Technology

The receptors under investigation at Orphagen Pharmaceuticals have been implicated in autoimmune disease, sleep-wake disorders, metabolic disease, and mood disorders. High throughput screening has yielded modulators with properties suitable for medicinal chemistry exploration and lead molecules derived from these are under intensive investigation.


Orphagen's technology position:
Orphagen scientists have demonstrated that certain unexplored orphan nuclear receptors are viable targets for drug discovery. The Company is developing a strong intellectual property position around these targets.

Therapeutic Areas

Autoimmune disease. Orphagen has pioneered the discovery of selective inhibitors of Th-17 cells, a novel class of T lymphocytes implicated in psoriasis, Crohn’s Disease, rheumatoid arthritis, and multiple sclerosis.

Circadian Regulation. Orphagen is exploring targets that control the body’s endogenous day-night cycle (circadian rhythm) in the brain. These targets have major potential for the treatment of sleep-wake and mood disorders.

Metabolic Disease. Diabetes and hyperlipidemia are major causes of morbidity and mortality in the U.S. Orphagen is investigating targets that regulate glucose and lipid metabolism with potential as novel therapeutics in this area.

About nuclear receptors
Nuclear receptors, such as the estrogen and thyroid hormone receptors, are highly successful drug targets, generating $12 billion in annual U.S. sales. They are transcription factors that are directly regulated by small molecules, and they have very diverse therapeutic uses. The unexplored orphan nuclear receptors have the potential to generate novel therapeutic agents and open new pharmaceutical markets.