Orphagen Pharmaceuticals, a privately-held emerging pharmaceutical company, described for the first time properties of small molecule antagonists to ROR-beta, an orphan nuclear receptor, at the Keystone Symposium on Nuclear Receptors taking place January 10-14 at Snowbird, Utah. Orphagen’s ROR-beta antagonists suppress critical photoreceptor genes, suggesting that this compound class could treat pathological conditions involving photoreceptor degeneration.
“ROR-beta is highly expressed in the mammalian retina where it has a vital role in the differentiation of photoreceptors, the light-sensitive cells of the visual process,” said Scott Thacher, CEO at Orphagen. “We have obtained small molecule drug-like compounds with potencies of less than 10 nM. To the best of our knowledge, we are the first to identify and characterize such compounds. We speculate that, due to the high level of ROR-beta expression in the photoreceptor, ROR-beta ligands have the potential to modulate photoreceptor survival in degenerative disease. This poster provides the first detailed description of the pharmacology of ROR-beta antagonists. We anticipate that there may be implications for therapeutic use of ROR-beta ligands in other tissues as well.”
Orphagen started its ROR-beta antagonist program with the support of SBIR grants from the National Institute of Mental Health (NIMH) and from NCATS (National Center for Advancing Translational Sciences).
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 ROR-beta, have been developed with partnership revenue and other non-dilutive funding sources, including grants.
For more information, contact: Scott Thacher (858) 481-6191