Drug Hunters. Exploring the Unexplored.
Delivering Novel Therapies for Cancer
and Other Serious Diseases
Orphagen is pioneering small molecule therapies to tackle cancer and other serious diseases. Our drug candidates block unexplored members of the nuclear receptor superfamily, a target class that has produced major treatments for inflammation, oncology, and metabolic disease. Our groundbreaking nuclear receptor programs, internally conceived and developed by us, are now on the path to preclinical and clinical development.
Our most advanced programs are:
- A first-in-class clinical candidate, OR-449, for the treatment of adrenocortical cancer (ACC), a rare cancer of the adrenal gland, through antagonism of the orphan nuclear receptor SF-1 (NR5A1). SF-1 appears to drive growth and survival in ACC and potentially in subsets of other cancers where OR-449 may also be developed.
- A differentiated oral small molecule therapy, OR-812, for ulcerative colitis, Crohn’s disease, and other conditions of gut inflammation.
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.
Orphagen CEO and founder Scott Thacher discusses Orphagen’s clinical candidate, OR-449, for treatment for adrenocortical cancer with Gary Hammer, M.D., Ph.D., Millie Schembechler Professor of Adrenal Cancer at the University of Michigan
Orphagen Team
Our scientists and management team are committed to the best we can bring to bear in pursuit of new therapies. We are drug hunters.
Pipeline
Our mission is translational drug discovery and development with novel targets leading to first in class therapeutics.