Orphagen Pharmaceuticals, a privately-held emerging pharmaceutical company, was recently profiled on the front page of BioWorld Today. A featured article on Orphagen Pharmaceuticals, titled “Orphagen Seeks Ligands for Orphan Nuclear Receptors”, highlighted the innovativeness of Orphagen’s approach to nuclear receptor drug discovery and the scarcity of competitors looking at unexplored targets.

“Everyone is willing to jump on a target once it’s got the seal of approval in multiple publications,” Orphagen CEO Scott Thacher noted in his interview with BioWorld Today. “Then you put five or 10 chemists on the problem and you get an edge. But people aren’t working on unexplored targets.” A small number of drug companies have commenced programs focused on adopting orphan targets, but most are dissuaded by the long time horizons.

Orphagen has now identified potential drug ligands to several previously unexplored orphan receptors since it began operations 10 years ago. 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, castration-resistant prostate cancer and endometriosis at Orphagen. Thacher believes there is ongoing interest from drug companies for first-in-class drugs: “If you go into a target before everyone else and take some risk, people will come to you and will partner because they want to be first to market with a new class of compounds.”
Orphagen’s first nuclear program was successfully partnered with a pharmaceutical company in 2008 and the company was able to re-invest proceeds from that partnership into other internal programs. By focusing on early stage discovery, where big pharmaceutical companies are increasingly looking to outsource, Orphagen believes its programs and approach will continue to generate interest.

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