Starting with a therapeutic-area agnostic approach, we are pursuing proprietary lead molecules with potential in oncology, CNS, autoimmune and retinal diseases. With relentless creativity and resolve, we are unafraid of tackling the unknown and passionate about creating a first-in-class pipeline that will deliver for patients with significant unmet needs.

Partnerships are a key element of our continued success in advancing our programs. Orphagen successfully partnered its first program for ROR-gamma antagonists with a mid-size pharmaceutical company ahead of all competitors in the field. Funding from its partnerships and other non-dilutive sources, including federal grants, has allowed Orphagen to advance its proprietary first-in-class drug discovery programs, including OR-449 for cancers such as adrenocortical cancer, which is IND-ready for clinical trials.

  • OR-449: Orphagen’s oncology lead candidate OR-449 is a potentially major step forward in treatment for adrenocortical carcinoma (ACC), an aggressive cancer of the adrenal gland. A silent killer with no effective treatment nor any significant improvement in medical therapy in over 50 years, ACC leaves patients with limited options and little hope for long-term survival.
  • Now at an exciting turning point for Orphagen, IND-enabling studies for OR-449 are nearly complete and we are poised for IND filing and the initiation of Phase 1 clinical trials in adult ACC patients.
  • The U.S. Food and Drug Administration (FDA) has granted OR-449 a Rare Pediatric Disease Designation (RPDD), meant to incentivize rapid development of OR-449 for the treatment of the pediatric form of adrenocortical ACC.
  • In addition to OR-449, Orphagen has built a diverse pipeline with several first-in-class drug discovery programs that modulate the transcriptional activity of other unexploited orphan nuclear receptors.
  • Our OR-812 program is a retinoic acid receptor alpha antagonist currently in lead optimization for inflammatory bowel disease (IBD). Current IBD therapy is dominated by injectable drugs. The small molecule OR-812 will offer a novel form of oral therapy with a differentiated mechanism of action.
  • RORβ antagonists. Our discovery program for ROR-beta antagonists has pursued endpoints within the arenas of CNS disease, retinal degeneration, and oncology. We seek new genomic insights for ROR-beta function, and our unique small molecule tools equip us to engage in collaborations for testing pharmacological and therapeutic hypotheses based on genomic findings.