BioMarin Pharmaceutical Inc announced the initiation of a phase-1/2 clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010.

"We plan to leverage our clinical, manufacturing and regulatory expertise to bring a new therapeutic option to the significant number of untreated Morquio patients around the world," said Henry Fuchs, chief medical officer of BioMarin. "GALNS has been shown in mice to reach important tissues including cartilage and different zones of the bone such as bone marrow, calcified bone and importantly, the growth plate. Our experiments have also shown that GALNS is taken up into human Morquio chondrocytes in vitro and reaches the lysosome to clear keratan sulfate."

Chris Hendriksz, consultant in Metabolic Disorders, Birmingham Children's Hospital, added, "This is a very exciting announcement for our patients who have been waiting so long for a potential therapy. Hope of a therapeutic is now within reach for these patients, and Birmingham Children's Hospital is very honoured to be part of this exciting development."

The phase-1/2 study is designed as an open-label, within-patient dose escalation trial in approximately 20 patients followed by a treatment continuation phase. All patients to be enrolled in the study have already been identified. During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN-110 in three consecutive 12-week dosing intervals. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies.

BioMarin has developed and manufactures two FDA-approved enzyme replacement therapies, one for the treatment of MPS I and one for the treatment of MPS VI. Naglazyme (galsulfase) for MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.

Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disorder characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS).

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.