Orphagen Pharmaceuticals, a privately-held pharmaceutical company, announced today that the National Cancer Institute (NCI) has indicated its intention to award the company $2.0 million under the Small Business Innovation Research (SBIR) program to support preclinical development of OR-449, a small molecule drug candidate designed and synthesized by Orphagen, as a first-in-class therapy for adrenocortical carcinoma (ACC), a rare cancer of the adrenal gland with very limited treatment options and a poor prognosis.
OR-449 is a potent and selective inhibitor of steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland. Multiple findings indicate that SF-1 has a crucial role in the pathogenesis of ACC.
“Orphagen has a strong platform for nuclear receptor discovery and our technology has allowed us to identify promising antagonists to a previously unexplored nuclear receptor, known as steroidogenic factor-1, or SF-1,” said Scott Thacher, CEO of Orphagen and Principal Investigator of this grant. “The discovery and characterization of OR-449, which led to this prestigious award, was the product of an immensely talented team, with expertise in compound screening, medicinal chemistry, the implementation of novel tumor models, and small molecule drug development. The creation of this new drug candidate for ACC patients would not have been possible without the dedicated efforts of Orphagen’s multidisciplinary team.”
Notice of first-year funding for this proposal can be found on the NIH web site at: RePORT ⟩ RePORTER (nih.gov).
About Orphagen: Orphagen discovers drug candidates for potential drug targets from the nuclear receptor family for which small molecule ligands, or 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 antagonists with JT Pharma ahead of all competitors in the field. Funding from this partnership and other non-dilutive sources, including federal grants, has allowed Orphagen to advance additional first-in-class drug discovery programs.