About Our Clinical-Stage Pharma Company - Nivalis Therapeutics
Nivalis Therapeutics is developing a novel class of disease modifying therapies that are designed to preserve intracellular GSNO (S-nitrosoglutathione), an endogenous molecule with cell signaling effects that are implicated in the pathophysiology of cystic fibrosis (CF). The Company’s lead candidate, cavosonstat (N91115) initially targets patients with the F508del mutation, the most common disease causing mutation in CF.
GSNO belongs to a class of compounds known as S-nitrosothiols or SNOs. It is the body’s most abundant low molecular weight SNO, and is formed from glutathione and nitric oxide. SNOs are normally present in the human lung but are depleted in the lungs of patients with CF. These depleted levels are due in part to increased breakdown of GSNO by the enzyme GSNO reductase (GSNOR).
In preclinical studies GSNO has been shown to increase the trafficking and maturation of the defective F508del-CFTR protein in airway cells from CF patients. Restoration of intracellular levels of GSNO may therefore represent a novel therapeutic strategy in CF. Consistent with this hypothesis, inhibition of GSNOR with cavosonstat significantly increased and prolonged the function of F508del-CFTR in airway cells from CF patients when added to a CFTR corrector and potentiator.
Nivalis Therapeutics has developed a portfolio of proprietary, investigational, small molecule inhibitors of GSNOR and its lead oral compound cavosonstat, is currently being evaluated in a Phase 2 clinical study in people with CF.
Nivalis Therapeutics is located in Boulder, Colorado.