This study is the second company-sponsored phase II trial and part of a broader program to evaluate the role of 6R-BH4 in the treatment of different types of cardiovascular diseases.
6R-BH4, commonly known as tetrahydrobiopterin, is an essential enzyme cofactor that is involved in the production of nitric oxide, a molecule that has been shown in clinical studies to play a key role throughout the cardiovascular system, including regulation of endothelial function.
The phase II study is designed to compare oral 6R-BH4 to placebo in subjects with intermittent claudication (IC) caused by peripheral arterial disease. The primary endpoint analysis will compare the change from baseline to week 24 in peak walking time between the 6R-BH4 and placebo groups.
6R-BH4 is the same enzyme cofactor currently being evaluated in BioMarin’s Phenoptin for phenylketonuria (PKU). In March 2006, BioMarin and its partner Serono reported positive results from the phase III study of Phenoptin for PKU. All primary and secondary endpoints of the study were met.