BioMarin's hypertension drug misses efficacy endpoints in Ph IIa trial; shares fall 14.4%

BioMarin Pharmaceutical's blood pressure drug candidate 6R-BH4 has failed to show efficacy in a mid-stage trial. A Phase IIa placebo-controlled double-blind study of the agent in patients with poorly-controlled hypertension demonstrated no statistically significant or clinically meaningful effect of on any efficacy or safety parameters measured, relative to placebo.

On the day of the news, February 20, shares in the Novato, California-based firm fell 14.4% to $17.50. Jean-Jacques Bienaime, BioMarin's chief executive, said: "we are surprised and disappointed by these results, especially considering the numerous encouraging preclinical and clinical studies of 6R-BH4 in diseases with endothelial dysfunction."

"We plan to analyze the data in detail to better understand the background therapy and other characteristics of patients in the study. We have no immediate plans to change the course of ongoing or planned clinical studies of 6R-BH4," he added.