Orphagen Pharmaceuticals, a privately-held pharmaceutical company, announced today that the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health has awarded the company a highly competitive Phase 1 Small Business Innovative Research (SBIR) grant to identify small molecule ligands for a novel drug discovery target with potential to treat sickle cell disease and other hematological disorders.

An exciting property of the drug discovery target, which is the focus of the NHLBI-funded research, is that it regulates the expression of the fetal form of the critical oxygen carrier, hemoglobin. Normally, fetal hemoglobin is not expressed in the adult. The goal of the research is to identify small molecule drug-like compounds that elevate expression of the fetal hemoglobin gamma chain in order to treat and potentially cure sickle cell disease. The gamma chain of fetal hemoglobin can replace the corresponding beta-chain of sickle hemoglobin, which carries the sickle trait, ameliorating or virtually eliminating symptoms of the disease.

The long-term objective of the research is to investigate the feasibility of treating sickle cell anemia with an orally-available small molecule that regulates this orphan receptor. This work is based on basic studies carried out in the laboratory of Douglas Engel, at the University of Michigan, a pioneer in understanding the mechanisms of hemoglobin gene regulation.

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, 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.

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