BioMarin Pharmaceutical Inc. (BMRN), a maker of drugs for rare diseases, rose to its highest value ever after its medicine for an enzyme deficiency disease met goals in a study, prompting plans to seek regulatory approval next year.
BioMarin jumped 31 percent to $49.07 in New York, its highest price since the shares began trading in July 1999, after releasing results today from the late-stage trial of its GALNS medicine. The Novato, California-based company has risen 43 percent this year.
The disease, mucopolysaccharidosis IVA, is also known as Morquio A syndrome, and it affects an estimated 2,500 to 3,000 patients worldwide, according to BioMarin. It can lead to short stature and problems in the joints, which may inhibit mobility. BioMarin’s drug, a replacement enzyme, helped patients walk 22.5 meters (73.8 feet) farther in six minutes than placebo, the primary goal of the study, the company said in a statement.
“We fully expect GALNS to be approved on the basis of this study,” Cory Kasimov, an analyst with JPMorgan Chase & Co. (JPM) in New York, wrote in a note to clients. “We view the result as clearly positive for BioMarin and see it as a best-case outcome.”
The drug was generally well-tolerated in the trial, the third and final phase generally required for regulatory approval. GALNS also helped with secondary goals of the study, including improvement in a three-minute stair climb and reduction in urinary keratan sulfate levels, a measure of the presence of the enzyme.
“The GALNS clinical program is currently the highest development priority at BioMarin, and this positive Phase 3 study serves as a potentially transformative milestone for the company,” BioMarin Chief Executive Jean-Jacques Bienaimé said in the statement.
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