Orphagen Pharmaceuticals, a privately-held emerging pharmaceutical company, announced today that the National Institutes of Health has awarded the Company $220,839 in funding through the Small Business Innovative Research (SBIR) program. The Phase I grant is designed for exploratory work on a new class of molecules known as antagonists of steroidogenic factor-1 (SF-1).

“SF-1 is highly expressed in the lesional tissue of endometriosis. It appears to regulate steroid synthesis and perhaps other pathogenic events in endometriotic tissue. Proprietary SF-1 antagonists identified at Orphagen will be used to characterize potential therapeutic action in endometriotic biopsies,” said Paul Crowe, Director of Biology and Principal Investigator of the grant.

“The NIH SBIR program has been a foundation for our efforts to explore new therapeutic areas. Federal support has made a pivotal difference in the high risk-high reward projects that we typically engage in,” said Scott Thacher, CEO of Orphagen.

Orphagen has made important discoveries in identifying potential drug ligands to previously unexplored orphan receptors, putative drug targets identified as part of the human genome project. Orphagen has captured a first-mover position for three of these targets, including steroidogenic factor-1, which is under investigation for Cushing’s syndrome, adrenocortical cancer, castrationresistant prostate cancer and endometriosis at Orphagen.

Many global pharmaceutical companies view discovery research as too risky, and are increasingly relying on external sources of innovation. Orphagen holds a strong intellectual property position because of its first-to-ligand approach in several programs, and plans to pursue future drug development through partners with complementary capabilities in pre-clinical and clinical research.

About Orphagen: Orphagen discovers drug candidates for potential drug targets 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 partners and/or outside sources of funding. These targets come from the nuclear receptor family of drug targets. On a per target basis, the nuclear receptors are one of the most successful target classes known to the pharmaceutical industry. Targets of interest to Orphagen encompass several of the so-called orphan nuclear receptors—potential therapeutic receptors that have yet to be exploited by the pharmaceutical industry.

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