Orphagen Pharmaceuticals, a privately-held pharmaceutical company, announced today that the National Institutes of Health (NIH) National Center for Advancing Translational Sciences (NCATS) has awarded the company $446,635 under the Small Business Innovative Research (SBIR) program to investigate novel small molecule drugs for the treatment of retinitis pigmentosa (RP). Each year thousands of Americans are diagnosed with RP, a complex neurodegenerative disease caused by mutations in more than 90 different genes. RP initially presents as loss of night vision, but progresses to loss of central vision and eventual blindness. There is no cure. Orphagen has identified small molecule compounds that could significantly delay degeneration in as many as forty percent of RP patients.

“Orphagen is proposing an entirely new disease-modifying approach for the treatment of RP, which is an orphan indication,” said Scott Thacher, Orphagen CEO and Principal Investigator of the grant. “Gene therapy has been proposed, but its applicability is limited due to the sheer number of individual genetic mutations that account for various subtypes of RP. By focusing on a vulnerability common to many forms of RP, we are developing a mutation-independent, small molecule approach that could extend the useful visual lifetime of patients by decades.”

Orphagen’s small molecules are the first drug-like ligands to an orphan nuclear receptor highly expressed in the adult retina. With the NIH funding, Orphagen aims to identify more potent compounds for evaluation in RP animal models representative of the human condition. Ultimately, Orphagen is seeking a collaborator to accelerate the drug through clinical development and commercialization. In addition to RP, Orphagen’s compounds may also have application in dry agerelated macular degeneration (AMD), the leading cause of vision loss in people over 60.

Orphagen is a first-mover in the identification of small molecule compounds to three orphan nuclear receptors. Support from the NIH is a critical part of the Company’s overall strategy to discover new classes of drugs and commercialize them through partnerships.

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. Funding from this partnership and other non-dilutive sources, including grants, has advanced several other first-in-class drug discovery programs.

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