BioMarin Pharmaceutical out of Novato, CA says that its mid-stage trial of 6R-BH4 for peripheral arterial disease missed both its primary and secondary goals. Patients taking tetrahydrobiopterin failed to demonstrate a statistically significant improvement over a placebo in peak walking time. Ditto for the secondary goal of onset time for impaired walking.
Researchers were hoping that the drug would offer a source of hope for patients who suffer when the buildup of plaque in their arteries blocks blood flow, spurring symptoms such as numbness. Researchers enrolled 190 patients for the trial. And they are studying the drug's effectiveness for other cardiovascular conditions.
"We are disappointed that the results of 6R-BH4 in peripheral arterial disease were not statistically significant," said Emil Kakkis, M.D., Ph.D., chief medical officer of BioMarin. "We have upcoming data in several BioMarin and investigator-sponsored studies of 6R-BH4 including proteinuria, pulmonary arterial hypertension and 6R-BH4 plus Vitamin C in patients with endothelial dysfunction. Along with the prior results in sickle cell disease, these data will determine the future of the 6R-BH4 cardiovascular program once all the studies are complete."
- check out the press release
- read the story from the San Francisco Business Times