BioMarin genetic disorder drug meets main goal, shares jump Mon Nov 5, 2012 8:53am EST (Reuters) - BioMarin Pharmaceutical Inc said a late-stage trial of its experimental genetic disorder drug met the main goal of improving patients' walking ability when administered weekly, sending the company's shares up 28 percent in premarket trading. The trial, named MOR-004, showed that weekly doses of the drug at 2 mg/kg improved the six-minute walk distance of patients by 22.5 meters over the placebo. "Given this is the largest Phase III enzyme replacement therapy conducted to-date, being statistically significant on the primary endpoint should strongly support approval," RBC Capital Markets analyst Michael Yee said in a note. However, patients getting the same dose of the drug GALNS every other week, did not show any meaningful or statistically significant change compared to the placebo. "The every other week dosing schedule was a 'long shot,' which if it had worked, would increase patient convenience," Yee said. All GALNS trials conducted prior to Phase III dosed in patients on a weekly schedule, so expectations for (the every other week) arm working were very low. The disorder - Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome - is a rare, inherited disorder caused by the deficiency of a particular enzyme, leading to skeletal dysplasia, short stature and joint abnormalities. The drug was also tested in another extension trial that showed its benefits continued to improve with further dosage, BioMarin said in a statement. GALNS was generally well-tolerated and adverse events were similar to those seen in the trials of other enzyme replacement therapies. There were no deaths and no patients withdrew from the study due to adverse events. BioMarin, which is repeatedly named as one of the most promising biotechnology companies and a potential takeover target for the Big Pharma, now plans to submit marketing applications for the drug, starting from the first quarter of 2013. The company currently has four drugs in the market, two of which are for treating other metabolic disorders caused by the absence of certain enzymes. Shares of the company closed at $37.41 on the Nasdaq on Friday, and were trading at $48 before markets opened on Monday. Reporting by Esha Dey in Bangalore; Editing by Joyjeet Das