Orphagen Pharmaceuticals, a privately-held emerging pharmaceutical company, presented a late-breaking poster describing the effect of antagonists to steroidogenic factor-1 (SF-1) on adrenal synthesis of glucocorticoids at the Endocrine Society’s 95th Annual Meeting in San Francisco, CA. Orphagen’s SF-1 antagonists suppress steroid synthesis in adrenal cells, suggesting that this compound class could treat Cushing’s syndrome, where pathological hypersecretion of the adrenal glucocorticoid, cortisol, occurs.

“SF-1 is highly expressed in the adrenal gland and is a major regulator of steroidogenesis. Proprietary SF-1 antagonists identified at Orphagen dose-dependently suppress steroid secretion in primary adrenal cell cultures,” said Paul Crowe, Director of Biology at Orphagen. “We think more advanced compounds could be designed to inhibit adrenal steroidogenesis in patients.”

Orphagen started its SF-1 antagonist program with the support of two SBIR grants from the National Cancer Institute (NCI). Antagonists to SF-1 are under investigation at Orphagen for Cushing’s syndrome as well as adrenocortical cancer (ACC), castration-resistant prostate cancer and endometriosis. Based on its clinical and basic relevance, the Orphagen poster was one of a very small number of late-breaking abstracts accepted for the 2013 ENDO meeting.

“This poster provides the first detailed description of the pharmacology of SF-1 antagonists in adrenal cells and was the result of intensive compound synthesis and characterization or Orphagen,” said Scott Thacher, CEO and founder of Orphagen. “We believe we are the leaders in this area and we are actively seeking partners to accelerate the pace of this first-in-class program.” Orphagen holds a strong intellectual property position because of its first-to-ligand approach in several programs, and aims to pursue future drug development through partners with complementary capabilities in preclinical and clinical research.

About Orphagen: Orphagen discovers drug candidates for potential drug targets from the nuclear receptor family for which small molecule ligands–potential drug-like molecules–have yet to be identified. Its goal is to identify, characterize, and position a new class of drug so that pre-clinical and clinical development can be initiated with commercial partners. Orphagen successfully partnered its first program for ROR-gamma with Japan Tobacco, several years ahead of competitors. Additional first-in-class drug discovery programs, such as for SF-1, have been developed with partnership revenue and other non-dilutive funding sources, including grants.

For more information, contact: Scott Thacher (858) 481-6191