Orphagen Pharmaceuticals, Inc.
October 17, 2023
Company Description: Orphagen is a pioneer in discovery and development of small molecule drugs that modulate orphan or unexplored members of the nuclear receptor family, one of the most successful drug target families known. Orphagen’s most advanced asset is OR-449, an orally-bioavailable, first-in-class, IND-ready small molecule for treatment of an aggressive and largely untreatable metastatic cancer of the adrenal gland, adrenocortical cancer (ACC). U.S. and EU prevalence is ~1,500 patients with market potential of $0.5 B/year. We seek funding for a Phase 1 trial. Given severe unmet need, accelerated approval by FDA after a single-arm Ph2 trial appears feasible. Long-term, OR-449 will become the backbone of all combination therapy in ACC given the central role of its target, SF-1, a transcription factor required for adrenal growth and development. Expansion indications with strong genomic support include a subset of squamous head & neck tumors & benign adrenal tumors with adverse endocrine impact.
CEO/Top Company Official
Lead Product in Development
OR-449, a Phase 1 ready, first-in-class small molecule for a rare and untreatable cancer, adrenocortical cancer.
Development Phase of Primary Product
Number Of Unlicensed Products
Two unlicensed products OR-449 -- Phase 1-ready -- for rare cancer and expansion indications OR-812 -- in early development-- for inflammatory bowel disease