NOVEL (UNDISCLOSED) Targets for inflammatory BOWEL disease and precision cancer


Orphagen’s strength is drug discovery at novel, unexplored nuclear receptors. Our discovery objective has been to validate new therapies at these targets in animal models of disease with our potent receptor ligands.

Two programs are in the discovery phase at two undisclosed targets. In each case, we have potent and unique ligands—new chemical entities—to use as tool for potential development compounds.

First, we are exploring a target for inflammatory disease. The most promising findings relate to inflammatory bowel disease. Our lead compound, OR-682, has excellent bioavailability, exposure and target engagement in mice. Our lead compounds and close analogues have properties consistent with a development candidate.

In the second program, we are exploring an orphan receptor target that is ectopically and sporadically turned on in several cancers, including sarcoma, small cell lung carcinoma, and castration-resistant prostate cancer. The target has the potential to drive precision cancer therapy. We have identified patient-derived tumor cells with a high level of target expression that respond to Orphagen ligands in cell culture and in vivo.



immuno cancer