New Drug Encourages BioMarin Investors

San Rafael's BioMarin Pharmaceutical Inc. is reporting some encouraging financial and medical results. BioMarin told investors earlier in November that the company expected to show revenues between $475 million to $510 million in 2012.

The company, which develops bio-pharmaceuticals for diseases and medical conditions, expects some big things in 2013 with the development of its product GALNS, a treatment for Morquio A Syndrome. BioMarin plans to submit a marketing application to the U.S. Food and Drug Administration for the drug in the first quarter of 2013.

“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,” said Jean-Jacques Bienaimé, CEO of BioMarin. “We are applying our track record of success in developing novel treatments for orphan diseases and our existing commercial infrastructure for Naglazyme to bring GALNS to patients as rapidly as we can.”

According to the company's test results, patients dosed with GALNS (BMN-110, N- acetylgalactosamine-6-sulfatase) at 2 mg/kg every week showed an improvement in six-minute walk distance at week 12 compared to baseline and showed continued improvement at week 24. Results also suggested an improvement in pulmonary functions.

The most common negative side effects included vomiting, pyrexia, headache, nausea and coughing.

“The positive results from this pivotal study will help support GALNS as the first therapy available to help the approximate 3,000 people worldwide suffering from MPS IVA — a rare, degenerative, life-threatening genetic condition with no available therapy,” said Hank Fuchs, M.D., Chief Medical Officer at BioMarin. “We are very pleased with the clarity that the MOR-004 study has provided us with respect to the appropriate dosing of GALNS. The weekly 2 mg/kg dose provided a statistically significant and clinically meaningful improvement in the study’s primary endpoint, and positive trends toward improvement in other clinically meaningful endpoints, including three-minute stair climb and pulmonary function tests. By contrast, the 2 mg/kg every other week dose was shown to be similar to placebo on the primary and clinical secondary and tertiary endpoints. We look forward to reviewing the results of this study with regulatory authorities, and applying for marketing authorizations starting in the first quarter of 2013.”

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