two areas:

• a robust oncology program including both tumor-specific and precision therapy opportunities that encompass not only adrenal cancer but also head & neck and other squamous cancers.
• a differentiated oral small molecule therapy for ulcerative colitis and Crohn’s disease

We talk actively with the clinical, scientific, investment, and patient communities to enable new kinds of therapy. Reach out to us.

On a Mission to Imagine the Unimagined.