Nivalis began operations in 2008 with an initial focus on administering exogenous GSNO as a potential therapeutic agent in asthma and CF. We completed Phase 1 testing of an inhaled formulation of GSNO , and we applied for, and were granted, Orphan Drug designation related to the potential use of GSNO to treat CF in the United States.
While evaluating GSNO as a potential therapeutic intervention, our discovery efforts resulted in the identification of novel small molecule inhibitors of GSNOR, the enzyme that breaks down naturally occurring GSNO in the human body. These small molecule inhibitors provided advantages as drug candidates, compared to the direct administration of GSNO, because GSNO is limited by route of administration, instability, and lack of intracellular penetration.
In 2010, our development efforts were redirected from GSNO to our GSNOR inhibitors and our discovery efforts focused on optimizing and evaluating drug candidates for oral dosing and expanding our portfolio of GSNOR inhibitors.