Results From Phase 2 Clinical Study of 6R-BH4 in Peripheral Arterial Disease Not Statistically S...( NOVATO Calif. Feb. 3 /- BioMarin Phar...)

NOVATO, Calif., Feb. 3 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced results from its Phase 2 multi-center, randomized, double-blind, placebo-controlled clinical study of 6R-BH4 in patients with symptomatic peripheral arterial disease (PAD). There was no statistical significance observed between the 6R-BH4 treatment and placebo groups.

The primary endpoint of the study, peak walking time (PWT), did not show a significant difference between 6R-BH4 and placebo, and the secondary endpoint, claudication onset time, also did not show a difference. Addition of Vitamin C to 6R-BH4 did not improve efficacy on PWT. Endothelial dysfunction evaluated by peripheral arterial tonometry in a subset of patients did not show a significant benefit with 6R-BH4. Urinary protein excretion did not decrease with 6R-BH4 treatment, though there may have been some effect in the subset of patients with microalbuminuria at baseline. 6R-BH4 was well-tolerated in peripheral arterial disease patients and had a safety profile similar to previous studies.

Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin stated, "We are disappointed that the results of 6R-BH4 in peripheral arterial disease were not statistically significant. 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."

Study Design

The Phase 2 multi-center, randomized, double-blind, placebo-controlled study enrolled 190 subjects and was conducted at 31 sites in the U.S. and Argentina. 161 patients comple
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