Orphagen Pharmaceuticals, Inc.
Orphagen is a pioneering biopharmaceutical company focused on developing novel therapies for cancer and serious diseases, including autoimmune and inflammatory conditions. Their mission is to create first-in-class oral drugs targeting nuclear receptors, with programs in oncology and autoimmune diseases, supported by strategic partnerships and federal grants.
Industries
Nr. of Employees
small (1-50)
Orphagen Pharmaceuticals, Inc.
San Diego, California, United States, North America
Products
SF‑1 (NR5A1) antagonist — clinical development candidate for adrenocortical carcinoma and SF‑1‑high tumor subsets
Orally‑bioavailable first‑in‑class small‑molecule antagonist of the steroidogenic factor‑1 (SF‑1 / NR5A1) nuclear receptor, developed as a targeted therapy for adrenocortical carcinoma and precision therapy for tumor subsets with high SF‑1 expression.
RARα antagonist — development candidate for inflammatory bowel disease
Potent and selective antagonist of retinoic acid receptor‑alpha (RARα) designed to block induction of gut‑homing markers on T cells, reduce inflammatory T cell migration, and ameliorate intestinal inflammation in preclinical colitis models.
RORβ antagonist small-molecule toolset — preclinical pharmacology programs
Small‑molecule antagonists and agonists for the retinoid‑related orphan receptor beta (RORβ) used to study pharmacology and explore therapeutic potential in indications such as small cell lung carcinoma and select leukemias.
SF‑1 (NR5A1) antagonist — clinical development candidate for adrenocortical carcinoma and SF‑1‑high tumor subsets
Orally‑bioavailable first‑in‑class small‑molecule antagonist of the steroidogenic factor‑1 (SF‑1 / NR5A1) nuclear receptor, developed as a targeted therapy for adrenocortical carcinoma and precision therapy for tumor subsets with high SF‑1 expression.
RARα antagonist — development candidate for inflammatory bowel disease
Potent and selective antagonist of retinoic acid receptor‑alpha (RARα) designed to block induction of gut‑homing markers on T cells, reduce inflammatory T cell migration, and ameliorate intestinal inflammation in preclinical colitis models.
RORβ antagonist small-molecule toolset — preclinical pharmacology programs
Small‑molecule antagonists and agonists for the retinoid‑related orphan receptor beta (RORβ) used to study pharmacology and explore therapeutic potential in indications such as small cell lung carcinoma and select leukemias.
Services
Collaborative research and licensing
Formation and execution of partnerships to co-develop or license discovery-stage small-molecule programs and to accelerate development through partner resources.
Preclinical development and IND-enabling support
Preclinical pharmacology, ADME‑PK, nonclinical safety studies, biomarker identification and IND planning to enable first‑in‑human studies.
Collaborative research and licensing
Formation and execution of partnerships to co-develop or license discovery-stage small-molecule programs and to accelerate development through partner resources.
Preclinical development and IND-enabling support
Preclinical pharmacology, ADME‑PK, nonclinical safety studies, biomarker identification and IND planning to enable first‑in‑human studies.
Expertise Areas
- Nuclear receptor drug discovery
- Oncology drug development
- Inflammatory disease therapeutics (IBD)
- Medicinal chemistry and lead optimization
Key Technologies
- High-throughput screening (HTS)
- Orthogonal ligand-binding assays
- Medicinal chemistry
- In vitro cell-based assays