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.
9276 Sorrento Valley Rd, Suite 500, San Diego, CA 92121
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
Formation and execution of partnerships to co-develop or license discovery-stage small-molecule programs and to accelerate development through partner resources.
Preclinical pharmacology, ADME‑PK, nonclinical safety studies, biomarker identification and IND planning to enable first‑in‑human studies.
Formation and execution of partnerships to co-develop or license discovery-stage small-molecule programs and to accelerate development through partner resources.
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
News & Updates
Funding to support preclinical development of Orphagen’s lead IBD candidate, OR-812, a first-in-class, small molecule inhibitor of the retinoic acid receptor alpha (RARα).
The Cancer Prevention & Research Institute of Texas (CPRIT) announced a $10.2 million grant to support IND filing and a Phase 1 clinical trial for OR-449, a novel therapy for adrenocortical carcinoma (ACC).
The FDA has granted OR-449 a Rare Pediatric Disease Designation for the treatment of pediatric adrenocortical carcinoma.
Data from preclinical studies on OR-449, a SF-1 antagonist, including its effects on steroid secretion in ACC tumors.
Funding to support preclinical development of Orphagen’s lead IBD candidate, OR-812, a first-in-class, small molecule inhibitor of the retinoic acid receptor alpha (RARα).
The Cancer Prevention & Research Institute of Texas (CPRIT) announced a $10.2 million grant to support IND filing and a Phase 1 clinical trial for OR-449, a novel therapy for adrenocortical carcinoma (ACC).
The FDA has granted OR-449 a Rare Pediatric Disease Designation for the treatment of pediatric adrenocortical carcinoma.
Data from preclinical studies on OR-449, a SF-1 antagonist, including its effects on steroid secretion in ACC tumors.