by Dr. Scott Thacher
Orphagen Pharmaceuticals is a privately owned, emerging pharmaceutical company located in San Diego, California. Orphagen focuses on major unmet medical needs by developing innovative small molecule drugs targeting orphan nuclear receptors. The company has hits and leads to several orphan nuclear receptors in various stages of preclinical discovery research. To date, Orphagen has secured over $ 2.7 M in external grant funding and has recently entered into a major partnership for pre-clinical and clinical development of one of its targets.
Orphagen’s accelerated approach to drug discovery is based on the early identification and characterization of small molecule ligands which act on orphan nuclear receptors. Our approach is a departure from current academic and industrial trends in early stage drug discovery. The current, widely used target discovery and proof of principle studies often lack focus. Studies of gene knockouts or broad phenotypic screens don’t directly answer the question of how a specific small molecule to an identified target will actually work in disease models. This conventional approach consumes enormous effort with fairly modest benefits, generating numerous tangential projects if poorly managed.
Orphagen’s uniqueness comes from our focus on a class of unexplored targets, the orphan nuclear receptors, together with our insights into how to identify small molecule ligands for these targets. These targets are part of a very successful target class, the nuclear receptors. Orphagen’s advantage is our expertise to recognize and exploit orphan nuclear receptor targets. Many of these targets have been known for years, but have remained largely unexplored. Orphagen has validated these orphan nuclear receptors as real drug targets. Most notably, we have demonstrated the efficacy of a class of small molecules to a previously ignored target now considered to have great potential for treatment of autoimmune disease. We partnered this program with Japan Tobacco, who realized the value of our first mover and IP position. We are now moving ahead on the same path with additional CNS, ocular, endocrine and autoimmune targets. We recently received Phase 2 SBIR funding for a proposal entitled, “Novel CNS Target for Sleep-Wake and Psychiatric Disorders” to fund preclinical studies on compounds to a novel target class discovered at Orphagen.
Orphagen’s “first-to-ligand” approach has several advantages. First, as we are an early mover in this field, we are able to file for broad patent protection for all ligands interacting with some select targets; second, we are able to rapidly devise molecular and cellular assays that support an overarching proof of principle concept and simultaneously drive small molecule drug discovery; and third, when leads are identified, Orphagen’s fundamental work points directly to in vivo proof of principle studies. Our approach enables our leadership in the discovery and validation of novel and patentable ligands to previously unexplored orphan nuclear receptors. Our business model is to partner these programs at an early stage or to advance them with a combination of internal funds, government and private foundation grants, private equity and pharmaceutical industry support.
Scott Thacher, Ph.D.
CEO and Director
Orphagen Pharmaceuticals
For all inquiries about Orphagen, please contact Dr. Scott Thacher at smt@orphagen.com
www.orphagen.com
